Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)

General Information
Summary	The purpose of this study is: To evaluate the safety and tolerability of RPE cellular therapy in patients with SMD . To evaluate potential efficacy endpoints to be used in future studies RPE cellular therapy.
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 Vision 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)	2011-12-13
End date (estimated)	2015-09-30
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	Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies Embryonic stem cell trials for macular degeneration: a preliminary report Transplantation of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells in Macular Degeneration
Administrative Information
NCT number	NCT01469832
ICTRP weblink	https://trialsearch.who.int/Trial2.aspx?TrialID=NCT01469832
EudraCT number	2011-000054-34
Other study identifiers	Name 7316-CL-0003 Name ACT hESC-RPE SMD 01 EU Description (Other Identifier: Sponsor)
Source weblink	http://www.clinicaltrials.gov/ct2/show/NCT01469832
Regulatory body approval	Name Medicines & Healthcare products Regulatory Agency (MHRA) Country United Kingdom
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	ACTe002-A
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	12