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

April 8, 2024 updated by: CHABiotech CO., Ltd

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)

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

3

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Korea, Republic of
    • Gyeonggi-do
      • Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712
        • CHA Bundang Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

20 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: MA09-hRPE administration
Biological: MA09-hRPE Cellular therapy
Biological: MA09-hRPE
MA09-hRPE cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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:

  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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
18 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

CHABiotech CO., Ltd

Investigators

  • Principal Investigator: Wonkyung Song, CHA Bundang Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2012

Primary Completion (Actual)

April 1, 2015

Study Completion (Actual)

April 1, 2015

Study Registration Dates

First Submitted

June 18, 2012

First Submitted That Met QC Criteria

June 19, 2012

First Posted (Estimated)

June 21, 2012

Study Record Updates

Last Update Posted (Actual)

April 10, 2024

Last Update Submitted That Met QC Criteria

April 8, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CHA_CTP_0903

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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