Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)

Study Overview
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Brief Summary
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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.

Official Title
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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)

Conditions
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Stargardt's Macular Dystrophy

Intervention / Treatment
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* Biological: MA09-hRPE


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Adult male or female over 20 years of age. Clinical diagnosis of advanced SMD. The visual acuity of the eye to receive the transplant will be no better than hand movement. The visual acuity of the eye that is not to receive the transplant will be no better than 24 (20/320) Early Treatment of Diabetic Retinopathy Study (ETDRS) letters. Exclusion Criteria: History of malignancy. History of myocardial infarction in previous 12 months. History of diabetes mellitus. Any immunodeficiency. Any current immunosuppressive therapy other than intermittent or low dose cortico steroids. Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV. Current participation in any other clinical trial. Participation within previous 6 months in any clinical trial of a drug by ocular or systemic administration. Any other sight-threatening ocular disease. Any chronic ocular medications. Any history of retinal vascular disease (compromised blood-retinal barrier). Glaucoma. Uveitis or other intraocular inflammatory disease. Significant lens opacities or other media opacity. Ocular lens removal within previous 3 months

Ages Eligible for Study
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Minimum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 50,000 cells<br>Biological: MA09-hRPE Cellular therapy | Biological: MA09-hRPE<br>* MA09-hRPE: 50,000 cells<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| safety and tolerance of transplantation | 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 | 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evidence of successful engraftment | 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 | 18 months |