Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)
The purpose of this study is:
- To evaluate the safety and tolerability of RPE cellular therapy in patients with SMD Group
- When-MA09-hRPE cell transplantation to evaluate the safety of surgical procedures.
- In future studies intended to assess the number of transplanted hRPE cells.
- In the past, MA09-hRPE cell therapy used in the study was to evaluate the validity of the potential.
- Homologous retinal pigment epithelial cells derived from embryonic stem cells, future studies of drugs that are used in representing the potential validity to evaluate the optimal dose.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I, Open-Label, 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)|
- safety and tolerance of transplantation [ Time Frame: 18 months ] [ Designated as safety issue: Yes ]
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of:
- 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 [ Time Frame: 18 months ] [ Designated as safety issue: No ]
Evidence of successful engraftmentEvidence of successful engraftment will consist of:
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
|Study Start Date:||September 2012|
|Estimated Study Completion Date:||June 2015|
|Estimated Primary Completion Date:||March 2015 (Final data collection date for primary outcome measure)|
Experimental: 50,000 cells
Biological: MA09-hRPE Cellular therapy
MA09-hRPE: 50,000 cells
Please refer to this study by its ClinicalTrials.gov identifier: NCT01625559
|Korea, Republic of|
|CHA Bundang Medical Center|
|Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712|
|Principal Investigator:||Wonkyung Song, MD. PhD.||CHA Bundang Medical Center|