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

Phase 1

Completed

• Conditions: Stargardt's Macular Dystrophy
• Interventions: Biological: MA09-hRPE

• Registration Number: NCT01625559
• Lead Sponsor: CHABiotech CO., Ltd

Brief Summary

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

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

Study Timeline

• Study First Submitted: 2012-06-18
• Study First Submitted QC: 2012-06-19
• Study First Posted: 2012-06-21
• Study Start: 2012-09
• Primary Completion: 2015-04
• Study Completion: 2015-04
• Status Verified: 2024-02
• Last Update Submitted: 2024-04-08
• Last Update Posted: 2024-04-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 3

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
MA09-hRPE administration	MA09-hRPE	Biological: MA09-hRPE Cellular therapy

Primary Outcome Measures

Name	Time	Method
safety and tolerance of transplantation	18 months	The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of: 1. Any grade 2 (NCI grading system) or greater adverse event related to the cell product; 2. Any evidence that the cells are contaminated with an infectious agent; 3. Any evidence that the cells show tumorigenic potential

Secondary Outcome Measures

Name	Time	Method
Evidence of successful engraftment	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

Trial Locations

Locations (1)

CHA Bundang Medical Center; 🇰🇷 Seongnam-si, Gyeonggi-do, Korea, Republic of