Clinical Program

Stargardt's Macular Dystrophy

The FDA-approved clinical trial is a Phase 1/2 open-label, multi-center, non-randomized, prospective study to test the safety and tolerability of sub-retinal transplantation of hESC-derived RPE (MA09-hRPE) cells in patients with Stargardt's Macular Dystrophy. The FDA has granted ACT's RPE cells "Orphan" status for treatment of Stargardt's Disease.

Patients will undergo subretinal injection of MA09-hRPE cellular therapy in one of four dose levels (50,000 cells - 200,000 cells). Patients will be enrolled sequentially, and within each cohort of 3 patients, each patient's clinical course over the first 6 weeks following cell transplantation will be reviewed by an independent (DSMB) before enrollment is opened for the next 2 patients. A full safety assessment of all 3 patients in each cohort will be made by the DSMB when the 3rd patient in each cohort completes 4 weeks of follow-up, and before the first patient in the next cohort receives a cell transplant. Each cohort will be enrolled sequentially in turn. The day of the cell implantation will be Day 0, and patients will remain in the study until the last visit at 12 months.

The primary endpoint is the safety and tolerance of transplantation of hESC-derived RPE cells MA09-hRPE at 12 months. Secondary outcome measures include the evidence of successful engraftment at 12 months.

The principal investigators of the study are Steven Schwartz, M.D., Jules Stein Eye Institute, University of California, Los Angeles (UCLA), and Peter J. Francis, M.D., Ph.D., Casey Eye Institute (CEI) at Oregon Health & Science University (OHSU), Portland, Oregon.

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