Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||Long Term Follow Up to a Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (MA09-hRPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)|
- The safety and tolerance of transplantation of hESC-derived RPE cells MA09-hRPE [ Time Frame: 15 years ] [ Designated as safety issue: Yes ]
- Any grade 2 (NCI grading system) or greater adverse event related to the cell product
- Any evidence that the cells are contaminated with an infectious agent
- Any evidence that the cells show tumorigenic potential
- Evidence of Successful Engraftment of MA09-hRPE Cells [ Time Frame: 15 years ] [ Designated as safety issue: No ]
- Structural evidence (OCT imaging, fluorescein angiography, autofluorescense photography, slit-lamp examination with fundus photography) that cells have been implanted in the correct location
- Electroretinographic evidence (mfERG) showing enhanced activity in the implant location
- Evidence of Rejection of MA09-hRPE Cells [ Time Frame: 15 years ] [ Designated as safety issue: No ]• Structural (imaging) evidence that implanted MA09-hRPE cells are no longer in the correct location or the presence of vascular leakage
|Study Start Date:||July 2012|
|Estimated Study Completion Date:||December 2030|
|Estimated Primary Completion Date:||December 2029 (Final data collection date for primary outcome measure)|
Sub-retinal transplantation of MA09-hRPE cells
Cohort 1 50,000 cells Cohort 2 100,000 cells Cohort 2a Better Vision 100,000 cells Cohort 3 150,000 cells Cohort 4 200,000 cells
This study is a long term, non interventional follow up to the phase I/II, open-label, non randomized, sequential, multi-center trial (referred to as the core protocol) in which a maximum of 13 SMD patients were treated. Ten poor vision subjects(20/400) were transplanted with sequential doses of hESC-RPE cells, starting at a dose of 50,000 hESC-RPE cells transplanted (three subjects), 100,000 hESC-RPE cells transplanted (three subjects), 150,000 hESC-RPE cells transplanted (three subjects) and increasing to a maximum dose of 200,000 hESC-RPE cells transplanted(one subject). Three better vision subjects(20/100) were transplanted with a dose of 100,000 hESC-RPE cells.
Eligible patients will be those who sign a consent form for the extension study, and who fulfill all inclusion/exclusion criteria.
The first visit of this extension protocol will correspond to the last visit of the core protocol, and will take place at 12 months post cell implantation.
During the first 2 years after completion of the core protocol, i.e. from Year 1 to Year 2 and from Year 2 to Year 3 post transplantation, the patients will be evaluated at approximately 3 month intervals; during years three thru five patients will be evaluated at approximately six month intervals. During years 6-15, patients will either be required to return to the study center or provide the name of an ophthalmologist in their locality who would provide an evaluation of the patient's vision and any abnormal findings and report these results to the study site annually.
Follow up will include obtaining information about visual acuity, ophthalmological findings and adverse events (AEs), specifically including cancer, neurological, autoimmune or hematological disorders that developed or worsened since the last visit.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02445612
|United States, California|
|Jules Stein Eye Institute, UCLA School of Medicine|
|Los Angeles, California, United States, 90095|
|United States, Florida|
|Bascom Palmer Eye institute|
|Miami, Florida, United States, 33136|
|United States, Pennsylvania|
|Wills Eye Institute-Mid Atlantic Retina|
|Philadelphia, Pennsylvania, United States, 19107|
|Principal Investigator:||Steven Schwartz, MD||Jules Stein Eye Institute, UCLA School of Medicine|
|Principal Investigator:||Carl D Regillo, MD||Wills Eye Institute-Mid Atlantic Retina|
|Principal Investigator:||Byron L Lam||University of Miami|