ACT is conducting with two separate FDA-approved Phase 1/2 clinical trials to test the safety of the human embryonic stem cell (hESC)-derived retinal pigment epithelial (RPE) cellular therapy for Dry Age-Related Macular Degeneration (Dry AMD) and for Stargardt's Macular Dystrophy (SMD).
The Phase 1/2 trials are prospective, open-label studies designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with SMD and Dry AMD.
Advanced Dry Age-Related Macular Degeneration (Dry AMD)
The open-label, non randomized, sequential, multi-center safety and tolerability trial is evaluating the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with dry AMD and to perform exploratory evaluation of potential efficacy endpoints to be used in future studies RPE cellular therapy. Twelve patients will be enrolled sequentially, and the clinical course of each patient treated will be reviewed by an independent Data Safety Monitoring Board (DSMB) before the next patient is enrolled. Each patient will receive a single uniocular subretinal infusion of MA09-hRPE cells in one of four dose levels (50,000 cells - 200,000 cells). more information about the enrollment status of these clinical trials, visit our company news section.
Primary endpoint is the safety of hESC derived RPE cells at 12 months. Secondary endpoints will be evaluated as exploratory evaluations for potential efficacy endpoints at 12 months.
The principal investigator of the study is Steven Schwartz, M.D., Jules Stein Eye Institute, University of California, Los Angeles (UCLA).
For detailed information, please visit: http://www.clinicaltrials.gov/ct2/show/study/NCT01344993