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Clinical Trial 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).


Clinical Trial 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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT01345006
Study type Interventional
Source Astellas Pharma Inc
Contact
Status Completed
Phase Phase 1/Phase 2
Start date June 16, 2011
Completion date August 10, 2015

See also
  Status Clinical Trial Phase
Completed NCT01625559 - Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD) Phase 1
Active, not recruiting NCT02749734 - Clinical Study of Subretinal Transplantation of Human Embryo Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Macular Degeneration Diseases Phase 1/Phase 2
Completed NCT02445612 - Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients
Completed NCT02941991 - A Follow up Study to Determine the 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)
Active, not recruiting NCT03364153 - Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1) Phase 2
Completed NCT01469832 - 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) Phase 1/Phase 2