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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01625559
Recruitment Status : Unknown
Verified August 2012 by CHABiotech CO., Ltd.
Recruitment status was:  Active, not recruiting
First Posted : June 21, 2012
Last Update Posted : February 18, 2015
Information provided by (Responsible Party):
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 Group
  • When-MA09-hRPE cell transplantation to evaluate the safety of surgical procedures.
  • In future studies intended to assess the number of transplanted hRPE cells.
  • In the past, MA09-hRPE cell therapy used in the study was to evaluate the validity of the potential.
  • Homologous retinal pigment epithelial cells derived from embryonic stem cells, future studies of drugs that are used in representing the potential validity to evaluate the optimal dose.

Condition or disease Intervention/treatment Phase
Stargardt's Macular Dystrophy Biological: MA09-hRPE Phase 1

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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)
Study Start Date : September 2012
Estimated Primary Completion Date : March 2015
Estimated Study Completion Date : June 2015

Arm Intervention/treatment
Experimental: 50,000 cells
Biological: MA09-hRPE Cellular therapy
Biological: MA09-hRPE
MA09-hRPE: 50,000 cells

Primary Outcome Measures :
  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:

    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 :
  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

Information from the National Library of Medicine

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Ages Eligible for Study:   20 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01625559

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Korea, Republic of
CHA Bundang Medical Center
Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712
Sponsors and Collaborators
CHABiotech CO., Ltd
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Principal Investigator: Wonkyung Song, MD. PhD. CHA Bundang Medical Center

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Responsible Party: CHABiotech CO., Ltd Identifier: NCT01625559     History of Changes
Other Study ID Numbers: CHA_CTP_0903
First Posted: June 21, 2012    Key Record Dates
Last Update Posted: February 18, 2015
Last Verified: August 2012
Additional relevant MeSH terms:
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Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases