Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy

General Information

Summary This is a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with Stargardt's Macular Dystrophy (SMD).
Description This study is a Phase I/II, open-label, non randomized, sequential, multi-center clinical trial. There will be 5 cohorts, the 4 low vision cohorts will contain 3 patients, the better vision cohort will contain 4 patients. The enrolled cohorts will be as follows: Three SMD patients- 50,000 MA09-hRPE cells transplanted Three SMD patients- 100,000 MA09-hRPE cells transplanted Four Better Vison SMD patients- 100,000 MA09-hRPE cells transplanted Three SMD patients- 150,000 MA09-hRPE cells transplanted Three SMD patients- 200,000 MA09-hRPE cells transplanted 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. The exception is the better vision group where all patients may be enrolled once DSMB approval has been received. Each cohort will be enrolled sequentially in turn, with the exception of the better vision cohort which may be enrolled in parallel with the other cohorts. The day of the cell implantation will be Day 0, and patients will remain in the study until the last visit at 12 months.
Clinical trials phase Phases 1/2
Start date (estimated) 2011-04-01
End date (estimated) 2015-08-31
Clinical feature
Label Stargardt Disease
Link http://purl.obolibrary.org/obo/NCIT_C85078
Description An autosomal recessive and rarely autosomal dominant inherited disorder caused by mutations in the ABCA4 or ELOVL4 genes respectively. It is characterized by macular degeneration that begins in late childhood resulting in progressive loss of vision.
Publications

Administrative Information

NCT Number NCT01345006
Other study identifiers
Name 7316-CL-0001
Name ACT SMD 01 MA09-hRPE
Description (Other Identifier: Sponsor)
Source weblink http://www.clinicaltrials.gov/ct2/show/NCT01345006
Regulatory body approval
Name Food and Drug Administration (FDA)
Country
United States
Public contact
Country
Sponsors Astellas Pharma Inc.

Cell Line

Stem cell lines obtained from

Recruitment

Recruitment Status Completed
Estimated number of participants 13