august 2007 a thomson centerwatch publication
TRANSCRIPT
U.S. Disease foundations will
invest about $75 million this year
in biopharmaceutical companies
to fund discovery and develop-
ment of new drugs and therapies
for their corresponding diseases.
This year’s figure represents a
10-fold increase since 2000, and
the figure should continue to
rise.This new avenue of industry
funding has been dubbed “ven-
ture philanthropy.”
The amount of money that
disease foundations contribute-
to industry research would not
pay the development costs of
even one drug today. But, the
strategic timing of the funding as
well as the additional resources
and expertise that the founda-
tions provide serve to enable
drug discovery and development
that wouldn’t be undertaken
otherwise and perhaps to speed
that development as well.
Venture philanthropy is a
term coined in the past
few years to describe
the funding that disease founda-
tions provide to biopharmaceuti-
cal companies to research poten-
tial drugs and therapies in their
respective diseases. U.S. disease
foundations’ investment in the
biopharmaceutical industry this
year will be about $75 million—
10 times as much as its investment
in 2000. The figure should contin-
ue to rise.
These foundations believe in
metrics, milestones and account-
ability. They don’t just want to
fund drug development; they
want to change it. In the biotech
sector, they may have met their
perfect match.
But why are non-profits giv-
ing money to for-profit biotechs
in the first place? On its face,
it’s counterintuitive,but a number
of factors have created the perfect
environment for venture philan-
thropy. Disappointed by acade-
mia’s inability and lack of incen-
tive to translate discoveries into
the clinic as well as a reduced
National Institutes of Health
(NIH) budget, foundations real-
ized they had to bring industry
into the equation or let promising
research languish in the lab.
Biotechs have been staring
for years into the so-called
“Valley of Death”—the preclini-
cal stage of development that
presents a too high-risk invest-
ment to attract venture capital-
ists anymore. These companies
couldn’t conduct translational
research in rare diseases without
external funding sources. Foun-
dations are the only organiza-
tions willing and able to fill that
see Venture Philanthropy on page 7
FDA’s Guidance on SupervisoryResponsibilities of Investigators
The FDA’s new guidance
document on investigators’
supervisory responsibilities is
useful for both experienced
investigators and novices who
want to understand FDA’s latest
thoughts about how they con-
duct clinical research.
This article explains the
draft guidance and provides
step-by-step insight for investi-
gators who need to be in com-
pliance with the FDA.
The U.S. Food and Drug
Administration (FDA)
released an important
draft guidance document in
May called “Protecting the
Rights, Safety, and Welfare of
Study Subjects—Supervisory
Responsibilities of Investigators.”
The guidance should be very
useful to investigators whether
those investigators are experi-
enced with or new to FDA-regu-
lated clinical trials. The intent of
this document is to help investi-
gators meet their responsibili-
see FDA Guidance on page 14
Month in Review . . . . . . . . .3 CentreStage Europe . . . . . . 5
EMEA’s Bold Appointment Reflects Agency’s New Broader Role
Grant Opportunities . . . . . .20
Eye On: MelanomaIn the PipelineOpportunities UnderwayTrialWatch
U.S. Disease Foundation Investment
in Biopharmaceutical Industry
Source: CenterWatch estimates, 2007
20072000
$7 million
$75 million
© Copyright 2007. Thomson CenterWatch. Duplication of this publication is prohibited.
Inside this issue...
Venture Philanthropy on the Rise
August 2007 A Thomson CenterWatch Publication Volume 14, Issue 08
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3August 2007CenterWatch
CWWeeklyA bulletin that reports on breaking news in the clinical trials industry. Available every Monday as afax or PDF, an annual subscription is $245 forCenterWatch subscribers.
JobWatchA web-based service and publication listing clinicalresearch jobs, career resources and a searchable resumedatabase. Call Sally Teebagy, (617) 856-1593, or visitwww.centerwatch.com.
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This service provides an international listing ofclinical trials currently enrolling patients, and directories of research centers and industryproviders. Call David Heck, (513) 754-1705.
CW PublicationsState of the Clinical Trials IndustryThe Emerging Markets of Clinical ResearcheDirectory of the Clinical Trials Industry Directory of Drugs in Clinical TrialsBecoming a Successful Clinical Research InvestigatorProtecting Study Volunteers in ResearchA Guide to Patient Recruitment and RetentionThe CRA’s Guide to Monitoring Clinical ResearchThe CRC’s Guide to Coordinating Clinical ResearchVolunteering for a Clinical Trial
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Month in Review
Editor’s Note: Full text articles of these
stories appeared last month in CWWeekly. For
more information about these articles, please
refer to the following CWWeekly issues:
Volume 11, Numbers 27–30.
CROs
Amsterdam-based CRO Clinquest Group has
acquired a stake in Enceladus Pharma-
ceutical, the Netherlands-based developer of
the inflammatory disorder treatment
Nanocort—the product is in phase II trials.
Clinquest also gathered a group of additional
investment groups to back Enceladus as it
brings more products to market. “The partic-
ipation of Clinquest in our activities allows us
to complete the phase IIa clinical proof-of-
concept trial with Nanocort that is currently
ongoing in The Netherlands. Therefore this
means a major boost to our clinical develop-
ment track,” said Bart Metselaar, founder and
chief executive officer of Enceladus. Clinquest
said it believed in Enceladus’ development
pipeline and in the future of Nanocort.
“By entering in this risk-sharing arrangement,
we are able to support the further develop-
ment of Nanocort and the entire liposomal
development platform most efficiently,” said
Cees Wortel, chief executive officer of
Clinquest Group.
Princeton, N.J.-based CRO PharmaNet
Development Group has moved its early-
phase subsidiary, Anapharm, to a new
150,000-square-foot facility in Quebec City.
The site includes laboratories, clinics and
open-plan work stations. PharmaNet said it
hopes to expand the facility by adding 200
beds in the future. The company expects to
open another 150-bed, early-phase unit in
Toronto in July. Anapharm is PharmaNet’s
only business that conducts phase I and bioe-
quivalence studies now that the company has
sold its entire Miami operation, which was
run by the former SFBC. “The investments in
Quebec City and Toronto reflect our confi-
dence in our Canadian operations and pro-
vide the room for future expansion as
Anapharm continues to grow,” said Mark
Ianni, president, early stage development at
PharmaNet Development Group.
Maryland-based CRO United BioSource
Corporation (UBC), in conjunction with
eClinical firm Medidata Solutions, and the
adaptive trials tools and modeling company
Tessella, have joined together to educate the
industry on the use of adaptive clinical trials in
drug development. The companies are pro-
ducing a series of free webinars—six in total—
that will feature expertise from industry pro-
fessionals and regulatory authorities on how to
overcome some of the challenges of these stud-
ies. The first webinar, entitled “Operational
Challenges and Strategic Planning,” will be
held on July 11th and can be viewed online at
www.enablingadaptivetrials.com. “Even with
the right regulatory environment and the right
technology, sponsors who are looking to exe-
cute adaptive clinical trials need to devote time
to planning for this change to the clinical
process,” said Michael Borkowski, general
manager of clinical technologies at UBC.
Wayne, Pa.-based CRO Encorium Group has
inked a $1.7 million deal to manage a phase II
trial in major depressive disorder for an
unnamed biotech company based in Japan.
Encorium will provide site management and
monitoring, data management, biostatistical
support and medical writing for the study.
Encorium stated it intends to pursue further
opportunities in the Japanese market and has
made that market one of its key long-term
business strategies. Recently, the company
signed a $3.8 million contract to conduct
The CenterWatch MonthlyISSN 1556-3367
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Stephen DeSantis
Research Mary Jo Lamberti, Ph.D.Paul Dewberry
Drug Intelligence Tracy Trundle
Trial Watch Tamar Skowronski
Design Paul Gualdoni
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Month in Review
see Month in Review on page 4
Month in Review
Thomson4 August 2007
phase IIb clinical trials with a new antiviral
agent for the treatment of shingles (herpes
zoster).
North Carolina-based CRO Quintiles is con-
solidating its U.S. global central laboratory
facilities and clinical development services
offices in Smyrna and Atlanta, Georgia, into a
200,000-square-foot facility based in Marietta.
The expansion will nearly double Quintiles’
research capabilities in the state. The move is
expected to cost about $19 million. The CRO
received an $11 million incentive package and
collaborative support from the Georgia
Department of Economic Development, the
Georgia Research Alliance, the Georgia Cancer
coalition and Cobb County, Georgia. The
company plans to add an additional 400 jobs
in the next four or five years.
Mumbai, India-based contract research
organization (CRO) Vedic Lifesciences has
established a partnership with Amarex, a
CRO based in Maryland. The deal calls for the
two companies to collaborate on clinical
development and regulatory affairs services
for botanical products in the United States
and India. Amarex said its focus on phase I
through IV, combined with Vedic’s expertise
with pre-clinical services and botanical com-
pounds will help both companies to tap into
the U.S. botanical studies market. Vedic
Lifesciences has completed 24 studies, with a
specialty in herbal medicine in India.
Technology
Ft. Lauderdale, Fl.-based eClinical company
OmniComm Systems has completed the
second stage of its Oracle Clinical integration
project. Stage one of the project allowed
OmniComm System’s clients to use the
company’s TrialMaster to export EDC data
into a standard format for importing into
Oracle Clinical. Stage II of the integration
projects gives clients access to Oracle’s global
library of trial referencing to design and build
custom trials using OmniComm’s TrialMaster
product.
DSG, a Malvern, Pa.-based eClinical company,
has established a partnership with Solomon
Group Ltd. (SGL), a CRO based in Taiwan,
with major operations in Shanghai and
Guangdong, China. DSG will provide SGL
with data management and electronic data
capture services to clinical trials in China. SGL
will sell, support and host DSG’s software
solutions eCaseLink, with Chinese language
capabilities. “Our new Chinese language EDC
capability will save our clients both time and
money when conducting trials in China
because users can work in their native lan-
guage without requiring translations of clini-
cal data,” stated DSG’s chief executive officer
Tony Varano.
Patient Recruitment
The Cystic Fibrosis Foundation (CFF) hopes
to increase the number of patients enrolling in
CF clinical trials by 200% through a new
awareness initiative being rolled out across the
organization’s network of 115 affiliated cen-
ters in the United States. Part of the initiative
calls for the creation of a toll-free information
hotline, online trial awareness information
and a series of patient educational materials.
The CFF is a non-profit organization whose
mission is to find treatments and improve care
for patients afflicted with the life-threatening
disease. CF affects 30,000 adults and children
in the U.S. alone. Finding enough subjects to
conduct robust clinical trials in CF is difficult
because of its rarity, yet results of a survey con-
ducted by the organization found that most
CF patients were not aware of the shortage of
trial participants. Nearly all of the drug devel-
opment research in CF is supported by the
foundation.
Accreditation
The Association for the Accreditation of
Human Research Protection Programs
(AAHRPP) has accredited its largest group of
institutions at one time in its six-year history.
In total there were 16 organizations that
gained AAHRPP approval. The certification is
valid for three years. “This is the largest num-
ber of accreditations we have awarded at one
time and we expect the numbers to continue
to grow as we see an ever expanding list of
applicants,” said Marjorie Speers, Ph.D., exec-
utive director, AAHRPP. The list of hospitals
or health systems that received full accredita-
tion includes Cincinnati Children’s Hospital,
MetroHealth System in Cleveland, Ohio, and
the University Hospitals Case Medical Center
in Cleveland. Among the list of universities
were: University of Cincinnati, University of
Kentucky, University of Utah, University of
Rochester, University of Southern California,
and Virginia Commonwealth University in
Richmond. Three Veterans Administration
facilities were granted full accreditation,
including the Cincinnati Department of
Veterans Affairs Medical Center, Department
of Veterans Affairs Salt Lake City Health Care
System, Veterans Affairs Pacific Islands Health
Care System in Honolulu,VA Palo Alto Health
Care System, and Veterans Affairs Healthcare
Network Upstate New York at Albany. Austin,
Texas-based IntegReview Ethical Review
Board also received full certification. The
Lexington VA Medical Center received quali-
fied accreditation, which means it met the
standards with only a few minor deficiencies
in the administrative processes, not involving
patient care. It will still be accredited for three
years and if these deficiencies are corrected
within that time, the institution will receive
full accreditation status.
Month in Reviewcontinued from page 3
CentreStage Europe
5August 2007CenterWatch
Professor Hans-Georg Eichler, a
clinical pharmacologist from the
University of Vienna, took on the
newly created post of senior medical officer at
the European Medicines Evaluation Agency
in February. In an exclusive interview with
CenterWatch, he describes the agency’s
broadening scope and how this will affect the
process of obtaining marketing authoriza-
tions for new drugs in Europe.
There wasn’t universal rejoicing when
the EMEA was born in 1995. Many politi-
cians within the then-15 member countries
of the European Union were skeptical
about the need for a pan-European agency,
believing it could undermine the status of
their own national authorities. Meanwhile,
some working in the pharmaceutical
industry were concerned that the new
organization might add a layer of costly
bureaucracy without necessarily diminish-
ing the difficulties in getting products
approved through the various national
procedures.
But in the first 12 years of its existence,
EMEA has shown that it can assist the
industry in finding a simpler and more
readily navigable route to bring medicines
to the market simultaneously throughout
the EU. Since then, the community has
expanded to its current 27 member states,
and the questions being asked of all phar-
maceutical regulators have become more
difficult to answer. So the creation of the
new post of senior medical officer was one
response to the agency’s evolving role.
“When the EMEA was set up, there were
a lot of people who thought it would never
fly, but it has taken off and it is doing its
duty. But it has been realized that we now
have to take its work to the next level,”
Eichler explained. “Legislation passed by
the European Parliament two years ago
gave new tasks to the agency’s secretariat. In
the past, its job was just to give administra-
tive and technical support to the commit-
tees [made up of experts representing each
member state], now it has a role to also
offer scientific support.”
The agency’s broadening role is reflect-
ed in the changes to its expert committee
system. At first, there were only the com-
mittee for proprietary medicinal products
(CPMP, now renamed the CHMP, or the
committee for medicinal products for
human use) and its veterinary products
equivalent, the CVMP. Then new commit-
tees were formed to deal with orphan
drugs, herbal products and, as of last
month, a committee for pediatric products.
Within the next year another committee
will be set up to look at so-called advanced
therapies.
As the number of committees grows, so
does the risk that they may reach conflict-
ing decisions on matters where they have
adjacent or overlapping interests. Rather
than risk sending out contradictory mes-
sages to applicants, Eichler has been
appointed with responsibility for identify-
ing and resolving any such conflicts.
But as well as monitoring its own per-
formance, the agency—together with the
national agencies in each member state—
wants to change the way medicinal prod-
ucts are assessed in Europe.
“It is no longer enough for the agency
to have a passive role in just accepting
applications as they come in. We also have
a responsibility to explore how we can
improve our work, to identify strengths and
weaknesses and to help drive forward a reg-
ulatory process that is more transparent,
more predictable and more consistent,” he
said. “Part of my remit is to coordinate this
research agenda. I will be looking for spe-
cific projects that will help the agency to
answer some vital questions. How can the
process for assessing risks and benefits be
improved? Are there new technologies out
there that can be applied? Also, communi-
cating to the wider world about the risks of
drug treatments is an important issue – so
how can we communicate the fact that
sometimes there is no ideal solution to the
problems of balancing risks and benefits.”
Eichler arrived at the agency’s head-
quarters in the redeveloped former docks
area in the East End of London, having held
the post of head of clinical pharmacology
at the Medical University of Vienna. After
training as a physician, his career has
included stints in research, industry and
public policy as a member of advisory
boards to the Austrian Ministry of Health
and a national delegate on EMEA’s orphan
products committee. He claims to have had
little difficulty adjusting to living and
working in a new country, and his flawless
English gives an indication why. His lan-
guage skills have been polished by his expe-
rience as a postgraduate student at the
University of Surrey, just south of London,
and as a visiting scientist at Ciba Geigy’s
former UK laboratory in Horsham and
Merck’s headquarters in New Jersey.
In his new post, Eichler reports directly
to the agency’s executive director, Thomas
Lönngren. His responsibilities lie outside
the organization’s existing departmental
structure, and he has no role in its day-to-
day operational activities of processing
license applications.
So how has he occupied his time in his
first months as senior medical officer? His
immediate task, he said, was to get proper-
ly oriented.
