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

NCT01625559 | Completed Phase 1 DMC

• 1 other identifier: CHA_CTP_0903
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Stargardt's Macular Dystrophy

Outcome Measures

Primary Outcomes (1)

• safety and tolerance of transplantation

  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

  18 months

Secondary Outcomes (1)

• Evidence of successful engraftment

  18 months

Study Arms (1)

MA09-hRPE administration

EXPERIMENTAL

Biological: MA09-hRPE Cellular therapy

Biological: MA09-hRPE

Interventions

MA09-hRPE BIOLOGICAL

MA09-hRPE cells

MA09-hRPE administration

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• CHABiotech CO., Ltd: lead

Study Sites (1)

CHA Bundang Medical Center

Seongnam-si, Gyeonggi-do, 463-712, South Korea

Location

Related Publications (1)

• Sung Y, Lee MJ, Choi J, Jung SY, Chong SY, Sung JH, Shim SH, Song WK. Long-term safety and tolerability of subretinal transplantation of embryonic stem cell-derived retinal pigment epithelium in Asian Stargardt disease patients. Br J Ophthalmol. 2021 Jun;105(6):829-837. doi: 10.1136/bjophthalmol-2020-316225. Epub 2020 Jul 29.

  PMID: 32727729 DERIVED

Study Officials

• Wonkyung Song

  CHA Bundang Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 18, 2012
• First Posted: June 21, 2012
• Study Start: September 1, 2012
• Primary Completion: April 1, 2015
• Study Completion: April 1, 2015
• Last Updated: April 10, 2024

Record last verified: 2024-02

Locations

KR (1)