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Trial record 2 of 26 for:    stargardt's macular dystrophy

Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Astellas Pharma Inc ( Astellas Institute for Regenerative Medicine ) Identifier:
First received: May 13, 2015
Last updated: February 17, 2017
Last verified: February 2017
This is the long term follow-up of a safety and tolerability trial to evaluate the effect of subretinal injection of human embryonic stem cell derived retinal pigment epithelium cells in patients with Stargardt's Macular Dystrophy (SMD).

Condition Intervention
Stargardt's Macular Dystrophy
Biological: MA09-hRPE

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Long Term Follow Up to a Phase I/II, Open-Label, Multi-Center, 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 Astellas Pharma Inc:

Primary Outcome Measures:
  • The safety and tolerance of transplantation of hESC-derived RPE cells MA09-hRPE [ Time Frame: 15 years ]
    • 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 Outcome Measures:
  • Evidence of Successful Engraftment of MA09-hRPE Cells [ Time Frame: 15 years ]
    • Structural evidence (OCT imaging, fluorescein angiography, autofluorescence 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

  • Evidence of Rejection of MA09-hRPE Cells [ Time Frame: 15 years ]
    • Structural (imaging) evidence that implanted MA09-hRPE cells are no longer in the correct location or the presence of vascular leakage

Enrollment: 12
Study Start Date: July 2012
Estimated Study Completion Date: August 2029
Estimated Primary Completion Date: August 2029 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Experimental: MA09-hRPE
Sub-retinal transplantation of MA09-hRPE cells
Biological: MA09-hRPE
Cohort 1 50,000 cells Cohort 2 100,000 cells Cohort 2a Better Vision 100,000 cells Cohort 3 150,000 cells Cohort 4 200,000 cells

Detailed Description:

This study is a long term, non interventional follow up to the phase I/II, open-label, non randomized, sequential, multi-center trial (referred to as the core protocol) in which a maximum of 13 SMD patients were treated. Ten poor vision subjects(20/400) were transplanted with sequential doses of hESC-RPE cells, starting at a dose of 50,000 hESC-RPE cells transplanted (three subjects), 100,000 hESC-RPE cells transplanted (three subjects), 150,000 hESC-RPE cells transplanted (three subjects) and increasing to a maximum dose of 200,000 hESC-RPE cells transplanted(one subject). Three better vision subjects(20/100) were transplanted with a dose of 100,000 hESC-RPE cells.

Eligible patients will be those who sign a consent form for the extension study, and who fulfill all inclusion/exclusion criteria.

The first visit of this extension protocol will correspond to the last visit of the core protocol, and will take place at 12 months post cell implantation.

During the first 2 years after completion of the core protocol, i.e. from Year 1 to Year 2 and from Year 2 to Year 3 post transplantation, the patients will be evaluated at approximately 3 month intervals; during years three thru five patients will be evaluated at approximately six month intervals. During years 6-15, patients will either be required to return to the study center or provide the name of an ophthalmologist in their locality who would provide an evaluation of the patient's vision and any abnormal findings and report these results to the study site annually.

Follow up will include obtaining information about visual acuity, ophthalmological findings and adverse events (AEs), specifically including cancer, neurological, autoimmune or hematological disorders that developed or worsened since the last visit.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The study population is by invitation only for previous participants in the core protocol.

Inclusion Criteria:

  • Must have met the eligibility of the core protocol.
  • Able to understand and willing to sign the informed consent to participate in the follow-up.
  • Treated with hESC-RPE cell transplant in the core protocol.
  • Previous participation in study.

Exclusion Criteria:

  • Unwilling or unable to participate in the study
  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 identifier: NCT02445612

United States, California
Jules Stein Eye Institute, UCLA School of Medicine
Los Angeles, California, United States, 90095
United States, Florida
Bascom Palmer Eye institute
Miami, Florida, United States, 33136
United States, Pennsylvania
Wills Eye Institute-Mid Atlantic Retina
Philadelphia, Pennsylvania, United States, 19107
Sponsors and Collaborators
Astellas Institute for Regenerative Medicine
Study Director: Medical Director Astellas Institute for Regenerative Medicine
  More Information

Responsible Party: Astellas Institute for Regenerative Medicine Identifier: NCT02445612     History of Changes
Other Study ID Numbers: 7316-CL-0004
ACT MA09-hRPE 001(SMD)LTFU ( Other Identifier: Sponsor )
Study First Received: May 13, 2015
Last Updated: February 17, 2017
Individual Participant Data  
Plan to Share IPD: Undecided

Keywords provided by Astellas Pharma Inc:
fundus flavimaculatus
long term follow-up
juvenile macular dystrophy

Additional relevant MeSH terms:
Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases processed this record on April 26, 2017