“When you join an organization with
this level of complexity, your first priority is
to figure out your way around it. Yes, I had
been involved already with the agency as a
member of its committees, but that only
gives you a superficial perspective on the
workings of an organization that forms
see Eichler on page 6
EMEA’s Bold Appointment Reflects Agency’s New Broader Role
CentreStage Europe
Thomson6 August 2007
part of a network with a number of con-
stituent parts—the Commission, the
national competent bodies [i.e., national
licensing authorities], industry, patients’
organizations and so on,” he said.
More recently, his focus has been on
efforts to identify and prioritize the issues
that will be tackled in this new research
agenda. He will also be seeking suitable
partners with which to carry out these
studies. He has no staff reporting directly to
him and, as he points out, within the busy
agency “there aren’t a lot of people sitting
around waiting for you to give them work
to do.” So he hopes to set up collaborative
projects with the various national agencies
and also establish much stronger links with
the academic world. Finding the resources
needed to carry out these studies promises
to be his biggest challenge over the next few
months. The agency has no guaranteed
budget for its research.
If Eichler succeeds in his mission to
strengthen the scientific foundations on
which the agency arrives at its scientific
opinions, what effects will this have on the
clinical trials process?
“You have to look at this research in a
broader context. What does industry want
from a regulator in developing new drugs?
The main thing is predictability. So by
refining our methodologies to ensure that
we produce more consistent results, then
our work will enhance that predictability,”
he noted.
Although the direct costs to industry of
license applications won’t change, he is
hopeful that improving the efficiency of the
approval process will improve. “With all the
regulatory agencies, EMEA, FDA and oth-
ers, there is a high rejection rate for license
applications, so a company must go
through the whole drug development
process only to fall at the last hurdle. I am
sure that many of those failed applications
could be avoidable. One thing that we will
need to look into is how to get companies
to ask us for scientific advice earlier in the
process. Making our actions more pre-
dictable will also help because it could
mean that the developers may know what
the outcome is going to be before they com-
plete the work. If they know where we put
the goalposts it will be a lot easier for them
to score.”
However closely companies and regula-
tors work together, there is always the pos-
sibility of unexpected events such as the
TeGenero incident in March 2006. The
health problems suffered by volunteers in a
phase I trial of the company’s product
TGN1412 prompted several European
states to introduce unilateral measures to
protect the safety of trial subjects and gave
new impetus to plans for revising the EU
Clinical Trials Directive.
EMEA will be hosting a meeting later
this year in which EU experts will assess
measures to minimize the risks to partici-
pants in phase I studies. It has also pro-
duced guidance for companies on strategies
for dealing with potentially high-risk prod-
ucts.
“The bottom line is that there is a need
for stronger supervision of these products.
Everyone is in agreement with that idea, but
the problem lies in how you define a high-
risk product. We are well aware of indus-
try’s concerns about scope creep – that risk-
averse regulators will label everything as
high risk and insist on the strictest controls.
Our guidelines provide criteria for classify-
ing new products, and I think this offers a
well-balanced view on the issue.”
Within the agency there is an apprecia-
tion that Europe is competing with other
regions in an increasingly global clinical tri-
als industry. But there are matters in which
cooperation between rival regulatory
authorities can benefit both sides.
“We are in discussions with the FDA in
an attempt to achieve a harmonized
approach. There have been occasions when
we have told companies preparing for a piv-
otal phase III study – ‘You must do it in this
way,’ while the FDA has been saying some-
thing completely different. We both recog-
nize that this doesn’t make the drug devel-
opment process any easier and so we are
trying to develop a common approach, par-
ticularly in the areas of orphan and pedi-
atric products,” Eichler said.
Another indication of this shared
approach to drug regulatory issues on both
sides of the Atlantic came with the FDA’s
appointment of Dr. Janet Woodcock as its
chief medical officer just a few weeks before
Eichler arrived in London. In her new post,
Woodcock takes responsibility for the
Critical Path Initiative designed to improve
the scientific basis of medical product
development and her responsibilities cover
much the same ground. “Given that both
sides recognize that we have an important
role in fostering drug development for the
benefit of public health, we expect to be
working closely together in the near
future,” he said.
—John Bonner
Eichlercontinued from page 5
Industry Reports
7August 2007CenterWatch
funding gap, but not without careful consid-
eration and a firm understanding of what
they want from biotechs in return.
But, disease foundations do not just give
money to industry. Money doesn’t solve
problems, such as protocol development
and patient recruitment, on its own. In addi-
tion to money, disease foundations provide
valuable resources and expertise to further
de-risk the joint venture between them and
biotech.
This article will focus on five of the
leading foundations that have a venture phi-
lanthropy component. Their programs
promise to be an increasingly important
part of the drug development landscape in
the decade to come. They are: The Cystic
Fibrosis Foundation, Multiple Myeloma
Research Foundation, Muscular Dystrophy
Association, Juvenile Diabetes Research
Foundation, and The Michael J. Fox
Foundation for Parkinson’s Research. Their
work and money will have positive implica-
tions not only for patients who will benefit
from the therapies that result, but also for
the industry as a whole.
Lost in TranslationThese five foundations have all created pro-
grams targeted to industry because, after
years of funding academic research, they
learned that it is very difficult to translate
promising academic discoveries into the
clinical setting, or they were aware of the
venture capital funding gap that had created
the “Valley of Death,” or they knew that NIH
wasn’t going to step in with funding, or
some combination of these factors. Potential
drugs and therapies getting lost in transla-
tion—or not even getting that far—drove
some foundations to re-evaluate their grant-
ing mechanisms and start new programs to
ensure industry could get into the mix.
While most foundations will continue to
award the larger portion of their research
funds to academia—where important dis-
coveries are made—the percentage of foun-
dation research funds targeted to industry
will rise.
Sharon Hesterlee, Ph.D., vice president
translational research, Muscular Dystrophy
Association, said, “We’d get progress reports
every year, and I noticed some grantees all
started to sound familiar. We had one per-
son in particular who was developing a gene
therapy approach for a disorder using all
kinds of techniques—vectors and parti-
cles—and had had reasonable success using
animal models using various techniques,
and every year we got a new grant [applica-
tion] for a new technique. I pulled a lot of
these files out and started calling the investi-
gators to find out what are the next steps,
and if you’re not planning to move forward
with therapy development, why not?
“It very quickly became clear that the
investigators were interested in doing some-
thing with this therapy but didn’t know
what, so what they were doing was just send-
ing new grants that incrementally improved
on the technology. They were very much
driven by the technology and whatever is the
chief hypothesis in the lab and keeping the
lab funded. They didn’t really know how to
take the next step forward and develop a
new therapy, especially with a new indica-
tion, a drug that hasn’t been in humans
before,” said Hesterlee.
Peter Lomedico, Ph.D., director of
industry partnerships at the Juvenile
Diabetes Research Foundation, also realized
that various forces were working against
successful translation. “A few years ago, we
looked at the research portfolio and realized
that there was considerable effort on the
academic side, the discovery research side,
and good progress was being made there,
but in terms of translating the discoveries,
getting more things into the clinic and driv-
ing toward development with the eventual
goal of reaching the marketplace, there was-
n’t a whole lot there in terms of trying to
deal with that. Certainly the changing
investor climate for biotech, changes in the
venture capital community and also within
big pharma really made clear that there is
this gap, whether you refer to it as ‘The
Valley of Death’ or some other phrase.
Clearly there is a challenge for an organiza-
tion like ours to see the fruits of the discov-
ery research mostly coming from the aca-
demic side and how to get that translated
over to companies so they can take on pro-
grams and drive through clinical develop-
ment and through the approval process,”
Lomedico said.
Kathy Giusti, founder and chief execu-
tive officer, Multiple Myeloma Research
Foundation, has a unique vantage point: She
has worked in the pharmaceutical industry,
worked for government, worked with acade-
mia, and is a multiple myeloma patient.“I’ve
seen all elements of drug development from
NIH to industry to academia to being a
patient myself, and I think it’s that that has
given me this ability to understand nobody
has bad intent, it’s just an old and broken
system that really needs to be updated. I
hope that we can play a major role in that,”
said Giusti.
It’s Not Just About the MoneyWhile each foundation provides funding to
biotechs to give them the ability and the
incentive to conduct research that could lead
to a cure of their disease, just throwing
money at the problem of translation isn’t
going to fix it. These foundations are well
aware of the fact that they bring a lot more
value to the biotechs than simple dollars and
cents can provide. CFF and MMRF have
see Venture Philanthropy on page 8
Venture Philanthropycontinued from page 1
patient databases and work to match
biotechs with sites where they will find the
right patients to study in. All of them have
access to deep expertise in their disease as
well as the knowledge of where their money
will potentially do the most good toward
reaching the goal of getting drugs and ther-
apies approved.
Deborah Brooks, president and chief
executive officer at The Michael J. Fox
Foundation for Parkinson’s Research,
described the role of funding as a part of the
value of what her foundation contributes to
industry.
Brooks said, “Funding is an aspect of
how we get defined, but it’s not the only way
we engage and provide value to industry. We
use our capital, in particular with industry,
through the lens of how we can keep PD
[Parkinson’s Disease] prioritized in their
portfolio of activity—that’s everything from
supporting specific therapeutic ideas as well
as investing in tools that de-risk broadly. We
look at the drug development pipeline, and
we try to understand the landscape as it
relates to Parkinson’s in as much detail as we
can. We probably have among the best cat-
bird seats in the world because it’s our daily
focus. We think about what’s missing, what
are we learning, what do we know, what
action can be taken, and how can we use our
resources and expertise and connect the
dots? And that translates into a portfolio of
activity. We bring the money, but we’re also
bringing the expertise in that access to addi-
tional information and resources and prob-
lem solving.”
At the BIO2007 conference held in
Boston, Robert Gallotto, vice president,
strategic planning and alliance manage-
ment, Altus Pharmaceuticals, said during
the “Venture Philanthropy and Foundation
Deals” panel presentation, “Capital is only
one part of the equation. It’s much more
the intellectual capital that was important
for us.”
Daniel Grau, chief operating officer of
CombinatoRx, a Cambridge, Mass.-based
biotechnology company, said at BIO2007,
“We also look for access that most venture
philanthropists can provide in spades in their
various indication fields, to various tools and
to leading advisors and expertise. This is a
very important benefit.” CombinatoRx is a
biotechnology company focused on develop-
ing new medicines built from synergistic
combinations of approved drugs. In less than
six years, the company has discovered and
advanced into clinical trials a portfolio of six
product candidates targeting multiple
immuno-inflammatory diseases and cancer,
at a total investment, including development
of its proprietary screening technology, of
less than $50 million. The company has four
venture philanthropy deals ongoing, one
each with Accelerate Brain Cancer Cures,
Cystic Fibrosis Foundation, CHDI
[Huntington’s Disease], and the Spinal
Muscular Atrophy Foundation.
Added Grau, “What’s been beneficial to
us is that in some cases with these deals
we’re moving deeper into a therapeutic area
that we already have a presence in.
Oncology would be an example of that. We
were not active in glioblastoma multiforme,
brain cancer, but we had an existing set of
products in the cancer field, so this allowed
us to go deeper into that area. In other cases,
we’re diversifying and building out new
R&D verticals.”
In addition to bringing money, resources
and expertise, foundations make sure that
the companies they give funds to are meet-
ing their obligations. The foundations have
built in the power to withhold payment for
performance that doesn’t measure up, and
they aren’t afraid to use it. Grants are mile-
stone-driven, and each foundation has met-
rics they use to judge a company’s perform-
ance on a grant-by-grant basis.
“We set the milestones in a logical
sequence. The point was that if you can’t
accomplish this step, there’s no sense mov-
ing forward or providing further money.
That gives us tighter control. With an aca-
demic grant, we get yearly progress reports.
There’s some merit to that maybe, but with
a for-profit company, we’re taking donor
money and giving it to a for-profit company
and we feel we have a fiduciary obligation to
make sure we have a lot of oversight over
how it’s being spent. It’s worked out well. We
have a very good idea of where the company
is. Often, milestones are delayed. That just
happens, and we have the option of with-
holding payments when milestones aren’t
accomplished. For a very small startup com-
pany, that can mean that people don’t get
paid. I’ve found that if you want something
done in record time, you can use that carrot-
stick approach,” said MDA’s Hesterlee.
Biotech AdvantageBiotechs often find themselves in a Catch-22:
If they do not have proof-of-concept data,
they cannot attract venture capital, but if
they do not have capital, they cannot supply
those data. To compound the difficulty of the
situation, when biotechs work toward getting
these data with big pharma, they have to
choose between getting their research costs
funded or dilutive activities such as giving up
their product rights, but venture philanthro-
py money does not require that biotechs
make that choice. Sometimes foundations’
money is even the difference between a
biotech’s existing or not.
“Venture philanthropy has actually
played a very important role for us in build-
ing our busines,” said Grau. “Funding from
the bench through patient proof-of-concept,
plus commercial rights. That’s one of the
crystal clear virtues of working with venture
Industry Reports
Thomson8 August 2007
Venture Philanthropycontinued from page 7
Industry Reports
9August 2007CenterWatch
philanthropists. If you were to work with a
specialty pharma or large pharma organiza-
tion, it’s very unusual that you would have
their commitment to cover all your research
costs and at the same time they would pro-
vide you with all the product rights. Those
things typically don’t go together, but they
can go together in the venture philanthropy
space, and that’s a very interesting opportu-
nity for biotech companies.”
Altus Pharmaceuticals’ Gallotto also
described the importance of his company’s
venture philanthropy deal with CFF. Altus
Pharmaceuticals is focused on developing
and commercializing novel protein thera-
peutics for patients with chronic gastroin-
testinal and metabolic diseases. The compa-
ny is developing a portfolio of products
based on its novel protein crystallization
technology and has the potential to redefine
the use of biopharmaceutical proteins for
gastrointestinal or metabolic diseases by
allowing the oral and parenteral delivery of
high value protein replacement products.
When Altus started working with CFF in
2000, it was a pre-IND company and didn’t
have the infrastructure to develop an effec-
tive product. It was in the typical biotech
catch-22. CFF committed funding in the
early stages. “They were helpful for us to
bridge the gap from an idea to animal proof-
of-principle data with a $1 million grant in
2000. From there, in 2001 we were able to
develop a broad collaboration and alliance
agreement with the CF Foundation. It allows
the CFF Foundation to fund up to $25 mil-
lion in milestone-based grants. This was
important for us because most of that fund-
ing was done prior to phase II . . . This cash
was non-dilutive. The ability to retain rights
to your molecules is uniquely important to
any biotech company,” said Gallotto.
Although this kind of money is a very
large investment for a foundation, it is rela-
tively small in the investment world. But,
aside from making translational research
possible, venture philanthropy and founda-
tion resources can attract venture capital far-
ther down the road.
“Venture capital organizations like the
fact that you’re working with these groups
that have unique perspective of the disease
that otherwise companies themselves would
not have. You have unique access to physi-
cians, nurses, patients who have a complete
understanding of these diseases and under-
stand how to design protocols. This was
very important from our perspective when
we went later on to raise outside capital from
the venture community. We took this collab-
oration with the CF Foundation with the
animal data that we had and our plan for
our IND, and we were able to raise $51 mil-
lion at the end of 2001 through a Series B
round. Without the CF funding prior to
IND, to phase I being completed, to the end
of phase II being completed, we may never
have gotten as far as we did in terms of this
product or even as a company with respect
to even existing,” said Gallotto.
Altus finished its initial public offering
(IPO) last year and has initiated phase III
clinical trials of its enzyme replacement
therapy in people with cystic fibrosis and
pancreatic insufficiency.
Foundation ModelsAlthough venture philanthropy is a collec-
tive term to describe disease foundation
investment in biotechnology for the devel-
opment of drugs and therapies in their
respective indications, it actually takes many
forms. Following is a description of the dif-
ferent foundation models.
Cystic Fibrosis Foundation (CFF).
Year founded: 1955
Money invested in research since
founding: $735 million (through 2006)
Industry program: Cystic Fibrosis
Foundation Therapeutics
Development Program (1998)
Money invested in industry drug
discovery & development since 1998:
$230 million
Money earmarked for industry drug
discovery & development in 2007:
$43 million
Biotech collaborators: 24+
Drugs in CF pipeline: 30+
CF patients in database: 23,000
Sites in clinical trial network: 64
The gene that causes cystic fibrosis was dis-
covered in 1989, and researchers know much
about the basic defect. This is the good news.
The difficulty getting pharmaceutical compa-
nies or biotechnology companies interested
in researching therapies for the disease is that,
even though it is the most common fatal
genetic disease in the U.S., it affects only
30,000 in this country and 70,000 worldwide.
Beall of The Cystic Fibrosis Foundation knew
that the foundation would have to establish a
new business model for his non-profit if it
wanted to interest pharma and biotech to
enter the field of cystic fibrosis.
