Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)
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|ClinicalTrials.gov Identifier: NCT01625559|
Recruitment Status : Unknown
Verified August 2012 by CHABiotech CO., Ltd.
Recruitment status was: Active, not recruiting
First Posted : June 21, 2012
Last Update Posted : February 18, 2015
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.
|Condition or disease||Intervention/treatment||Phase|
|Stargardt's Macular Dystrophy||Biological: MA09-hRPE||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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)|
|Study Start Date :||September 2012|
|Estimated Primary Completion Date :||March 2015|
|Estimated Study Completion Date :||June 2015|
Experimental: 50,000 cells
Biological: MA09-hRPE Cellular therapy
MA09-hRPE: 50,000 cells
- safety and tolerance of transplantation [ Time Frame: 18 months ]
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 ]
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|