CIRM Funded Clinical Trials

Phase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa


Henry Klassen
Disease Area: 
Investigator:
Institution:
CIRM Grant:
Award Value:
$8,295,750
Trial Sponsor:
Trial Stage: 
Phase 2
Trial Status: 
Recruiting
Targeted Enrollment:
85
ClinicalTrials.gov ID:
Details: 

Retinitis pigmentosa (RP) is a severe form of blindness that runs in families with an incidence of 1:4000. Good target for stem cell therapy due to the defined loss of specific cells. Proposed mechanism: Rescue the light sensing photoreceptors. Follow-on study based on Phase 1/2a clinical trial. Continue to assess safety and establish efficacy.

Design: 

Single dose. Randomized 1:1. Placebo-controlled with crossover option after 12 month follow up of treated group.

Goal: 

Safety and efficacy - improvement in visual function at 12 months. 

Status: 

This study is currently recruiting participants. RMAT designation awarded.