“We felt that the best thing we could do
was reduce these companies’ financial risk by
supporting early stage drug discovery. Our
business model is to effectively translate the
understanding of the basic defect in the gene
into new therapies for our patients,”said Beall.
He began in 1998 by investing in a thera-
peutic development program comprising a
network of seven clinical research centers,
designated as the Cystic Fibrosis Foundation’s
Therapeutics Development Network that
see Venture Philanthropy on page 10
specialize in conducting phase I and II stud-
ies for treatment of cystic fibrosis. The net-
work also had a coordinating center. In
1999, CFF received a $20 million grant from
the Bill and Melinda Gates Foundation, and
in 2000, the foundation launched Cystic
Fibrosis Foundation Therapeutics (CFFT),
its non-profit drug discovery and develop-
ment affiliate that administers contracts
with biotech companies. TDP is a program
of CFFT.
Initiation of the collaborations with
industry, level of investment and deal struc-
ture vary. CFFT agrees to fund, on a match-
ing basis, the development of products/com-
pounds for the purpose of identifying new
drugs for cystic fibrosis. CFFT may approach
a biotech studying a compound being stud-
ied in another indication and give that com-
pany anywhere between $50,000 and $25
million to test it in cystic fibrosis. Some deals
with biotech are royalty-based—if the drug
is approved, the foundation gets a multiple
back. Sometimes the deals are a combination
of royalties and a multiple. The CFF also
requires additional compensation for
extraordinary sales results. If there is a sus-
pension in development activities, the CFF
obtains worldwide rights to develop the
product with agreement to negotiate royal-
ties to the original partner after CFF’s invest-
ment is returned.
Negotiated portions of the monetary
awards from CFFT are dependent on the
accomplishment of predetermined, success-
driven milestones. CFFT may withdraw
from the project under certain circum-
stances, including failure to achieve mile-
stones. CFFT requires the establishment of
a Scientific Advisory Council made up of
CFFT, sponsor and joint representatives.
The foundation has not taken any equi-
ty in any of the companies to this point. The
foundation re-invests all the money it
makes into more research. When a drug was
approved by the U.S. Food and Drug
Administration [FDA] a few years ago, the
foundation flipped the royalties and sold
the future rights to it for about $18 million
and used that money for the initial capital-
ization for its high throughput screening
program.
CFF has also created a new funding
vehicle, called the Technology Access
Program, for emerging drug discovery tech-
nology. The foundation awards up to $1
million per year for three years to focus on
new or complementary approaches to tar-
gets that may promote maturation of F508
CFTR. The cystic fibrosis transmembrane
conductance regulator (CFTR) is mutated
in patients with CF. The most common CF-
associated mutation is deletion of pheny-
lalanine at residue 508, CFTR delta F508.
Two awards were made last year.
A biotech or pharma that has a product
or idea it wants to test in cystic fibrosis can
use the network run by the Therapeutic
Development Program. CFF helps compa-
nies conduct clinical trials and does the
early phase drug discovery programs.
In February, CFF expanded its investiga-
tive site network, run by its Therapeutic
Development Program (TDP), with a $3
million award distributed among 45 new
research centers in 20 states nationwide.
The money is being used to build each site’s
infrastructure, expand its staff, or help with
patient recruitment operations. It is a two-
year program, which means sites will have
to perform studies and follow Good
Clinical Practice (GCP) procedures in order
to stay in the network the following year.
CFFT has established an operational center
in Seattle to coordinate with the network,
which is staffed with more than 40 person-
nel and can assist in protocol development
and monitoring. The investment is meant
to prepare the new sites to become part of
CFF’s existing network of 18 sites. By 2009,
CFFT hopes to have all 45 sites, plus addi-
tional sites added in the coming years.
“People like our business model because
we hold ourselves accountable. We are the
only voluntary health organization that
measures success in terms of our pipeline.
The pipeline itself is our metric for future
success. Our medical programs have con-
tinued to grow at a rate of nearly 19% for
each of the last three years,” said Beall.
Multiple Myeloma Research
Foundation
Year founded: 1998
Industry program: Lead grant
Research entity: Multiple Myeloma
Research Consortium (MMRC,
2004)
Academic centers in MMRC: 13
Money earmarked for drug develop-
ment in 2007: $11 million (indus-
try share=$2 to $3 million)
Biotech collaborators: 30+
Compounds: 37 single agents/
combinations
Patients in database: 30,000
Tissue bank: 1,000 bone marrows
and matching peripheral blood
In 1998, when Kathy Giusti founded the
Multiple Myeloma Research Foundation,
fewer than five drugs were being evaluated
in multiple myeloma. Affecting 45,000 peo-
ple in the U.S., it is a heterogeneic disease.
For this reason, Giusti believes that “myelo-
ma is probably going to be cured by a cock-
tail of therapies. It may not be exactly the
same for every patient. As a result, we need a
significant repertoire of really good, targeted
therapies from biotech and pharmaceutical
companies. And unfortunately with mini-
Venture Philanthropycontinued from page 9
Industry Reports
Thomson10 August 2007
mal funding from NIH or reduced funding
from NIH and the difficulty in raising [ven-
ture capital] funds, we thought we needed to
step in to build the bridge between the vali-
dation of drugs and clinical trials.”
In 2004, Giusti founded a non-profit
affiliated with MMRF called the Multiple
Myeloma Research Consortium comprising
13 academic centers that have signed mem-
bership agreements. MMRF conducted a
request for applications for centers. Those
that had the highest number of patients,
clinical expertise, in the right geographic
area and had a good blend of translational
research were the ones selected. MMRF
developed the model and all policies and
procedures for the consortium, and the
foundation raises the money to fund it.
Each center is held accountable to specific
milestones and timelines. Metrics include:
the time that a trial comes to MMRF as a
concept to the point which a protocol is
written to the time of IRB approval to first
patient enrolled to study close. Each center
is given a scorecard rating its performance
MMRF employs five people dedicated to
facilitating clinical research conducted at
the consortium’s academic centers and is
able to call on the skills of multiple myelo-
ma experts as well. Giusti has identified
problem areas in the clinical research
process, including protocol development
and contracting. For both areas, Giusti
offers solutions to speed the drug develop-
ment process.
“One of the major obstacles in drug
development is developing a protocol. A
new biotech may not have the expertise to
develop a protocol in myeloma and to
understand which sites would be best for
them to work with. We immediately offer
them that expertise. We work with the gen-
tlemen who have done all the leading
myeloma trials who can provide all the pro-
tocol expertise. We also know exactly what
trials are going on at all of our sites, and if
competing trials are happening. We can tell
our pharma partners who we think can
move quickly and would have capacity to
get this trial done.
“Contracting is one of the major obsta-
cles in getting trials done quickly, so we
developed master templates here so that we
could speed the contracting process. I hired
my own attorney and I pay outside counsel
to get all the contracting done,” said Giusti.
Communication is key for the centers,
which are required to be on monthly tele-
conferences and at quarterly face-to-face
meetings. On calls and at meetings, there
are usually also companies presenting their
compounds or discussing the protocol or
patient enrollment. Ongoing trials require a
weekly teleconference.
A unique feature of what MMRF offers
its consortium is access to its tissue bank,
patient database and its patient navigator
program.
Much of what MMRF does entails
matching biotechs with the right academic
center. The foundation will allocate $11
million for research investment this year.
Two to three million dollars of that amount
will be allocated to MMRF’s lead grant pro-
gram, which funds go directly to biotechs
that MMRF selects from those that apply.
“Speed to market is everything we’re
about because no company is going to
come to myeloma because it’s a big market.
But, they would come here because they
have a foundation like ours that can facili-
tate the process so much that you say, ‘We
could get our drug to market faster because
this group will help us from concept to
enrollment to the education of the drug,
and even with FDA relations because we do
FDA roundtables as well. We take great
pride in being an end-to-end solution for
biotech and pharmaceutical companies,”
said Giusti.
Muscular Dystrophy Association
Year founded: 1950
Money spent on research since found-
ing: $670 million
Industry Program: Translational
Research Program (TRP)/corporate
grant (2004)
Money invested in industry since start
of TRP program: $5.5 million+
Projected industry funding in 2007:
$3-$4 million
Biotech collaborators: 2
Patient database: No, but working
toward it
Industry projects: 1 active, 2 completed
The Muscular Dystrophy Association
(MDA) is an umbrella organization for
about 40 neuromuscular diseases that in the
aggregate affect close to 500,000 people in
the U.S. but, individually, they are all orphan
diseases. There have been no drugs
approved by the FDA specifically for mus-
cular dystrophy ever.
“We tailored a program to address spe-
cific categories of this gap we saw in the
translational aspects of drug development,”
said Hesterlee.
The MDA created the Translational
Research Program (TRP), which has four
categories of funding: IND Planning Grant,
Clinical Research Training Grant,
Infrastructure Grant and Corporate Grant.
The first two grant categories are designed
for sites, the third is to fund a national
patient database and the fourth category of
grant money is dedicated to industry. “We
designed a mechanism to fund companies
directly that are involved in early stage drug
development—all the steps leading up to
an IND (investigational new drug applica-
tion) and then a phase I or phase II clinical
trial. We saw it as affirmative action for rare
Industry Reports
11August 2007CenterWatch
see Venture Philanthropy on page 12
disease. If we can put some money into this
high-risk early stage, the idea is to move
things along until there’s some efficacy data
in humans, and then [companies] can start
getting venture capital money.” All four
programs are designed to increase the
number of biotechs working in muscular
dystrophy.
Two biotechs have completed TRP cor-
porate grants and one of them and a new
organization are working on a current
grant. MDA does not pre-allocate funds
every year, except for the research training
grants. MDA requires matching funds from
the biotechs. Companies need to match
MDA funds for a project with at least 50%
of their own money going into that same
budget.
Both MDA and funded organizations
choose a group of outside experts in the
field to form a steering committee that will
oversee the grant milestones. “We find that
the steering committee meetings function
not just to make a judgment on the mile-
stone, but they’re very good advisory com-
mittees. The companies find them to be a
good resource,” said Hesterlee.
In addition to what it offers through its
corporate grant program, MDA has discov-
ered some creative ways to enable and
speed drug development through strategic
funding. MDA has found a way to help
both Genzyme and Wyeth conduct clinical
trials in muscular dystrophy patients. In
each case, MDA approached the companies
and asked what it could do to help them
conduct clinical research in muscular dys-
trophy. Patients enrolled in the companies’
studies were often very ill and needed med-
ical care outside the clinical trial while in
the hospital. Because insurance companies
would not pay for this medical care, and the
companies were not allowed to, MDA
offered to give grants out of its translation-
al research budget to each of the sites
enrolling patients for the companies’
respective studies. Two of Wyeth’s sites
accepted funding that totaled more than
$55,000 last year. The money in this case
could also be used for travel in addition to
clinical trial subjects’ hospital costs. Three
of Genzyme’s sites were each given a grant
of $50,000 in 2003 to cover clinical trial
subjects’ hospital costs.
“Maybe there’s more than one way to
get from point A to point B, but just fund-
ing research through a standard NIH
model was never going to get us the drugs.
That was clear. I think that we had to do
this. I don’t think it was a choice. It’s our
mission to develop therapies and cures for
these diseases, not just find promising
research. It’s to see them all the way
through the clinics. If we don’t do this type
of program, then we’re letting people
down,” said Hesterlee.
MDA is planning a formal evaluation of
the program this year. Because much of the
funding has been focused on Duchenne’s
muscular dystrophy, MDA is going to look
at whether it’s increased the number of
phase I/II clinical trials in that disease.
Secondly, MDA is going to look at whether
the number of biotechnology and pharma-
ceutical companies working in this area has
increased. “Having drugs approved is the
long-term goal. It’s something we might
use in 10 years to see how the program’s
progressing,” said Hesterlee.
Juvenile Diabetes Research
Foundation
Year founded: 1970
Investment in research since founding:
$1 billion
Industry program: Industry Discovery
and Development Partnership
Program (2004)
Projected industry funding in 2007:
$14 million
Industry collaborations: 15
Product candidates: 5 in clinical trials,
10 in preclinical discovery and
evaluation
Patient database: No
The Juvenile Diabetes Research Foundation
(JDRF) has awarded more than $1 billion to
research of type 1 diabetes, most of it to aca-
demia. In that time, it has come to under-
stand some of the basic biology of the dis-
ease and some of the aspects of its patho-
genesis. In 2004, it launched its Industry
Discovery and Development Partnership
Program (IDDPP) specifically to fund
proof-of-concept in animal models and
proof-of-concept in man studies in type 1
diabetes sponsored by industry.
IDDPP’s grants are milestone-driven,
and the foundation asks the companies to
match their funding with an equal or greater
amount. JDRF has an industry application
process. The foundation accepts inquiries
and applications on a rolling basis. JDRF
also approaches companies.
“If your goal is to impact disease pro-
gression and patient quality of life and effect
a cure for a particular disease, the best way
that’s going to happen is for companies to
get involved and take therapeutics or diag-
nostics or medical devices through develop-
ment, through the approval process and out
into the marketplace . . . The academic inves-
tigators who are necessary to start the
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Thomson12 August 2007
Venture Philanthropycontinued from page 11
Industry Reports
13August 2007CenterWatch
process can’t do it by themselves. We really
need to work together and engage industry to
take over some of these opportunities and
drive the marketplace ... We want to see more
clinical research programs and more product
candidates moving toward development, so
one metric of success is as we shift more and
more into supporting clinical development
programs, that means there are more product
candidates moving toward the marketplace,”
said Lomedico.
Michael J. Fox Foundation for
Parkinson’s Research (MJFF)
Year founded: 2000
Investment in research since founding:
$94 million+
Industry program: Therapeutics
Development Initiative (2006)
Money awarded to industry (2006):
$4.6 million
Money committed to industry (2007/8):
$5 million
Patient database: No
Parkinson’s disease affects approximately
500,000 people in the U.S. Its target is
unknown. The foundation has funded more
than $94 million in research directly or
through partnerships since it was founded
in 2000.
“We don’t understand a single cause of
PD, we don’t see in the natural history of the
disease the same experience for every patient.
We can’t tell you that there are subtypes of the
disease, but we suspect it. We are trying to do
some investigating to understand that better,
which means an improved therapy might
work for a small group of patients or it might
work for a broad group of patients. It might
help treat the underlying cause of the disease,
or it might manage symptoms better. It’s such
a mix of potential positive outcomes that we
maintain a portfolio that is advancing as
many of those things that we think are sensi-
ble and we can afford to do,” said Brooks.
The foundation launched its Thera-
peutics Development Initiative (TDI) last
year to expand its industry investment. This
is the foundation’s only program that is
specifically targeted to industry, though
other funding commitments often have an
industry component. Last year 10 industry
research teams were awarded $4.6 million.
These research teams are focused primarily
on the development of neuroprotective
treatments and cell replacement approach-
es. In 2007/08 the foundation plans to
award $5 million through TDI. The pro-
gram has two grant cycles—autumn, 2007
and spring 2008.
Grantees meet in-person for mid-point
assessments of their projects. MJFF’s metrics
focus on the accountability of individual
grantees, making sure that they do what they
promised to in their applications. Mid-point
meetings bring the grantees together in one
place and also offer an opportunity to solve
problems together.
“There’s a real unmet need to capture the
kernels of these ideas at very early stages that
could be transformative and powerful and
really innovative solutions and shepherd
those along by identifying them early, provid-
ing capital in places where there’s no capital
to be had because it’s just too risky and see if
we can breathe some life into these opportu-
nities with the patients in mind,” said Brooks.
Future OutlookDisease foundations have been learning from
each other, sharing best practices and are
committed to spreading the word about ven-
ture philanthropy. While they don’t have
financial relationships with each other, their
common purpose—to discover and develop
therapies for incurable disease—binds them
together and has created a strong community.
Robert J. Beall, president and CEO of the
Cystic Fibrosis Foundation, is considered the
catalyst for investing strategically in industry
research, and he has been generous with his
time and ideas. He was the first to launch an
industry program and has helped others nav-
igate the shoals of the venture philanthropy
model.
“Bob Beall from Cystic Fibrosis sits on
our board of directors, and we’ve learned so
much from him. If I hadn’t had Bob helping
me, I might have wasted time, energy and
money. I’m a big believer in foundations
learning from each other because it makes
certain that the money invested has a broad-
er impact,” said MMRF’s Kathy Giusti.
