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

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2012 by CHA Bio & Diostech
Sponsor:
Information provided by (Responsible Party):
CHA Bio & Diostech
ClinicalTrials.gov Identifier:
NCT01625559
First received: June 18, 2012
Last updated: October 22, 2012
Last verified: August 2012
  Purpose

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 Intervention Phase
Stargardt's Macular Dystrophy
Biological: MA09-hRPE
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: 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)

Resource links provided by NLM:


Further study details as provided by CHA Bio & Diostech:

Primary Outcome Measures:
  • safety and tolerance of transplantation [ Time Frame: 18 months ] [ Designated as safety issue: Yes ]

    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:
  • Evidence of successful engraftment [ Time Frame: 18 months ] [ Designated as safety issue: No ]

    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



Estimated Enrollment: 3
Study Start Date: September 2012
Estimated Study Completion Date: October 2014
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 50,000 cells
Biological: MA09-hRPE Cellular therapy
Biological: MA09-hRPE
MA09-hRPE: 50,000 cells

  Eligibility

Ages Eligible for Study:   20 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01625559

Contacts
Contact: Hunhee Kang +82 2 3468 2894 hunheekang@chamc.co.kr

Locations
Korea, Republic of
CHA Bundang Medical Center Recruiting
Seongnam-si, Gyeonggi-do, Korea, Republic of, 463-712
Contact: Wonkyung Song, MD. PhD    82-31-780-5479      
Principal Investigator: Wonkyung Song, MD. PhD         
Sponsors and Collaborators
CHA Bio & Diostech
Investigators
Principal Investigator: Wonkyung Song, MD. PhD. CHA Bundang Medical Center
  More Information

No publications provided

Responsible Party: CHA Bio & Diostech
ClinicalTrials.gov Identifier: NCT01625559     History of Changes
Other Study ID Numbers: CHA_CTP_0903
Study First Received: June 18, 2012
Last Updated: October 22, 2012
Health Authority: Korea: Food and Drug Administration

Additional relevant MeSH terms:
Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases

ClinicalTrials.gov processed this record on September 18, 2014