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Completed

NCT Number: NCT01625559

Safety and Tolerability of 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

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Key information

Conditions

Stargardt's Macular Dystrophy

Age range

20 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

CHA Bundang Medical Center

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

About this study

  • 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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

MA09-hRPE

Biological

MA09-hRPE cells

Primary outcomes

  1. 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 outcomes

  1. 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

Sponsors and collaborators

Lead sponsor

CHABiotech CO., Ltd

Industry

Registry information

Official study title

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)

Important dates

Study start
2012
Primary completion
2015
Study completion
2015
First posted
Jun 21, 2012
Registry last updated
Apr 10, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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