MJFF’s Brooks has also benefited from
the experiences of other foundation heads
and been able to share some innovative ideas
of her own. She said,“We learn so much from
hearing about the strategies that other groups
have crafted as problem solvers. There are a
handful of these like-minded groups that are
aggressively trying to solve a problem that is
stunningly complex and has essentially been
delegated. The natural market doesn’t solve it
for small diseases or complex diseases. These
groups are comparing notes and not every-
thing that every group does is relevant to
what every group does. It has a lot to do with
the state of your science, what your budget is
and that might have to do with how big your
community is and how wealthy they are. It
has been advantageous to have other people
working aggressively in this field that we can
learn from and share with. We’ve been both a
great beneficiary of other people’s wisdom,
and we’ve figured out some interesting things
that other people are excited about hearing
from us on.”
Foundations’ shift from basic research
funding that went to academia alone to fund-
ing drug discovery and development by
industry as well has brought about changes in
see Venture Philanthropy on page 14
ties for protecting human subjects and
ensuring the integrity of the data from clin-
ical trials. This guidance also clarifies FDA’s
expectations concerning the investigator’s
responsibility for supervising a clinical trial
in which some tasks are delegated to
employees or colleagues of the investigator
or other third parties.
In FDA regulations, the investigator is
the person who conducts a clinical investi-
gation of a drug, biologic, or medical
device. The FDA definition for “investiga-
tor,” which is a specific regulatory term,
may be found at 21 CFR 312.3(b) for
drugs/biologicals and at 21 CFR 812.3(i)
for medical devices. Because in a clinical
trial there may be multiple investigators
(the principal investigator, one or more
sub-investigators and co-investigators), it is
important to recognize that only one of
these investigators has the ultimate respon-
sibility for the clinical trial. Throughout
this article, that investigator will be referred
to as the principal investigator (PI). The PI
is the intended audience for this draft guid-
ance document.
Under the regulations at 21 CFR 312
(Investigational New Drug Application or
IND) and 21 CFR 812 (Investigational
Device Exemptions or IDE), a PI is respon-
sible for:
ensuring that a clinical investigation is
conducted according to the signed
investigator statement for investigations
of drugs and biological products or a
signed investigator agreement for inves-
tigations of medical devices, and the
investigational plan and applicable reg-
ulations;
protecting the rights, safety, and welfare
of subjects under the PI’s care; and
for controlling the drugs, biological
products, and medical devices under
investigation.
This draft guidance document clarifies
the PI’s responsibilities in the conduct of
clinical investigations subject to these regu-
lations, particularly the responsibilities to
supervise the conduct of the clinical investi-
gation, and to protect the rights, safety, and
welfare of study subjects in drug, biologic,
and medical device clinical trials. The draft
guidance document also provides recom-
mendations on how the PI should supervise
the study-related actions of persons not in
the direct employ of the PI, including cer-
tain study staff and parties conducting asso-
ciated testing and assessments.
Draft guidance is not generally intend-
ed for widespread use; it is intended to
solicit public comment. Once FDA has
received and reviewed all comments from
the public, the agency will release the final
Industry Reports
Thomson14 August 2007
FDA Guidance continued from page 1
U.S. Clinical Investigators Submitting INDsNumber of investigators
Source: CenterWatch analysis, 2005; FDA.
2004E200320022001
27,928 27,97723,865
22,145
the way stakeholders view the drug discovery
and development process. “This was a real
change in culture. One of the things we do is
get the basic scientists at the table with [bio-
pharmaceutical companies] and it really gives
them a sense of engagement in the process.
They’re not threatened by us moving from
largely a basic research organization to one
where we’re very involved in translation. It’s
been the nucleus of a major gifts program.
You tell businessmen about our model, how
we hold ourselves accountable through that
pipeline, and people are willing to invest in
that process,” said Beall at BIO2007.
In the future, these five foundations will
be called upon to share their expertise with
other foundations not yet participating in the
venture philanthropy model.
“The message is getting around to other
foundations, and other patient groups are see-
ing the important role that industry plays.
And they’re looking to learn from their col-
leagues, including us and the other founda-
tions, so they can learn how to engage indus-
try more effectively,” said Lomedico.
While the dollar amount invested in
industry research is quite small, foundations
have found the strategic places to make a
small amount of money work the hardest.
Without it, in most if not all cases, the chance
of developing a new therapy would not exist.
An investment by a foundation is capable of
attracting tens of millions of dollars of ven-
ture capital afterward.
Hesterlee concluded,“Venture philanthro-
py is a small amount of money in the overall
sea of money floating around out there, but I
think because all the groups are really trying to
apply it so strategically, it’s going to make a big
difference—sort of like the lever Archimedes
said could move the world.”
—Sara Gambrill
Venture Philanthropycontinued from page 13
Industry Reports
15August 2007CenterWatch
version of the guidance document. This
process may take months or even years to
complete. Nevertheless, a draft guidance
document can provide helpful clues about
what FDA officials are thinking on a partic-
ular topic. The discussion in this draft doc-
ument is useful to any PI wishing to under-
stand FDA’s thinking when it addresses the
PI’s supervisory role.
Many of the PI’s responsibilities when
conducting clinical trials of drugs or bio-
logics under the IND regulations are
included in the required investigator’s
signed statement, the Form FDA 1572
(henceforth referred to as “the 1572”), in
which the PI makes several key commit-
ments. Those commitments are:
To conduct the studies in accordance
with the relevant, current protocol(s)
and to only make changes in a protocol
after notifying the sponsor, except when
necessary to protect the safety, rights, or
welfare of subjects,
To personally conduct or supervise the
described investigation(s),
To inform any patients, or any persons
used as controls, that the drugs (biolog-
ics) are being used for investigational
purposes and to ensure that the require-
ments relating to obtaining informed
consent in 21 CFR 50 and institutional
review board (IRB) review and
approval in 21 CFR 56 are met,
To report to the sponsor adverse experi-
ences that occur in the course of the
investigations(s) in accordance with 21
CFR 312.64,
That he/she has read and understands
the information in the investigator’s
brochure, including the potential risks
and side effects of the drug (biologic),
To ensure that all associates, colleagues,
and employees assisting in the conduct
of the studies are informed about their
obligations in meeting the above com-
mitments,
To maintain adequate and accurate
records in accordance with 21 CFR
312.62 and to make those records avail-
able to FDA for inspection in accor-
dance with 21 CFR 312.68,
That an IRB that complies with the
requirements of 21 CFR 56 will be
responsible for the initial and continu-
ing review and approval of the clinical
investigation; to promptly report to the
IRB all changes in the research activity
and all unanticipated problems involv-
ing risks to human subjects or others; to
not make any changes in the research
without IRB approval, except where
necessary to eliminate apparent imme-
diate hazards to human subjects, and
To comply with all other requirements
regarding the obligations of clinical
investigators and all other pertinent
requirements in 21 CFR 312.
The PI should note that his/her signa-
ture under these commitments is followed
by a warning that a willfully false statement
is a criminal offense under section 1001 of
Title 18 of the United States Code (U.S.C.
Title 18, Sec. 1001). In essence, signing the
1572 and then willfully or intentionally not
meeting these commitments is making a
false statement under the law.
A copy of the 1572 is included in the
guidance document as Attachment A.
Interestingly, FDA has attached an expired
version of the document (it expired on
January 31, 2006). The current version
available on FDA’s website expires on May
31, 2009. PIs should check the expiration
date of the form before signing it and ask
the sponsor to provide a valid version if the
form is expired. In addition, the 1572 does
not list all the PI’s requirements. The PI
should refer to the regulations (21 CFR
Parts 50, 56 and 312) to ensure familiarity
with all of FDA’s requirements for the con-
duct of drug and biologics clinical trials.
Clarification of CertainInvestigator ResponsibilitiesThis section of the guidance document is
intended to clarify the PI’s responsibility to
supervise the conduct of the clinical inves-
tigation and to protect the rights, safety and
welfare of study participants in drug and
medical device clinical trials.
Supervising
The PI who conducts clinical investigations
of drugs or biological products under an
IND commits to personally conduct or
supervise the investigation. The PI who
conducts clinical investigations of medical
devices under an IDE commits to supervise
all testing of the device involving human
subjects. It is common practice for the PI to
delegate certain study-related tasks to
see FDA Guidance on page 16
Proportion of New Investigators to New Trials
Source: FDA, June 2005.
1996–20001991–19951986–19901980–1985
1.72
2.633.20
3.87
2001–2004
4.28
Average trials per investigator, 3.30
employees, colleagues or other third parties
(individuals or entities not under the direct
supervision of the PI). When tasks are del-
egated by the PI, the PI is responsible for
providing adequate supervision of those to
whom tasks are delegated and the PI is
accountable for regulatory violations
resulting from failure to adequately super-
vise the conduct of the clinical study.
It is useful to read FDA’s own words in
these matters. As noted succinctly by FDA
in a 2002 “Notice of Initiation of
Disqualification Proceedings and
Opportunity to Explain” (NIDPOE) letter,
“We remind you that it is your responsibil-
ity, as the…Principal Investigator, to ensure
that the investigation is conducted accord-
ing to the…investigational plan, and appli-
cable regulations….you are responsible for
personally conducting or supervising the
clinical investigation. While in a superviso-
ry role, you may delegate…such delegation
requires careful supervision….”
FDA further wrote “As the [PI], you
remain responsible for overseeing and
reviewing their work…and must make cer-
tain that they are following the…plan.
Regardless of what…you may delegate, you
remain ultimately responsible for the prop-
er conduct of clinical studies in which you
are the investigator of record.”
This inelegant title represents a more
severe enforcement action than an FDA
Warning Letter. The NIDPOE letter is
issued to the PI who has submitted false
information or committed fraud or mis-
conduct, or who has repeatedly or deliber-
ately failed to comply with FDA regula-
tions. While the letter may be five years old,
FDA’s words seldom have an expiration
date. FDA’s overall expectations then and
now vis-à-vis the PI’s supervision of clini-
cal trial activities are quite clear in these
words.
In assessing the adequacy of supervi-
sion by a PI, FDA focuses on four major
issues: 1) whether delegated individuals
were qualified to perform such tasks, 2)
whether study staff received adequate
training on how to conduct the delegated
tasks and were provided with an adequate
understanding of the study, 3) whether
there was adequate supervision and
involvement in the ongoing conduct of the
study, and 4) whether there was adequate
supervision or oversight of any third par-
ties involved in the conduct of a study to
the extent such supervision or oversight
was reasonably possible. FDA makes the
following recommendations on these top-
ics.
1. What is appropriate delegation of
study-related tasks?
According to FDA, the PI should ensure
that any individual to whom a task is dele-
gated is qualified by education, training,
and experience to perform that delegated
task. Appropriate delegation is primarily an
issue for tasks that would be considered to
be clinical or medical in nature, such as
evaluating study subjects to assess clinical
response to an investigational therapy (e.g.,
global assessment scales, vital signs) or pro-
viding part of the medical care provided to
subjects during the course of the study.
Most clinical/medical tasks require formal
medical training and also may have licens-
ing or certification requirements. Such
licensing requirements will vary from state
to state. The PI should take such qualifica-
tions/licensing requirements into account
when considering to whom it would be
appropriate to delegate specific tasks.
During inspections, FDA has identified
instances in which study tasks have been
delegated to individuals lacking appropri-
ate qualifications. Examples of inappropri-
ate delegation include:
Screening evaluations, including obtain-
ing medical histories and assessment of
inclusion/exclusion criteria, conducted
by individuals with inadequate medical
training (e.g., a medical assistant),
Physical examinations performed by
unqualified personnel,
Evaluation of adverse events by individ-
uals lacking appropriate medical train-
ing, knowledge of the clinical protocol,
and knowledge of the investigational
product,
Industry Reports
Thomson16 August 2007
FDA Guidance continued from page 15
More Investigators, Lots More Studies
Source: FDA, June 2005.
20,000
40,000New studies
New investigators
2001-20041996-20001991-19951986-19901981-1985
6,07910,701
16,435
25,791
36,839
8,6166,6695,1414,0673,540
10,000
30,000
First Time InvestigatorsAverage number of first time investigators
Source: FDA, June 2005.
1996–20001991–19951986–19901980–1985
3,5404,067
5,1416,669
2001–2004
8,616
Industry Reports
17August 2007CenterWatch
Assessments of primary study end-
points (e.g., tumor response, global
assessment scales) by individuals lack-
ing appropriate medical training and
knowledge of the protocol, and
Informed consent obtained by individ-
uals who lack the medical training,
knowledge of the clinical protocol, or
familiarity of the investigational prod-
uct needed to be able to discuss the risks
and benefits of a clinical trial with
prospective subjects.
According to FDA regulations, the PI is
responsible for conducting studies in
accordance with the protocol. Some proto-
cols specify the qualifications of the indi-
viduals who are to perform certain proto-
col-required tasks, and these protocols
must be followed even if state law permits
differently qualified people to perform the
task. For example, even if the state in which
the study site is located permits nurse prac-
titioners to perform physical examinations
under the supervision of a physician, if the
protocol specifies that physical examina-
tions must be done by a physician, a physi-
cian must perform such exams. There have
been similar situations where the protocol
only permits the PI to conduct the
informed consent process and to sign the
consent form. This unnecessary restriction
often has resulted in adverse audit findings
when the PI is personally unable to sign the
form. The imposition of this restriction
should be carefully thought through in
light of real-world events that occur during
a clinical trial, and which may result in
noncompliance.
PIs should also be cautioned that decid-
ing who is qualified to perform a particular
protocol-related task should not be
reduced to “completing a checklist” or con-
ducting a superficial review of a curricu-
lum vitae. People without formal medical
degrees may ably perform various med-
ical/clinical tasks, provided they have had
the proper training and experience. On the
other hand, a medical background is no
guarantee that study staff will not commit
serious errors in study-related tasks of a
medical/clinical nature. The determination
of who is qualified by education, training,
and experience to perform a study-related
task should be made thoughtfully and the
criteria that are used to make the determi-
nation should be more robust than simply
the letters after a person’s name.
FDA notes in the guidance document
that the PI should maintain a list of the
appropriately qualified persons to whom
significant trial-related duties have been del-
egated. This list should describe the delegat-
ed tasks, identify the training that individu-
als have received that qualifies them to per-
form delegated tasks, and identify the dates
of involvement in the study. A PI should
maintain separate lists for each study con-
ducted by the PI. Most sponsors create study
delegation logs (often combined with a staff
signature log) but these logs may not have
space for adding each staff person’s training.
Rather, it makes more sense to also provide
each PI with a staff training log where study-
specific training can be documented.
Combined or separated, written documen-
tation of delegated tasks and staff training is
very important.
2. What is adequate training?
The PI should ensure that there is adequate
training for all staff participating in the
conduct of the study. The PI should antici-
pate the possibility of staff turnover during
the conduct of the study (particularly if the
study is of long duration) and plan to
ensure that there is adequate training of
any replacement staff. The PI should ensure
that staff:
Have a general familiarity with the
study and the protocol,
Have a specific understanding of the
details of the protocol and the investi-
gational product, relevant to the tasks
they will be performing,
Are aware of regulatory requirements
and acceptable standards for the con-
duct of clinical trials, both in respect to
conduct of the clinical trial and human
subject protection,
Are competent to perform the tasks that
they are delegated, and
Are informed of any pertinent changes
during the conduct of the trial and edu-
cated or given additional training as
appropriate.
If the sponsor provides training materi-
als for the PI on the conduct of the study,
the PI should ensure that staff members
see FDA Guidance on page 18
New Trials GrowingAverage new trials per year
Source: FDA, June 2005.
1996–20001991–19951986–19901980–1985
6,07910,701
16,435
25,791
2001–2004
36,839
Industry Reports
Thomson18 August 2007
receive the sponsor’s training or informa-
tion from the training that is pertinent to
their roles in the study.
3. What is adequate supervision of the
conduct of an ongoing clinical trial?
The PI should have a detailed plan for the
supervision and oversight of a clinical trial.
Supervision and oversight should be pro-
vided even for individuals who are highly
qualified and experienced. A plan might
include the following elements, to the
extent they apply to a particular trial:
Routine meetings with staff to review
trial progress and update staff on any
changes to the protocol or other proce-
dures,
Routine meetings with the sponsor’s
monitors,
A procedure for correcting problems
identified by study personnel, outside
monitors or auditors, or other parties
involved in the conduct of a study,
A procedure for documenting the per-
formance of delegated tasks in a satis-
factory manner and, where appropriate,
verifying findings (e.g., observation of
the performance of selected assess-
ments or independent verification by
repeating selected assessments),
A procedure for ensuring that the con-
sent process is being conducted in
accordance with 21 CFR 50 and that
study subjects understand the nature of
their participation, risks, etc.,
A procedure for ensuring that informa-
tion in source documents is accurately
captured on the Case Report Forms,
A procedure for dealing with data
queries and discrepancies identified by
the study monitor, and
Procedures for ensuring study staff
comply with the protocol, adverse event
assessment and reporting, and other
medical issues that arise during the
course of the study.
