BALANCE

WP 6: Preparation clinical trial (M9-M31) (AMC, HepArt & UEDIN)

Objectives

• Objective:• To obtain regulatory approval for the planned phase I/IIa study

and to make practical preparations for clinical testing.

• 6.1. to prepare all necessary organisational arrangements

• 6.2. to complete study protocol, investigator brochure and acquire approval from regulatory bodies

• 6.3. to instruct and train clinical personnel

BALANCE

WP6: Description of work

• 6.1. - Designing investigator database (website)

- Notifying the study (CCMO & MHRA)

- Trial insurance aspects

- Contracts for finances

• 6.2. - Setting up: clinical protocol, IMPD, investigators brochure, CRF’s, monitoring forms, informed consent.

• - Getting approval of CCMO and MHRA

• 6.3 - instruction and training ICU personnel

BALANCE

WP6: Deliverables & Milestone

• Deliverables

- Report on organisational arrangements (M31)

- Regulatory approval (M31)

- Instruction personnel finished (M28).

• MILESTONE

Approval of clinical trial (M31)

BALANCE

WP 7:Clinical trial(AMC, PharmaCell & UEDIN)

Objectives

• To show safety and feasibility of HepaRG-BAL in 10 ALF patients

• 7.1. To produce and transport the bioreactor cultures to hospital locations

• 7.2. To determine safety & feasibility of BAL treatment in ALF patients

• 7.3. To determine the effect of patient treatment on HepaRG functionality

BALANCE

WP7: Description of work

• 7.1. Supplying bioreactor cultures (PhC)

- 20 fully functional and quality controlled HepaRG-BALS

- Shipment, using transport system of WP3 (M28)• 7.2. Treatment of patients and data analysis (UEDIN & AMC)

- Treatment according to clinical protocol

- Data analysis and reporting results and adverse events to monitoring committee

• 7.3. Post treatment analysis of bioreactor cultures:

- Study of possible toxic effect of ALF plasma on functionality and secretome

BALANCE

WP7: Deliverables & Milestone

• Deliverables

- Report on supply of bioreactor cultures to hospitals.Certificate of quality analysis and delivery report (PhC)

- Report on safety, efficacy and feasibility of HepaRG BAL treatment of ALF patients (UEDIN & AMC)

- Report on the effect of ALF plasma on HepaRG BAL (UEDIN & AMC).

• MILESTONESafety and feasibility of HepaRG BAL treatment of ALF patients (M36)