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

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)

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01625559

Enrollment

Registered

2012-06-21

Start date

2012-09-30

Completion date

2015-04-30

Last updated

2024-04-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Stargardt's Macular Dystrophy

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

Interventions

BIOLOGICALMA09-hRPE

MA09-hRPE cells

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

20 Years to No maximum

Healthy volunteers

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

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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

Countries

South Korea

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 17, 2026