The PI should have sufficient time to
properly conduct and supervise the clinical
trial. The intensity of the supervision
should be appropriate to the staff, the
nature of the trial, and the subject popula-
tion. In FDA’s experience, the following fac-
tors may compromise the ability of an indi-
vidual PI to provide adequate supervision
of the conduct of an ongoing clinical trial:
Inexperienced study staff,
Overburdened study staff,
Complex clinical trials (e.g., many
observations, large amounts of data col-
lected),
Large number of subjects enrolled at a
site,
A patient population that is quite sick,
Conducting a large number of studies
concurrently,
Conducting a study from a remote (i.e.,
off-site) location, and
under the oversight of a single PI, par-
ticularly where those sites are not near
each other (e.g., sites that are geograph-
ically distant, in another city, county,
state, or country).
FDA indicates in this guidance docu-
ment that the agency prefers for any site
with substantial enrollment to have an
identified PI with clear responsibilities, but
if that is not arranged, FDA believes there
should ordinarily be an individual respon-
sible for the conduct of the clinical trial at
each trial site, identified as a sub-investiga-
tor. Sub-investigators should report direct-
ly to the PI (i.e., the PI should have clear
responsibility for evaluating the sub-inves-
tigator’s performance and should have the
authority to hire/fire the sub-investigator).
4. What are an investigator’s responsi-
bilities for oversight of other parties
involved in the conduct of a clinical trial?
Study staff not in the direct employ of the
PI—The staff involved directly in the
conduct of a clinical investigation may
include individuals who are not in the
direct employ of the PI. For example, a
site management organization (SMO)
may hire a PI to conduct a study and
provide the PI with a study coordinator
or nursing staff employed by the SMO.
In this situation, the PI should take
steps to ensure that the staff not under
his/her direct employ are qualified to
perform delegated tasks (as discussed
above) and that the staff have received
adequate training on carrying out the
delegated tasks and on the nature of the
study, or the PI should provide such
training.
The PI is responsible for supervision
of the study tasks performed by this
staff, even though they are not in
his/her direct employ during the con-
duct of the study and this responsibility
exists no matter how qualified and
experienced these staff members are. In
the event that the staff ’s performance of
study-related tasks is not adequate and
cannot be made satisfactory by the PI,
the PI should document the observed
deficiencies in writing to the staff ’s
supervisor(s). Depending on the severi-
ty of the deficiencies, the clinical trial
may need to be voluntarily suspended
until personnel can be replaced.
Parties other than study staff—There are
often critical aspects of a study per-
formed by parties not involved directly
in patient care or contact, and not
FDA Guidancecontinued from page 17
Industry Reports
19August 2007CenterWatch
under the direct control of the PI. For
example, clinical chemistry testing,
radiologic assessments and electrocar-
diograms are commonly done by a cen-
tral independent laboratory retained by
the sponsor or the PI. Under these
arrangements, the laboratory or diag-
nostic procedure provider usually pro-
vides the test results directly to the
sponsor and to the PI. Because the
activities of these parties are critical to
the outcome of the study, and because
the sponsor retains the services of the
laboratory, the sponsor is responsible
for seeing that these parties are fulfilling
their responsibilities for the study.
Less frequently, a study may require
that the PI arranges to obtain informa-
tion critical to the study that cannot be
obtained at the PI’s facility. For example,
if the study protocol requires testing
with special equipment or expertise not
available at the PI’s facility, the PI might
make arrangements for someone out-
side the facility to perform the test. In
this case, the results are provided direct-
ly to the PI, who then submits the infor-
mation to the sponsor. Where such
assessments are retained by the PI, the
PI should take steps to ensure that the
facility is adequate (e.g., has the required
certifications or licenses). The PI may
also institute procedures to ensure the
integrity of data and records obtained
from the party providing the informa-
tion (e.g., a process to ensure that
records identified as coming from the
party are authentic). Procedures are par-
ticularly important when assessments
are crucial to the evaluation of the effi-
cacy or safety of an intervention or to
the decision to exclude subjects who
would be exposed to unreasonable risk.
The PI should carefully review the
reports from these external sources for
results that are inconsistent with clinical
presentation. To the extent feasible, and
considering the specifics of study
design, the PI should evaluate whether
results appear reasonable, individually
and in aggregate. If the PI detects possi-
ble errors or suspects that results from a
central laboratory might be question-
able, the PI should contact the sponsor
immediately.
Exception for certain device studies—In
some cases, specialized expertise from a
device sponsor is needed to perform
certain tasks. For example, when there
is no one at the clinical site who can
program an investigational pacemaker,
the expertise may be provided by the
sponsor’s personnel, such as a field clin-
ical engineer. The field clinical engineer
should be supervised by the sponsor
and not by the PI. When a field clinical
engineer is designated by the sponsor to
perform a specific activity within the
investigational plan, this activity should
be described in the protocol. The PI
retains responsibility for ensuring that
the protocol is followed.
Protecting study subjects
The PI is responsible for protecting the
rights, safety, and welfare of subjects under
his/her care during a clinical trial. The PI
should provide a reasonable standard of
medical care for study subjects for medical
problems arising during participation in
the trial that are, or could be related, to the
study intervention. The PI should be readi-
ly available to provide such care during the
study or should ensure that other identi-
fied, qualified individual(s) are available to
provide such care. Failure to adhere to the
protocol can expose subjects to unreason-
able risks.
1. Reasonable medical care necessitated
by participation in a clinical trial
During and following a subject’s participa-
tion in a trial, the PI should ensure that
adequate medical care is provided to a sub-
ject for any adverse events, including clini-
cally significant laboratory values, related
to the trial. The PI should inform a subject
when medical care is needed for an inter-
current illness. The PI should inform the
subject’s primary physician about the sub-
ject’s participation in the trial if the subject
has a primary physician and if the subject
agrees to the primary physician being
informed.
If the PI does not possess the necessary
skills to provide adequate medical care for
the subject, the PI should make every effort
to obtain appropriate care. For example, if
a carotid stent is placed in a subject by an
see FDA Guidance on page 20
Profile of Investigators Filing FDA 1572Percent of respondents
Source: FDA, 2005.
MD
MB, CHB, PharmD,PhD, Other
DO
91%
3%6%
interventional neuroradiologist and the
subject suffers a cerebral stroke, the neuro-
radiologist should assess the clinical status
of the subject and transfer the subject to a
neurology service. Subjects should receive
appropriate medical evaluation and treat-
ment until resolution of any condition
related to the study intervention that devel-
ops during the course of their participation
in a study, even if the follow-up period
extends beyond the end of the study at the
investigative site.
2. Reasonable access to medical care
To protect subjects from unnecessary risks,
the PI should be available to subjects dur-
ing the conduct of the trial at his/her site.
Availability is particularly important where
subjects are receiving a drug that has signif-
icant toxicity or abuse potential. For exam-
ple, if a study drug has potentially fatal tox-
icity, the PI should be readily available by
phone or other electronic communication,
and in reasonably close proximity to study
subjects (e.g., not in another state or on
prolonged travel). Study subjects should be
clearly educated on the possible need for
such contact and on precisely how to
obtain it, generally by providing pertinent
phone numbers, web sites, etc., in writing.
Prior to undertaking the conduct of a
study, a prospective PI should consider
whether he/she can be available to the
extent needed given the nature of the trial.
If the PI is not going to be available for
some period during the study, clinical
responsibility for study subjects should be
delegated to a specific qualified physician
who will be readily available to subjects.
This delegation should be documented in a
1572 or investigator agreement (the physi-
cian should be listed as a sub-investigator)
and also submitted to the IRB for review
(as a change in the research activity requir-
ing IRB review). If the PI is a non-physi-
cian, the PI should make adequate provi-
sion for any necessary medical care that the
PI is not qualified to provide.
3. Protocol violations that present
unreasonable risks
There are occasions when a failure to
adhere to the protocol may be considered a
failure to protect the rights, safety, and wel-
fare of subjects. For example, failure to
adhere to inclusion/exclusion criteria that
are specifically intended to exclude subjects
for whom the study drug or device poses
unreasonable risks (e.g., enrolling a subject
with decreased renal function in a trial in
which decreased function is exclusionary
because the drug may be nephrotoxic) may
be considered failure to protect the rights,
safety, and welfare of the enrolled subject.
Similarly, failure to perform safety assess-
ments intended to detect drug toxicity
within protocol-specified time frames (e.g.,
CBC for an oncology therapy that causes
neutropenia) may be considered failure to
protect the rights, safety, and welfare of the
enrolled subject. The PI should seek to
minimize such risks by adhering closely to
the study protocol.
There are many PIs who are lacking in
their understanding of the scope of FDA’s
regulatory requirement that the PI careful-
ly supervise any clinical trial under his/her
responsibility. While sponsors routinely
provide study training (including general
Good Clinical Practice topics) to the PI and
staff before the start of a clinical trial, that
training often is inadequate for the inexpe-
rienced PI or staff member. FDA has made
a substantial contribution to the training of
study staff by issuing this draft guidance
document.
—Anna J. DeMarinis, MA, MT, CQA
Industry Reports
Thomson20 August 2007
FDA Guidancecontinued from page 19 C
utaneous melanoma is a malignan-
cy of skin cells known as
melanocytes that contain pigment
and color the skin. Less frequently, melanoma
may also develop within the eye (intraocular
or ocular melanoma). According to the
American Cancer Society, there were 62,000
cases of melanoma in the United States in
2006, which caused 8,000 deaths.
Adults over the age of 20 are more often
affected by melanoma than are children and
adolescents. Risk factors include unusual
moles (nevi), exposure to natural sunlight or
to artificial ultraviolet light, family or person-
al history of melanoma, white or pale skin,
red or blond hair, freckles and blue eyes.
Warning signs for melanoma include spe-
cific changes in the appearance of a mole or
pigmented skin region. Diagnosis can be
confirmed by biopsy, and further evaluation
should confirm the stage and extent of
metastasis. Prognosis and treatment are
affected by the location, size and stage of
melanoma.
Although melanoma accounts for about
5% of all skin cancers, it is responsible for
approximately 75% of all skin cancer-related
deaths. If melanoma is removed when it is
confined to the outermost skin layer, it is
potentially curable, but deeper lesions or
metastatic disease have a poor prognosis and
expected survival of only six to nine months.
Five-year survival in Stage IV ranges from
only 6.7% to 18.8%, depending on the site of
metastasis.
Existing treatment options for melanoma
include surgery for local or wide local exci-
sion with or without lymph node removal,
chemotherapy, radiotherapy and biologic
therapy. However, there has been little
improvement in outcomes during the past 30
years. In advanced stage melanoma, response
rates to available treatments are no greater
than 20%, and most of the available drugs are
highly toxic.
Grant Opportunities
21August 2007CenterWatch
CenterWatch has identified a pipeline of
17 drugs in various stages of development for
melanoma treatment, many of which are bio-
logical agents or vaccines.
Antigenics is in phase III development of
Oncophage (vitespen; formerly HSPPC-96),
a personalized vaccine based on antigens
extracted from each specific patient’s tumor
combined with endoplasmin, or heat shock
protein. Because the vaccine targets only
tumor cells, it should theoretically have
improved efficacy with reduced adverse
effects. The U.S. Food and Drug
Administration (FDA) has granted
Oncophage fast track and orphan drug desig-
nations for metastatic melanoma as well as
for renal cancer.
Once Oncophage is prepared from the
patient’s tumor, it is usually administered on
an outpatient basis beginning within four to
eight weeks after surgery, with one injection
once weekly for four weeks, then one injec-
tion every other week. In a phase III trial in
metastatic melanoma, median survival with
at least 10 injections of Oncophage was 29%
greater than with the physician’s choice of
regimen.
To date, more than 12 clinical trials of
Oncophage have enrolled more than 750 can-
cer patients, many of whom have had
advanced stage cancer refractory to tradition-
al cancer treatments. Adverse effects may
include injection-site reactions, generalized
and joint pain, constipation, fatigue, fever,
diarrhea, edema, loss of appetite and weight,
nasopharyngitis, arthralgia, dizziness, anxi-
ety, depression, cough, breathing problems,
headache, vomiting, anemia, sleep distur-
bances and weakness.
Another autologous cell vaccine is M-
Vax, in phase III testing by Avax and already
approved in Switzerland for the treatment of
stage 3 and 4 melanoma. This vaccine is made
by adding the hapten dinitrophenyl (DNP) to
the patient’s melanoma cells, which are then
injected within the dermis in combination
with the immunological adjuvant bacilli
Calmette-Guerin. Most patients experience a
delayed-type hypersensitivity (DTH) to the
DNP-modified melanoma cells, and approxi-
mately half also develop the same immune
response to unmodified tumor cells.
In a trial of 214 patients with stage 3
melanoma, M-Vax therapy after lymph node
removal was associated with 44% five-year
survival, compared with 20% to 25% for sur-
gery alone. Survival was directly related to
induction of DTH response to unmodified
autologous melanoma cells. Another study
showed antitumor responses in 11 of 83
patients with stage 4 melanoma and measur-
able metastases treated with M-Vax. The
most frequent side effects include mild nau-
sea, vomiting and injection-site reactions.
IDM Pharma is in phase II testing of
Uvidem, a vaccine made from the patient’s
own dendritophages, or specialized immune
cells derived from the patient’s own white
blood cells, that are exposed in the laboratory
to specific tumor antigens derived from the
patient’s cancer cell extracts.
Oxford Biomedica’s candidate for a
melanoma vaccine is Hi8, now in phase II
development. This vaccine is composed of a
DNA plasmid (DNA.Mel3) and a non-repli-
cating Modified Vaccinia Ankara (MVA) viral
vector (MVA.Mel3) containing 7 human
HLA-A2 or HLA-A1-restricted CTL epitopes
which are designed to stimulate immune sys-
tem recognition and destruction of cancer
cells by CD8+ T-cells.
In early trials, Hi-8 MEL was well tolerat-
ed and produced immunological melanoma-
specific CD8+ T-cell responses. Nearly 20%
of patients had tumor responses; median sur-
vival was 100 weeks for immune responders
versus 37 weeks for non-responders
(p<0.001) and 42 weeks for a group of con-
trol non-HLA A2 patients who received stan-
dard of care.
Genasense, in phase III testing by Genta,
is an antisense oligonucleotide targeting Bcl-
2, a protein over-expressed in many types of
cancer. An ongoing randomized, open-label,
cross-over pharmacokinetic study is evaluat-
ing combination therapy with Genasense and
dacarbazine (DTIC) in patients with hepatic
impairment and advanced malignant
melanoma.
Medarex is in phase III development of
ipilimumab (MDX-010), a fully human anti-
body targeting CTLA-4, a molecule on T cells
that suppresses the immune response. An
open label, single arm trial of ipilimumab
monotherapy is underway in patients with
previously treated Stage 3 or Stage 4 metasta-
tic melanoma unresponsive to at least one
other regimen of another melanoma therapy.
A phase III pivotal trial is also ongoing in
combination with the melanoma peptide
vaccine MDX-1379.
Allovectin-7, a plasma/lipid complex con-
taining the human DNA gene sequences that
encode HLA-B7 antigen and Beta2
microglobulin, is in phase III testing by Vical.
Together, these two antigens form a Class I
Major Histocompatibility Complex (MHC-
I) antigen. Injecting Allovectin-7 directly into
melanoma lesions may boost the patient’s
immune response to both local and metasta-
tic tumors.
Synta is in phase IIb trials with STA-4783,
an inducer of heat shock protein 70 (Hsp70)
on the surface of tumor cells that attracts nat-
ural killer (NK) immune cells and activates
NK-mediated tumor cell death. This novel,
injectable, small molecule induces an oxida-
tive stress response in cells, which is charac-
terized by increased production of gene fam-
ilies that protect against different cellular
stresses such as intense heat, oxygen radicals
or heavy metal exposure.
