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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) 2011-06-16
End date (estimated) 2015-08-10
Clinical feature
Label Stargardt disease
Link http://purl.obolibrary.org/obo/DOID_0050817
Description An age related macular degeneration that is characterized by progressive vision loss usually to the point of legal blindness.
Publications

Administrative Information
NCT number NCT01345006
ICTRP weblink https://trialsearch.who.int/Trial2.aspx?TrialID=NCT01345006
Other study identifiers
Name 7316-CL-0001
Name ACT SMD 01 MA09-hRPE
Description (Other Identifier: Sponsor)
Name ACT MA09-hRPE SMD-001
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
Email astellas.registration@astellas.com
Public email astellas.registration@astellas.com
Last name Study Director: Global Therapeutic Area Head & Chief Medical Officer, Astellas Institute for Regenerative Medicine
Country
United States
Sponsors Astellas Institute for Regenerative Medicine

Cells
Source pluripotent stem cell lines
Which differentiated cell type is used
Label retinal pigment epithelial cell
Link http://purl.obolibrary.org/obo/CL_0002586
Description An epithelial cell of the retinal pigmented epithelium.

Recruitment
Recruitment Status Completed
Estimated number of participants 13