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

Overview

The purpose of this study is to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD

Full Title of Study: “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 Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: April 2015

Detailed Description

– to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD – to evaluate the preliminary efficacy of MA09-hPRE in patients with SMD

Interventions

  • Biological: MA09-hRPE
    • MA09-hRPE: 50,000 cells

Arms, Groups and Cohorts

  • Experimental: 50,000 cells
    • Biological: MA09-hRPE Cellular therapy

Clinical Trial Outcome Measures

Primary Measures

  • 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

Secondary Measures

  • 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

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 20 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • CHABiotech CO., Ltd
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Wonkyung Song, Principal Investigator, CHA Bundang Medical Center

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