In a double-blind, randomized phase IIb
trial at 21 U.S. sites in the U.S., 81 patients
Eye On: Melanoma
see Melanoma on page 22
Grant Opportunities
Thomson22 August 2007
In the Pipeline: Melanoma
Drug Company Contact Additional Information
Phase I
Exherin (ADH-1) Adherex (919) 484-8484 An angiolytic, it binds to N-cadherin, a cell-surface www.adherex.com adhesion molecule that encourages blood vessel cells
to bind to each other. Selectively causes tumor blood vessels to rupture and slows or stops the flow ofblood necessary for cancer cells to grow. Is being investigated in combination with limb infusion melphalan
plasmid DNA Inovio (858) 597-6006 encodes a tumor antigen for delivery using Inovio’s vaccine www.innovio.com MedPulser electroporation technology
Provecta Provectus (865) 769-4011 a concentrated drug formulation of rose Bengal,www.pvct.com a radiochemosensitizer
were randomized to treatment with STA-
4783 in combination with paclitaxel or pacli-
taxel alone. Combination therapy was associ-
ated with a 50% reduced risk of disease pro-
gression and a three times higher objective
response rate. STA-4783 was well tolerated,
with adverse events for combination therapy
typical for those expected with paclitaxel
alone (fatigue, hair loss, constipation, nausea,
numbness, arthralgia, insomnia, diarrhea and
anemia).
Another therapeutic that alters the func-
tion of a heat shock protein is tanespimycin,
in phase II testing by Kosan. This ansamycin
antibiotic binds to and inhibits Hsp90, pre-
venting the proper folding and stability of
many proteins involved in cancer and leading
to cancer cell death. In clinical studies, Hsp90
inhibitors appear to re-sensitize cancer cells
to drugs to which they have developed resist-
ance, and they may also enhance the initial
activity of existing cancer therapies.
OncoVEX, which is intended for use in
solid tumors, is in phase II development by
BioVex. This modified oncolytic version of the
herpes simplex virus type-1 (HSV-1) incor-
porates Biovex’s OncoVEX platform with the
granulocyte-macrophage colony stimulating
factor (GM-CSF) gene. After reproducing
within cells, the HSV-1 virus lyses or ruptures
the cell membrane, killing the cell. Deleting
the gene encoding the ICP34.5 protein from
the HSV-1 DNA allows HSV to replicate in
tumor cells but not in normal cells. Adding
the GM-CSF gene enhances stimulation of
anti-tumor response by immune system cells.
In early trials, OncoVEXGM-CSF was well
tolerated with few serious side effects.
SciClone and Sigma-Tau are developing
Zadaxin (thymalfasin), a 28-amino acid syn-
thetic peptide resembling a natural peptide
isolated from the thymus gland. In a phase II
trial of combination therapy with Zadaxin
plus chemotherapy in 488 patients with stage
IV malignant melanoma, thymalfasin plus
DTIC without interferon alpha was associat-
ed with triple the overall response rate and
three-month improvement in overall survival
compared with DTIC plus interferon alpha
treatment.
ZymoGenetics and Novo Nordisk are in
phase IIa testing of IL-21, an injectable
recombinant human Interleukin 21 cytokine
with regulatory effects on cytotoxic T cells
(CTL) and Natural Killer (NK) cells. In ani-
mal models of cancer, IL-21 increases NK cell
activity and disease-specific CTLs and
reduces the number of tumors.
Amplimexon, an injectable formulation
of the cyanoaziridine compound imexon, is
in phase I/II development by AmpliMed. By
targeting an enzyme involved in DNA syn-
thesis, this drug inhibits cancer cell division;
other mechanisms of action include disrup-
tion of mitochondria and increased uptake of
other chemotherapies into cancer cell DNA.
The dismal prognosis and adverse events
associated with conventional and currently
available treatments for melanoma mandates
an innovative approach. Time will tell
whether the target biological agents in the
pipeline will ultimately be more effective and
less toxic than standard treatments.
—Laurie Barclay, M.D.
Melanomacontinued from page 21
Grant Opportunities
23August 2007CenterWatch
Drug Company Contact Additional Information
Phase I/II
Amplimexon AmpliMed (520) 529-1000 an injectable formulation of imexon, which inhibits www.amplimed.com cancer cell division and targets an enzyme involved in
DNA synthesis; being evaluated in combination with dacarbazine
BNP 1350 BioNumerik (210) 614-1701 a silicon-containing agent from a novel class ofwww.bionumerik.com lipophilic camptothecins
Phase IIa
IL-21 ZymoGenetics/ (206) 442-6600 an injectable recombinant human IL-21; has Novo Nordisk www.zymogenetics.com regulatory effects on cytotoxic T cells (CTL) and
Natural Killer (NK) cells
Phase II
OncoVEX BioVex (617) 444-8445 a modified oncolytic version of the herpes simplex www.biovex.com virus type-1 (HSV-1) that incorporates Biovex’s
OncoVEX platform with the granulocyte-macrophage colony stimulating factor (GM-CSF) gene
Uvidem IDM Pharma (949) 470-4751 a vaccine composed of autologous dendritophages www.idm-biotech.com loaded in vitro with specific tumor antigens derived
from the patients’ cancer cell extracts
tanespimycin Kosan (510) 732-8400 an ansamycin antibiotic which binds to Hsp90 www.kosan.com (Heat Shock Protein 90) and alters its function
Hi8 melanoma Oxford Biomedica (858) 677-6500 a vaccine based on Oxford’s the Hi8 PrimeBoostvaccine www.oxfordbiomedica.com technology platform. It utilizes a DNA plasmid
(DNA.Mel3) and a MVA viral vector (MVA.Mel3)containing seven human HLA-A2 or HLA-A1-restricted CTL epitopes; designed to enhance the body’s own ability to recognize and destroy cancerous cells
Zadaxin SciClone/Sigma-Tau (650) 358-3456 a 28-amino acid peptide originally isolated from the www.sciclone.com thymus gland and now produced synthetically
Phase IIb
STA-4783 Synta (781) 274-8200 an inducer of heat shock protein 70 (Hsp70) on the www.syntapharma.com surface of tumor cells; attracts natural killer (NK)
immune cells and activates NK-mediated tumor cell death
Phase III
Oncophage Antigenics (212) 994-8200 a personalized vaccine based on tumor antigens www.antigenics.com extracted from the patient’s tumor mixed with
endoplasmin (heat shock protein)
M-Vax Avax (215) 241-9760 an autologous cell hapten-modified vaccinewww.avax-tech.com
Grant Opportunities
Thomson24 August 2007
Multiple TherapeuticAreasStill Seeking Investigators
Kendle International1200 Carew Tower, 441 Vine StreetCincinnati, OH 45202
Contact: Karen KempfEmail: [email protected]
Drug name: Not applicableIndication: AnySpecialty: Some key specialities being sought
are: Rheumatologists, Gastro-enterologists, Oncologists,Hematologists, Pediatricians,Allergists, Acute Neurologists,Internal Medicine, OB/GYN,Women’s Health
Phase: I-IVNotes: Join the Kendle investigator team
as we study breakthrough thera-peutic agents. Our teams ofInvestigator professionals play acritical role in the advancement ofmedical science across the world.Please send an email to [email protected] to be contactedregarding joining our Investigatorteam.
Welch Allyn, Inc.4341 State Street Rd., Box 220Skaneateles, NY 13153-0220
Contact: Kathi DurdonEmail: [email protected]
Device name: Not availableIndication: Cardiac, pulmonary, neonatology,
pediatrics, emergency medicine,internal medicine, family medi-cine, acute care, critical care
Specialty: Device experienced researchersPhase: Class I & II DeviceNotes: Seeking device-experienced/inter-
ested clinical researchers forupcoming studies. Class I & II inneonatal, pediatric and adultpopulations; AMC, hospital andprivate practice settings.
TrialWatch is designed to help
sponsors and CROs identify a
pool of investigators for their
upcoming trials. Each sponsor that is listed
here has confirmed that it will be actively
selecting sites during the next few weeks,
and would like to receive inquiries from
investigative sites. Sponsors and CROs
that would like to use this service should
contact Tamar Skowronski at (617) 856-5974
or email [email protected].
Visit our web site at www.centerwatch.com/
professional/trialwatch.html to use Trial-
Watch online.
For investigators, this listing provides pre-
qualified leads for clinical grants. Please note:
Unless a phone or fax number is given, do
not call the sponsor or CRO. Sponsors have
provided this information to CenterWatch
with the understanding that investigative sites
will mail cover letters, CVs and other infor-
mation about their facilities, staff and
patients. Please inform the sponsor or CRO
that you learned of the project through
CenterWatch.
Investigators—Have you called a sponsor in
this issue of TrialWatch and learned that the
sponsor is no longer seeking sites? If so, please
call (617) 856-5974 to let us know so that we
may contact the sponsor and update our list-
ings accordingly.
TrialWatch
Note: If you would like further information on any drug listed above, or to review our comprehensive database of drugs in development, please visit www.centerwatch.com.
Drug Company Contact Additional Information
Phase III (continued)
Genasense Genta (908) 286-9800 targets Bcl-2, a protein over-expressed in many www.genta.com forms of cancer.
Ipilimumab Medarex (609) 430-2880 a fully human antibody that targets CTLA-4,(MDX-010) www.medarex.com a molecule on T cells that suppresses the immune
response
Allovectin-7 Vical (858) 646-1100 a DNA/lipid complex containing the human www.vical.com gene that encodes HLA-B7 antigen
Grant Opportunities
25August 2007CenterWatch
Cardiology/Vascular DiseaseStill Seeking Investigators
Duke Clinical Research Institute2400 Pratt St.Durham, NC 27705
Contact: Kifu FaruqEmail: [email protected]
Drug name: Oral direct Factor Xa inhibitorIndication: Non-valvular atrial fibrillation in
patients at high risk for eventsSpecialty: Cardiology, Neurology, Internal
Medicine, Hematology(Anticoagulation Clinic)
Phase: IIINotes: Primary objective is to demon-
strate that the efficacy of a oncedaily direct factor Xa inhibitor, isnon-inferior to dose-adjusted war-farin for the prevention of throm-boemoblic events in pts with non-valvular atrial fibrillation meas-ured by the composite of strokeand non-CNS systemic embolism.
Nicholas Piramal India Ltd.
Contact: Anjali SonawaneEmail: [email protected];
Drug name: Niyat (Atrovastatin+Niacin)Indication: HypercholesterolemiaSpecialty: CardiologyPhase: IVComments: Seeking Indian investigators
ProMedDx, LLC10 Commerce WayNorton, MA 02766
Contact: Marion M. Santa InesEmail: [email protected]
Drug name: Not applicableIndication: Congestive Heart FailureSpecialty: CardiologyPhase: Not applicableNotes: Only one study visit will be
required for this study. The onlyrisks to participants are those asso-ciated with a routine blood draw.
Tasly Pharmaceuticals, Inc.One Research Court, Suite 450-54Rockville, MD 20850
Contact: Jason GuoEmail: [email protected]
Drug name: T89Indication: Prevention of occurrence of stable
anginaSpecialty: PI specializing in cardiovascular
disease (angina), in a hospital, uni-versity, or medical research centers
Phase: II/IIbNotes: 2-herb drug for chronic stable
angina. Open, 500mg, baseline-controlled. 40 patients. Patientstake T89, stop long term nitrate,reduce current anti-angina to 50%on day 0, and to 0% on day 28 ifjustified, free to use on-demandnitrate as needed. Exercise tread-mill testing on days -14, 0, 28 & 56.
DermatologyStill Seeking Investigators
Light BioScience933 First Colonial Rd. Suite 204Virginia Beach, VA, 23454
Contact: Susan Kaplan RN, PhDEmail: [email protected]
Device name: GentleWavesIndication: Acne treatmentSpecialty: DermatologyPhase: II/ IIINotes: This is a 6-8 week trial of a new
treatment for facial acne inpatients ages 14-60 using LEDtechnology.
Emergency MedicineStill Seeking Investigators
Intranasal Therapeutics, Inc.1513 Bull Lea BlvdLexington, KY 40511
Contact: Kim NewEmail: [email protected]
Drug name: Not availableIndication: Acute PainSpecialty: ER Physician with research experi-
ence treating acute pain.Phase: II
EndocrinologyNew Leads
Anaclim6325 Digital Way, Suite 401Indianapolis, IN 46278
Contact: Randy BrownEmail: [email protected]
Drug name: Not availableIndication: Type II DiabetesSpecialty: EndocrinologyPhase: IVNotes: Insulin study for patients who are
currently receiving oral meds forthe treatment of Type II Diabetes.
Still Seeking Investigators
AlthoTech Pharmaceutical Inc.310 Glen Manor Dr. W.Toronto, Ontario, Canada M4E 2X7
Contact: Peter TomlinsonEmail: [email protected]
Drug name: ATPI-C01Indication: Treatment of ischemic and neuro-
pathic foot ulcersSpecialty: Clinical diabetologist; clinical der-
matologistPhase: INotes: ATPI-C01 is a patented intrale-
sional formualation of EGF fortreating diabetic foot ulcers.Treatment effective in end-stageDFU sized from 1-80 cm2, wherethe amputation is the only avail-able choice.
Genaera Corporation5110 Campus DrivePlymouth Meeting, PA 19462
Contact: Jim EllisEmail: [email protected]
Drug name: Not availableIndication: Diabetes Type IISpecialty: Phase I unit Obese Diabetes Type
IIPhase: INotes: We are looking for a Phase I unit
that can recruit 28 Obese Type IIDiabetes patients (7 subjects in 4dose groups), for a single IV dosestudy, patients will be housed for72 hours.
ProMedDx, LLC10 Commerce WayNorton, MA 02766
Contact: Marion M. Santa InesEmail: [email protected]
Drug name: Not applicableIndication: DiabetesSpecialty: EndocrinologyPhase: Not applicableNotes: Only one study visit will be
required for this study. The onlyrisks to participants are those asso-ciated with a routine blood draw.
Grant Opportunities
Thomson26 August 2007
GastroenterologyStill Seeking Investigators
Advanced Biologics580 Union Square DriveNew Hope, PA 18939
Contact: Wen LinEmail: [email protected]
Drug name: Not availableIndication: C. Difficile-associated diarrheaSpecialty: Infectious disease,
GastroenterologyPhase: IIINotes: Looking for sites in US and
Canada.
Alba Therapeutics800 West Baltimore St., Suite 800Baltimore, MD 21201
Contact: Sarah CappelloEmail: [email protected]
Drug name: AT-1001Indication: Celiac DiseaseSpecialty: Gastroenterology; Immunology-
with access to adult celiac diseasepatients. Experienced in conduct-ing clinical trials.
Phase: IIbNotes: Alba is recruiting investigators for
the following clinical trial: Subjects18-65, diagnosed with celiac dis-ease by small bowel biopsy, glutenfree for up to 6 mons and anti-tTGneg. Will evaluate the efficacy ofAT-1001 vs. drug placebo/gluten,in preventing exacerbation of celi-ac symptoms and intestinal per-meability when exposed to gluten.
Duke Clinical Research InstitutePO Box 17969Durham, NC 27715
Contact: Ron RoddyEmail: [email protected]
Drug name: Not availableIndication: Moderate to severe Crohn’s
DiseaseSpecialty: IBD specialist, GastroenterologyPhase: IIINotes: Looking for subjects refractory to
steroids, immunosupressants, andbiologicals. We need at least fivesubjects recruited over a 10 monthperiod.
ProMedDx, LLC10 Commerce WayNorton, MA 02766
Contact: Marion M. Santa InesEmail: [email protected]
Drug name: Not applicableIndication: Non-viral Liver DiseaseSpecialty: GastroenterologyPhase: Not applicableNotes: Only one study visit will be
required for this study. The onlyrisks to participants are those asso-ciated with a routine blood draw.
Wyeth Research500 Arcola Road, B4002Collegeville, PA 19426
Contact: Maria DattiloEmail: [email protected]
Drug name: PantoprazoleIndication: GERDSpecialty: Gastroenterology; MetabolismPhase: IIINotes: The study is seeking patients
between the ages of 1 through 11months with a diagnosis of pre-sumptive GERD requiring phar-macologic treatment.
Wyeth Research500 Arcola Road, B-4021Collegeville, PA 19426
Contact: Stacey LoReEmail: [email protected]
Drug name: pantoprazole/Protonix OralSuspension
Indication: GERDSpecialty: Neonatologist/Neonatal/Pediatric
GastroenterologistPhase: III with PK and pH assessmentsNotes: Need sites with access to a
Neonatal ICU. Protocol will studypHmetry and pharmacokinetics inpatients who are either term orpost term infants with presumedGERD who are within the neona-tal period of less than or equal to28 days. Procedures include twopHmetry assessments (1 atBaseline and 1 at Steady State)along with Single and MultipleDose PK collection. Study isenrolling but needs additionalinvestigators experienced in con-ducting pHmetry in infants, inorder to bolster recruitment.
