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) 2011-11-01
End date (estimated) 2015-09-30
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 NCT01469832
ICTRP weblink http://apps.who.int/trialsearch/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
Country
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 Pharma Inc.

Cell Line

Stem cell lines obtained from

Recruitment

Recruitment Status Completed
Estimated number of participants 12