HematologyStill Seeking Investigators
Cylene Pharmaceuticals5820 Nancy Ridge Drive, Suite 200San Diego, CA 92121
Contact: Claire PadgettEmail: [email protected]
Drug name: CX-3543Indication: Relapsed or refractory CLLSpecialty: Hematology; oncologyPhase: II
PRA International630 Dresher RoadHorsham, PA 19044
Contact: Toni BaileyEmail: [email protected]
Drug name: Not availableIndication: Graft vs. host diseaseSpecialty: Hematology; oncology; gastroen-
terologyPhase: IIINotes: Recruiting sites with patients who
have had a Bone MarrowTransplant (BMT) or Blood StemCell Transplant (PBSCT) and havea new onset of acute GvHD. Thisis a Phase III study. Each site isexpected to recruit 2-3 patients.The target populations arebetween ages 18-75 years.
Immunology/Infectious DiseaseNew Leads
BDpath Group310 Queen Street South, Unit 208Kitchener, ONCanada N2G 1K2
Contact: Yong ZhangEmail: [email protected]
Drug name: Not availableIndication: AIDSSpecialty: Immunology / infectious diseasePhase: II
Still Seeking Investigators
Advanced Biologics580 Union Square DriveNew Hope, PA 18939
Contact: Wen LinEmail: [email protected]
Grant Opportunities
27August 2007CenterWatch
Drug name: Not availableIndication: C. Difficile-associated diarrheaSpecialty: Infectious disease,
GastroenterologyPhase: IIINotes: Looking for sites in US and
Canada.
Catalyst Pharmaceuticals1111 S. Arroyo Parkway, Suite 200Pasadena, CA 91105
Contact: Antoinette TorresEmail: [email protected]
Drug name: Not availableIndication: Herpes ZosterSpecialty: Immunology/infectious diseasePhase: IIb
Covance Inc210 Carnegie Center BlvdPrinceton, NJ
Contact: Elizabeth PircEmail: [email protected]
Drug name: Not availableIndication: Influenza txSpecialty: Infectious Desease in a hospital
settingPhase: II
ICON CRO100 Commerce DriveNewark, DE 19713
Contact: Jillian CrillyEmail: [email protected]
Drug name: Not availableIndication: Hepatitis BSpecialty: Immunology, infectious diseasePhase: III
NephrologyStill Seeking Investigators
Advanced Magnetics, Inc.Clinical Trials Dept.61 Mooney StreetCambridge, MA 02138
Contact: Carrie DelaneyEmail: [email protected]
Drug name: ferumoxytolIndication: anemiaSpecialty: NephrologyPhase: IIINotes: Multicenter study
Angiotech Pharmaceuticals13921 Park Center Road, Suite 100Herndon, VA 20171
Contact: Andrew HarrisonEmail: [email protected]
Drug name: Vascular WrapTM paclitaxel andLifespan ePTFE Vascular Graft
Indication: Vascular Access for End StageRenal Disease
Specialty: Nephrology, Vascular Surgery,Interventional Radiology
Phase: PivotalNotes: A randomized, single blind trial to
assess the effectiveness of main-taining patency and safety of thepaclitaxel-eluting Vascular Wrapafter surgical implantation withthe Lifespan Graft in the upperextremity for hemodialysis vascu-lar access.
MedSource16902 El Camino RealHouston, TX 77058
Contact: Hope McPhersonEmail: [email protected]
Drug name: Not availableIndication: Renal AdenocarcinomaSpecialty: Nephrology, OncologyPhase: IIINotes: I am looking for investigators who
might be interested in participat-ing in a Phase III Vaccine study forpatients with Renal CellAdenocarcinoma. This is a greatdrug with a wonderful safety pro-file. Please let me know if any ofyour sites might be interested inmore information.
PRA International630 Dresher Rd.Horsham, PA 19044
Contact: Monica VolpeEmail: [email protected]
Drug name: Not availableIndication: Chronic kidney diseaseSpecialty: Nephrology – must be able to con-
duct phase I trials in addition tophases II & III
Phase: I-III
NeurologyNew Leads
Ortho-McNeil Janssen Scientific Affairs(OMJSA)62 Crescent Creek WaySelkirk, NY 12158
Contact: Rhonda LichtenwalnerEmail: [email protected]
Drug name: Not availableIndication: Essential tremorSpecialty: NeurologyPhase: IINotes: Seeking investigators that can use
central IRBs
Schering-Plough2000 Galloping Hill RoadMailstop K-15-3-3113Kenilworth, NJ 07033
Contact: Bohang Chen, MDEmail: [email protected]
Drug name: SCHIndication: Movement DisordersSpecialty: Tardive Dyskinesia, Drug Induced
Movement Disorder,Schizophrenia
Phase: IINotes: We are looking for investigators
who have conducted or are con-ducting studies for TardiveDiskinesia, Drug InducedMovement Disorder orSchizophrenia. The PIs can beinternational, but we prefer NorthAmerica.
Still Seeking Investigators
D.L. Anderson International3100 McKinnon Str, Ste #100Dallas, TX 75201
Contact: Nadia FarragEmail: [email protected]
Drug name: Not availableIndication: Partial epilepsySpecialty: NeurologyPhase: III
Ovation Pharmaceuticals, Inc.Four Parkway North, Suite 200Deerfield, IL 60015
Contact: Carmen MiceliEmail: [email protected]
Grant Opportunities
Thomson28 August 2007
Drug name: Anti-epilepsy drugIndication: Pediatric and adult patients with
active epilepsySpecialty: Neurologists with in-patient phase
I pK capabilitiesPhase: I
PrecisionMed132 N. Acacia AveSolana Beach, CA 92075
Contact: Carole MarksEmail: [email protected]: No DrugIndication: Probable Alzheimer’s Disease or
Mild Cognitive ImpairmentSpecialty: Neurology; PsychiatryPhase: Biological Sample CollectionNotes: This is a long term follow-up of
subjects with Mild Alzheimer’sDisease or Mild CognitiveImpairment. Subjects are seenevery 6 months and have a blooddraw, spinal tap, and rating scales.Any medications are allowed.
Teva Neuroscience999 de Maisonneuve Blvd. West, Suite 550Montreal, QuebecH3A 3L4
Contact: Lynda MillerEmail: [email protected]
Drug name: CopaxoneIndication: Relapsing Remitting Multiple
SclerosisSpecialty: NeurologyPhase: IV
OB/GynNew Leads
Covance Inc.Site Activation Services150 4th Avenue North, Suite 600Nashville, TN 37219
Contact: Erin GreunkeEmail: [email protected]
Drug name: PhenoxodiolIndication: Ovarian CancerSpecialty: Gynecologic OncologyPhase: IIINotes: The purpose of this project is to
see if phenoxodiol in combinationwith weekly carboplatin, comparedto a placebo with weekly carbo-platin, is effective against late stageovarian cancer. Ages Eligible forStudy: Females 18 years and above
Still Seeking Investigators
Ciphergen Biosystems, Inc.6611 Dumbarton CircleFremont, CA 94555
Contact: Gillian CrutcherEmail: [email protected]
Drug name: Not applicableIndication: Ovarian CancerSpecialty: Ob/Gyn; Gyn/OncPhase: Not applicableNotes: This study aims to differentiate
cancer from non-cancer inpatients with pelvic mass. The testis specifically directed towardsovarian cancer. We require onepre-op blood draw and the study iscompleted after the pathologyreport.
Cytokine PharmaSciences, Inc.150 S. Warner Road, Suite 420King of Prussia, PA 19406
Contact: Barbara PowersEmail: bpowers@
cytokinepharmasciences.com
Drug name: Misoprostol vaginal insertIndication: Cervical ripening and reducing
time to deliverySpecialty: Obstetrician or family practice
doctor with hospital privileges andin-patient clinial research capabili-ties
Phase: IIINotes: Have 35 sites, looking for 5 addi-
tional sites; study will recruit 900women requiring induction oflabor. Must induce at least 5women per week to qualify. Studystarts in April, 2006.
OncologyNew Leads
Covance Inc.Site Activation Services150 4th Avenue North, Suite 600Nashville, TN 37219
Contact: Erin GreunkeEmail: [email protected]
Drug name: PhenoxodiolIndication: Ovarian CancerSpecialty: Gynecologic OncologyPhase: III
Notes: The purpose of this project is tosee if phenoxodiol in combinationwith weekly carboplatin, comparedto a placebo with weekly carbo-platin, is effective against late stageovarian cancer. Ages Eligible forStudy: Females 18 years and above
Pharmatech, Inc.789 Sherman St., Suite 600Denver, CO 80203
Contact: Erin BeaverEmail: [email protected]
Drug name: CoFactorIndication: Metastatic colorectal cancerSpecialty: OncologyPhase: IIINotes: Official Title: A Phase III Multi-
Center Randomized Clinical Trialto Evaluate the Safety and Efficacyof CoFactor and 5-Fluorouracil (5-FU) Plus Bevacizumab VersusLeucovorin and 5-FU PlusBevacizumab as Initial Treatmentfor Metastatic ColorectalCarcinoma
Still Seeking Investigators
Accentia Biopharmaceuticals Inc.450 Park Avenue, South 12th FloorNew York, NY 10016
Contact: William Calhoun, MSEmail: [email protected]
Drug name: BioVaxidIndication: Follicular, Non-Hodgkin’s
LymphomaSpecialty: Immunology; Hematology;
OncologyPhase: IIINotes: The objective of this Phase 3 study
is to confirm the safety and effica-cy of BIOVAXID, an autologoustumor derived idiotype vaccine, asmeasured by prolongation of dis-ease-free survival of patients withfollicular (NHL) during their firstcomplete remission induced byPACE. Seeking several investigativesites in US, Canada and Mexico.Sites must be willing to use onlyPACE for remission induction. NoCHOP-R or Rituxin permitted.Patient-specific vaccine manufac-tored from biopsy materials.
Grant Opportunities
29August 2007CenterWatch
A.P. Pharma Inc.123 Saginaw DriveRedwood City, CA 94063
Contact: Erin O’BoyleEmail: [email protected]
Drug name: APF530: TEG-POE polymerBased Formulation Containing2% Granisetron
Indication: Prevention of Chemo-InducedNausea and Vomiting (CINV)
Specialty: OncologyPhase: IIINotes: The comparator in this trial is
Aloxi. Patients will be allowed toparticipate in up to 4 treatmentcycles and do not have to bechemotherapy naive.
Cephalon, Inc.CCRIChildren’s Hospital of Philadelphia3535 Market St., Suite 1200Philadelphia, PA 19104
Contact: Eileen DorseyPhone: (215) 590-1295Email: [email protected]
Drug name: Not availableIndication: Pediatric breakthrough painSpecialty: Pediatric oncologyPhase: II
Ciphergen Biosystems, Inc.6611 Dumbarton CircleFremont, CA 94555
Contact: Gillian CrutcherEmail: [email protected]
Drug name: Not applicableIndication: Ovarian CancerSpecialty: Ob/Gyn; Gyn/OncPhase: Not applicableNotes: This study aims to differentiate
cancer from non-cancer inpatients with pelvic mass. The testis specifically directed towardsovarian cancer. We require onepre-op blood draw and the study iscompleted after the pathologyreport.
Covance Inc.210 Carnegie CenterPrinceton, NJ 08540
Contact: Brenda RolfeEmail: [email protected]
Drug name: Phenoxodiol with carboplatinIndication: Relapse / recurrent carboplatin
resistent ovarian cancerSpecialty: Gynecology, OncologyPhase: IIINotes: Efficacy Study Comparing
Phenoxodiol in Combination withCarboplatin versus Carboplatinwith Placebo in Patients withPlatinum-Resistant or Platinum-Refractory Late-Stage EpithelialOvarian, Fallopian or PrimaryPeritoneal Cancer Following atLeast Second Line PlatinumTherapy
Cylene Pharmaceuticals5820 Nancy Ridge Drive, Suite 200San Diego, CA 92121
Contact: Claire PadgettEmail: [email protected]
Drug name: CX-3543Indication: Relapsed or refractory CLLSpecialty: Hematology; oncologyPhase: II
Delcath Systems1100 Summer StreetStamford, CT 06905
Contact: Dr. Samuel HerschkowitzPhone: (718) 624-6277Email: [email protected]
Drug name: MelphalanIndication: Cancer (melanoma)Specialty: Oncology, Interventional radiologyPhase: IIINotes: Delivery system for the isolated
hepatic arterial infusion ofchemotherapy to patients withmetastatic liver tumors.
GenVec, Inc.65 West Watkins Mill RoadGaithersburg, MD 20878
Contact: Renee Riggs-GarrettPhone: (240) 632-5582Email: [email protected]
Drug name: TNFeradeIndication: Pancreatic CancerSpecialty: OncologyPhase: II/IIINotes: Please contact us for additional
information to participate in thisstudy.
GTx , Inc.3 North DunlapMemphis, TN 38163
Contact: Susan FowlerEmail: [email protected]
Drug name: GTx-024Indication: Non-Small Cell Lung CancerSpecialty: OncologyPhase: IIbNotes: This study is for patients with
stage 2, 3, and 4 NSCLC who haveexperienced weight loss / musclewasting (cachexia).
ICON Clinical Research Canada Inc.7405 Transcanada Hwy, Suite 300St. Laurent, QC, H4T 1Z2 Canada
Contact: Stefan MilenkovEmail: [email protected]
Drug name: Not availableIndication: Non-small cell lung cancer
(NSCLC)Specialty: OncologyPhase: II
Imaging Diagnostic Systems6531 NW 18th CourtPlantation, FL 33313
Contact: Deborah O’BrienEmail: [email protected]
Device name: Laser Breast imaging deviceIndication: Detection of breast cancerSpecialty: Radiology mammographyPhase: Clinical collection phaseComments: Looking for clinical sites to collect
patient data in the dense breastclassification scheduled for biopsy.Need high volume sites.
Introgen Therapeutics, Inc.2250 HolcombeHouston, TX 77030
Contact: Julie Sicam, MT (ASCP) MSHSEmail: [email protected]
Drug name: Advexin (INGN 201)Indication: Squamous Cell Carcinoma of the
Head and NeckSpecialty: Oncologist/ENT SurgeonPhase: III
Grant Opportunities
Thomson30 August 2007
Notes: A Phase III, Multi-Center, Open-Label, Randomized Study toCompare the Effectiveness andSafety of IntratumoralAdministration of INGN 201 inCombination with ChemotherapyVersus Chemotherapy Alone inPatients with RecurrentUnresectable Squamous CellCarcinoma of the Head and Neck(SCCHN).
Javelin Pharmaceuticals125 CambridgePark DriveCambridge, MA 02140
Contact: Jennifer BurgEmail: [email protected]
Drug name: Intranasal KetamineIndication: Breakthrough pain in cancer
patientsSpecialty: Anesthesia; Pain Management;
OncologyPhase: III
MedSource16902 El Camino RealHouston, TX 77058
Contact: Hope McPhersonEmail: [email protected]
Drug name: Not availableIndication: Renal AdenocarcinomaSpecialty: Nephrology, OncologyPhase: IIINotes: I am looking for investigators who
might be interested in participat-ing in a Phase III Vaccine study forpatients with Renal CellAdenocarcinoma. This is a greatdrug with a wonderful safety pro-file. Please let me know if any ofyour sites might be interested inmore information.
Pharmacyclics999 East Arques AvenueSunnyvale, CA 94085
Contact: Priscilla HornEmail: [email protected]
Drug name: Xcytrin (Motexafin Gadolinium)Injection
Indication: Non-Small Cell Lung CancerSpecialty: Medical OncologyPhase: II
Notes: Randomized Phase II study ofMotexafin Gadolinium as secondline treatment of unresected local-ly advanced (Stage IIIB) or metas-tastic non-small cell lung cancer.Do not respond if you are a CROor SMO.
PPD3900 Paramount ParkwayMorrisville, NC 27560
Contact: Peidi GuEmail: [email protected]
Drug name: Not availableIndication: CTM; head & neck cancer therapySpecialty: OncologyPhase: I and II
Schering-Plough2015 Galloping Hill RoadKenilworth, NJ 07033
Contact: Siu-Long YaoEmail: [email protected]
Drug name: Not availableIndication: CancerSpecialty: Oncologist or physician experi-
enced with oncology trials.Phase: INotes: We are interested in sites that can
utilize central IRBs and have expe-rience with Phase I trials.
Seattle Genetics21823 30th Drive SEBothell, WA 98021
Contact: Dr. Eric SieversEmail: [email protected]
Drug name: SGN-30Indication: Cutaneous ALCL (Anaplastic
Large Cell Lymphoma)Specialty: Hematologist OncologistPhase: II/IIINotes: This is a rare form of lymphoma.
We would be interested in contact-ing research sites even if they onlysee 1-3 cases per year.
Vical Incorporated10390 Pacific Center CourtSan Diego, CA 92121
Contact: Linda Strause, PhDEmail: [email protected]
Drug name: Allovectin-7Indication: Metastatic MelanomaSpecialty: Medical OncologistPhase: III
Notes: This is a head-to-head superioritytrial of Allovectin-7 vs. first linechemotherapy in metastaticmelanoma patients where surgeryis not a curative option. This trialwill enroll 375 patients, random-ized 2: 1 to Allovectin-7. Pleasecontact Vical at [email protected] or call (877) 343-6389.
OphthalmologyStill Seeking Investigators
Rx Development Resources3110 Cherry Palm Drive, Suite 350Tampa, FL 33619
Contact: Penny CobbEmail: [email protected]
Drug name: Not availableIndication: Adenovirus/conjunctivitisSpecialty: Ophthalmology; Infectious diseasePhase: IINotes: Please contact me as soon as possi-
ble if you feel your site couldpotentially enroll 4-5 subjects in a3 month period.
Rx Development Resources3104 Cherry Palm Drive, Suite 260Tampa, FL 33619
Contact: Penny CobbEmail: [email protected]
Drug name: Not availableIndication: Dry AMDSpecialty: OphthalmologyPhase: IINotes: We are currently seeking potential
retinal research sites that have aMP-1 MicroPerimeter and a HRAII. At least one piece is required tobe considered as a potential site.
OtolaryngologyStill Seeking Investigators
Alcon Research, Ltd6201 South Freeway, TC-40Fort Worth, TX 76134
Contact: Sally F. BeezleyEmail: [email protected]
Drug name: Not availableIndication: Acute Otitis Media with
Tympanostomy Tube (AOMT)Specialty: Ear Nose and Throat (ENT) or
PediatricianPhase: III
Grant Opportunities
31August 2007CenterWatch
Notes: Must have access to the followingequipment: Vacuum suctioning,tympanometry, and audiology. Noplacebo arm in study; all activetreatment.
Pain ManagementStill Seeking Investigators
Javelin Pharmaceuticals125 CambridgePark DriveCambridge, MA 02140
Contact: Jennifer BurgEmail: [email protected]
Drug name: Intranasal KetamineIndication: Breakthrough pain in cancer
patientsSpecialty: Anesthesia; Pain Management;
OncologyPhase: III
Pediatrics/NeonatologyNew Leads
Biosolve638 Via CristinaNewbury Park, CA 91320
Contact: Andrea FriedmanEmail: [email protected]
Product: Infant formulaIndication: Infant nutritionSpecialty: Investigator with access to a popu-
lation of healthy infants followedfrom 3 months to a year. Infantsmay be breastfed or formula fed;either exclusively, or mixed.
Phase: II/IIINotes: Investigator must have access to
Hologic DEXA and/or Peapd.Anthropometrics will be moni-tored for this growth study. Inaddition, two blood draws, ataproximately 3 and 6 months ofage will be required.
GlaxoSmithKline - Vaccines/Biologicals2301 Rennaissance BoulevardKing of Prussia, PA 19406
Contact: Kerri MalloryEmail: [email protected]
Drug name: Not applicableIndication: VaccineSpecialty: PediatricsPhase: II-IV
Notes: GlaxoSmithKline Biologicals-Vaccines has a continuous risingneed for investigators and researchsites to assist with clinical researchtrials. We are interested in develop-ing mutually successful relation-ships with qualified sites.
Still Seeking Investigators
Boehringer Ingelheim Pharmaceuticals,Inc.900 Ridgebury Road, PO Box 368Ridgefield, CT 06877-0368
Contact: Patricia NormanEmail: [email protected]
heim.com
Drug name: Micardis (telmisartan)Indication: Pediatric HypertensionSpecialty: Pediatricians / Pediatric
NephrologistsPhase: IINotes: 4 week active treatment trial.
Children 6-17. Investigators withPK experience preferred.
Cephalon, Inc.CCRIChildren’s Hospital of Philadelphia3535 Market St., Suite 1200Philadelphia, PA 19104
Contact: Eileen DorseyPhone: (215) 590-1295Email: [email protected]
Drug name: Not availableIndication: Pediatric breakthrough painSpecialty: Pediatric oncologyPhase: II
Wyeth Research500 Arcola Road, B4002Collegeville, PA 19426
Contact: Maria DattiloEmail: [email protected]
Drug name: PantoprazoleIndication: GERDSpecialty: Gastroenterology; MetabolismPhase: IIINotes: The study is seeking patients
between the ages of 1 through 11months with a diagnosis of pre-sumptive GERD requiring phar-macologic treatment.
Wyeth Research500 Arcola Road, B-4021Collegeville, PA 19426
Contact: Stacey LoReEmail: [email protected]
Drug name: pantoprazole/Protonix OralSuspension
Indication: GERDSpecialty: Neonatologist/Neonatal/Pediatric
GastroenterologistPhase: III with PK and pH assessmentsNotes: Need sites with access to a
Neonatal ICU. Protocol will studypHmetry and pharmacokinetics inpatients who are either term orpost term infants with presumedGERD who are within the neona-tal period of less than or equal to28 days . Procedures include twopHmetry assessments (1 atBaseline and 1 at Steady State)along with Single and MultipleDose PK collection. Study isenrolling but needs additionalinvestigators experienced in con-ducting pHmetry in infants, inorder to bolster recruitment.
Phase I/Healthy VolunteersStill Seeking Investigators
Bristol-Myers SquibbPO Box 4000Princeton, NJ 08543-4000
Contact: Diane RandallEmail: [email protected]
Drug name: Not availableIndication: SchizophreniaSpecialty: PsychiatryPhase: I
Genaera Corporation5110 Campus DrivePlymouth meeting, PA 19462
Contact: Jim EllisEmail: [email protected]
Drug name: Not availableIndication: Diabetes Type IISpecialty: Phase I unit Obese Diabetes Type
IIPhase: I
Notes: We are looking for a Phase I unitthat can recruit 28 Obese Type IIDiabetes patients (7 subjects in 4dose groups), for a single IV dosestudy, patients will be housed for72 hours.
Johnson & Johnson PRD
Contact: Yvonne Tan Phone: (800) 362-6345, x 74060 Email: [email protected]
Drug name: Not available Indication: Healthy volunteers Specialty: Experience with healthy volunteersPhase: I Notes: Very interested in seeking cen-
ters/CROs on the West Coast thatrun phase I healthy-volunteerstudies, preferably in SouthernCalifornia.
Johnson & Johnson PRD
Contact: Juli E. LeDuc, M.A., CCRC,CCRA
Email: [email protected]
Drug name: variousIndication: Schizophrenia; sarcoma; HIV;
healthy-patient studiesSpecialty: Phase I unitsPhase: INotes: Seeking MDs with some Phase I
research experience who are ableto utilize central IRB for patientstudies as well as healthy-volunteerstudies. Sites should be located inthe United States only, Northeastregion—as far West as Indiana,and as far South as Virginia andEastern Canada.
Schering-Plough2015 Galloping Hill RoadKenilworth, NJ 07033
Contact: Siu-Long YaoEmail: [email protected]
Drug name: Not availableIndication: CancerSpecialty: Oncologist or physician experi-
enced with oncology trialsPhase: INotes: We are interested in sites that can
utilize central IRBs and have expe-rience with Phase I trials.
Psychiatry/PsychologyStill Seeking Investigators
Appian Services, LLC10107 White Cascade DriveCharlotte, NC 28269
Contact: Grier Harris, PharmDEmail: [email protected]
Drug name: Not availableIndication: Cocaine abuse and addictionSpecialty: PsychologyPhase: IINotes: Study: new agent for cocaine
addiction. Population: adults withestablished history of cocaine use.Enrollment goal is 60, to completea minimum of 40. Study duration:14-16 weeks. Total of 34-40 visits.Target enrollment period is 6months.
Bristol-Myers SquibbPO Box 4000Princeton, NJ 08543-4000
Contact: Diane RandallEmail: [email protected]
Drug name: Not availableIndication: SchizophreniaSpecialty: PsychiatryPhase: I
PulmonologyStill Seeking Investigators
Forest Research InstituteHarborside Financial Center, Plaza VJersey City, NJ 07311
Contact: Jessica HughesEmail: [email protected]
Drug name: Not availableIndication: Chronic Obstructive Pulmonary
DiseaseSpecialty: PulmonologyPhase: IIINotes: Experienced with exercise toler-
ance studies. Site needs to haveaccess to plethysmograph and elec-tronically braked cycle ergometer.
Novartis1 Health PLaza, 435-1175East Hanover, NJ
Contact: Sandeep AthalyeEmail: [email protected]
Drug name: Not availableIndication: COPD, Cystic FibrosisSpecialty: MD with Respiratory
SpecializationPhase: I-IIComments: CROs with strength in COPD or
Cystic Fibrosis fields preferred.
Pacific Clinical Center17337 Ventura Blvd., Suite #226Encino, CA 91316
Contact: Janet JohnsonEmail: [email protected]
Drug name: Not availableIndication: AsthmaSpecialty: PulmonologyPhase: IIINotes: Investigator must be Board certi-
fied and located in the Los Angelesarea.
RheumatologyStill Seeking Investigators
CombinatoRx, Inc.245 First St., 16th Fl.Cambridge, MA 02142
Contact: Gretchen L. ChildsEmail: [email protected]
Drug name: CRx-102Indication: OsteoarthritisSpecialty: Osteoarthritis; Internal MedicinePhase: IINotes: Study Objectives: To investigate
the efficacy and dose response ofCRx-102, a novel oral synergisticfixed dose combination product,in Osteoarthritis of the Knee.
CombinatoRx, Inc.245 First St., 16th Fl.Cambridge, MA 02142
Contact: James KeaneEmail: [email protected]
Drug name: CRx-102Indication: Rheumatoid ArthritisSpecialty: Rheumatology; Internal MedicinePhase: IIbNotes: Study Objectives: To investigate
the efficacy of CRx-102, a noveloral synergistic fixed dose combi-nation product, in RheumatoidArthritis patients on stableDMARD therapy.
Grant Opportunities
Thomson32 August 2007
Grant Opportunities
33August 2007CenterWatch
Pacific Clinical Center17337 Ventura Blvd., Suite #226Encino, CA 91316
Contact: Janet JohnsonEmail: [email protected]
Drug name: Not availableIndication: ArthritisSpecialty: RheumatologyPhase: IIINotes: Investigator must be Board certi-
fied and located in the Los Angelesarea.
Strategic Science & Technologies58 Charles StreetCambridge, MA 02141
Contact: Eric FosselEmail: [email protected]
Drug name: Transdermal ibuprofenIndication: Arthritis and painSpecialty: Any who deal with arthitis, joint or
muscle painPhase: PilotNotes: Our transdermal ibuprofen prod-
uct is potentially revolutionary asit provides effective localized reliefof pain at a much lower total bodydose than oral ibuprofen and itavoids the complication of stom-ach irritation and ulceration.
Wyeth Research500 Arcola Rd.Collegeville, PA 19426
Contact: Patti ShottEmail: [email protected]
Drug name: Not availableIndication: Rheumatoid ArthritisSpecialty: RheumatologyPhase: II and III
SurgeryNew Leads
Auxilium Pharmaceuticals40 Valley Stream ParkwayMalvern, PA 19355
Contact: Brian WalkerEmail: [email protected]
Drug name: AA4500Indication: Dupuytren’s ContractureSpecialty: Orthopedic Surgeon; Hand
SurgeonPhase: III
Notes: Auxilium Pharmaceuticals is cur-rently recruiting doctors to partici-pate in a Phase III clinical trialinvolving patients withDupuytren’s Contracture. Thistrial is studying an enzyme injec-tion as a non-surgical treatmentfor patients who have Dupuytren’sContracture for Dupuytren’sDisease.
Still Seeking Investigators
Angiotech Pharmaceuticals13921 Park Center Road, Suite 100Herndon, VA 20171
Contact: Andrew HarrisonEmail: [email protected]
Drug name: Vascular WrapTM paclitaxel andLifespan ePTFE Vascular Graft
Indication: Vascular Access for End StageRenal Disease
Specialty: Nephrology, Vascular Surgery,Interventional Radiology
Phase: PivotalNotes: A randomized, single blind trial to
assess the effectiveness of main-taining patency and safety of thepaclitaxel-eluting Vascular Wrapafter surgical implantation withthe Lifespan Graft in the upperextremity for hemodialysis vascu-lar access.
Anika Therapeutics, Inc.Clinical Department236 West Cummings ParkWoburn, MA 01801
Contact: Clinical DepartmentEmail: [email protected]
Device name: UnavailableIndication: OsteoarthritisSpecialty: Orthopedic surgeryPhase: IIINotes: Anika Therapeutics, Inc. is cur-
rently seeking orthopedic surgeonsfor a Phase III trial of OA.Preference will be given to investi-gator with prior research experi-ence. Please email current CV andcontact information if interested.
SkyePharma Inc.10450 Science Center DriveSan Diego, CA 92121
Contact: Marius Ardeleanu, MDEmail: [email protected]
Device name: DepoBupivacaineIndication: Postoperative analgesiaSpecialty: Anorectal surgeryPhase: IINotes: Looking for suitable clinical site to
conduct a dose-ranging study inpatients undergoing 2- or 3-col-umn excisional hemorrhoidecto-my using Milligan-Morgan orFerguson-type techniques undergeneral anesthesia, includingnewer approaches with specializedinstruments, such as LigaSure andharmonic scalpel. Need site withlarge volume of cases to ensuretimely enrollment (target enroll-ment for each site is 10 subjects in3 months).
UrologyStill Seeking Investigators
PPD, Inc.7551 Metro Center Dr, Ste 300Austin, TX 78744
Contact: Dave MatthewsEmail: [email protected]
Drug name: Not availableIndication: Benign Prostatic Hyperplasia
(BPH)Specialty: UrologyPhase: II
Sanofi-Aventis9 Great Valley ParkwayMalvern, PA 19355
Contact: Martha RitzEmail: [email protected]
Drug name: Not availableIndication: Overactive bladder/urge inconti-
nenceSpecialty: Urology; OB/GYN; Family CarePhase: IIb
Grant Opportunities
Thomson34 August 2007
Opportunities Initiating
Indication Sponsor Drug/Device Date Planned Additional Information
Phase I
cancer Kinex KX2-391 September 2007 Plans to enroll subjects with solid tumors and lymphoma in the US
Phase II
Gram-positive Arpida iclaprim (iv) H2, 2007 Plans to enroll 130 subjects with pneumonia hospital-acquired, ventilator-associated
or healthcare-associated pneumonia
organ rejection LifeCycle Pharma LCP-Tacro 2007 Plans to enroll 60 kidney transplant recipients in the US and Canada
acute ischemic stroke Nuvelo alfimeprase H2, 2007 Plans to enroll 100 subjects within 3-9 hours of stroke onset.
non-small cell Peregrine bavituximab 2007 Plans to enroll up to 49 subjects in Indialung cancer
Phase III
chronic renal failure Affymax Hematide Q4, 2007 Plans to 2,200 subjects with chronic renalfailure, including those on dialysis and not on dialysis.
type 1 diabetes Generex Oral-lyn Q3/Q4, 2007 Plans to enroll 750 subjects internationally
liver cancer Progen PI-88 2007 Plans to enroll 800 subjects with hepatocellular carcinoma internationally
obesity Vivus Qnexa H2, 2007 Plans to enroll 4,500 obese subjects (BMI greater than 30) and those with associated co-morbidities
This section provides investigative sites with a listing of phase
I through phase IV programs that are being initiated by
sponsors. Sites can use this information to track up and
coming studies that may offer grant opportunities. Please note:
these companies have not announced that they are actively seeking
candidates sites.
T H O M S O N C E N T E R WAT C H
22 Thomson Place · Boston, MA 02210 www.centerwatch.com
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The 2007 edition includes:100 new charts Hundreds of updated charts from the 2006 editionNew articles on each chapter topicUpdated pipeline activity for major therapeutic areas
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At Inclinix, we take your perspective – the clinicalperspective – and integrate it into a recruitment processthat delivers results. Well before the trial begins, Inclinixuses our technology solutions to make informed investigator selections. We equip each site with the tools necessary to convert interested study candidates into consented patients. Because enrollment time lines are critical, we providedirect-to-patient advertising and contact center solutions to refer even more quali�ed candidates into your trial.
With productive investigators and quali�ed patients,we keep them interested and motivated to ensure trial completion using customized retention strategies.
Don’t leave your Clinical Trial Recruitment to chance.Call Inclinix today.
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