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

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.
ClinicalTrials.gov Identifier: NCT02445612
Recruitment Status : Completed
First Posted : May 15, 2015
Last Update Posted : November 13, 2020
Information provided by (Responsible Party):
Astellas Pharma Inc ( Astellas Institute for Regenerative Medicine )

Brief Summary:
The purpose of this study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced Stargardt's Macular Dystrophy (SMD) from one to five years following the surgical procedure to implant the MA09-hRPE cells.

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

Detailed Description:

This study is a long term, follow up to the ACT MA09-hRPE 001 phase I/II trial, the phase I/II trial (referred to as the core protocol) was an open-label, non-randomized, dose escalation, multi-center trial. Thirteen SMD patients were treated in this trial. Ten patients with profound vision loss (visual acuity <=20/400) received a single subretinal injection of MA09-hRPE cells, starting at a dose of 50,000 MA09-hRPE cells (three patients), 100,000 MA09-hRPE cells transplanted (three patients), 150,000 MA09-hRPE cells transplanted (three patients) and increasing to a maximum dose of 200,000 MA09-hRPE cells transplanted (one patient). Three patients with severe to moderate vision loss (visual acuity <= 20/100) received a dose of 100,000 MA09-hRPE cells. All patients who participated in the core protocol are eligible for participation in this follow-up protocol.

The first visit of this long term follow-up protocol will correspond to the last visit of the core protocol, and will take place at 12 months post cell implantation. Informed consent will be obtained at this visit.

Patients will be evaluated at 18, 24, 36, 48 and 60 months post-transplant (or more frequently as clinically indicated). Follow-up will include obtaining information about ophthalmological findings and events of special interest as defined in the Primary Outcomes. At the last visit of this follow-up study, whether at 60 months post-transplant or at early discontinuation, patients will be invited to participate in a Safety Surveillance Study for an additional 10 years, under a separate protocol, which will continue to monitor the long term risks of MA09-hRPE cell transplantation.

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Study Type : Observational
Actual Enrollment : 13 participants
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)
Actual Study Start Date : July 11, 2012
Actual Primary Completion Date : June 21, 2019
Actual Study Completion Date : June 21, 2019

Resource links provided by the National Library of Medicine

Group/Cohort Intervention/treatment
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
Other Name: ASP7316

Primary Outcome Measures :
  1. Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product [ Time Frame: 4 years ]
    This will include obtaining information about ophthalmological findings and Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, pregnancy in a female subject or the partner of a male subject and pregnancy outcome, any adverse event (AE) that causes a subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer, ectopic or proliferative cell growth (Retinal pigment epithelium (RPE) or non-RPE) with adverse clinical consequence, and unexpected, clinically significant AEs possibly related to the cell transplant procedure or the investigational product (MA09-hRPE cells).

Secondary Outcome Measures :
  1. Incidence of graft failure or rejection [ Time Frame: 4 years ]
    Evidence of graft failure or rejection will consist of: Presence of retinal edema, cystoid macular edema, retinal white dots, retinal hemorrhage, serous retinal detachment, subretinal exudates, subretinal fibrosis, or vascular and/or optic disc leakage, elevated intraocular pressure or hypotony. Evidence of unanticipated and persistent or increasing non-infectious ocular inflammation (e.g., vasculitis, retinitis, choroiditis, vitritis, pars planitis, anterior segment inflammation/uveitis).

  2. Number of patients with changes in ocular examinations or images [ Time Frame: 4 years ]
    The number of patients with clinically significant absolute values or changes from baseline in Intra-ocular pressure (IOP) and Best Corrected Visual Acuity (BCVA) will be summarized.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
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 been treated with hESC-RPE cell transplant in the core protocol.
  • Able to understand and willing to sign the informed consent to participate in the follow-up study.

Exclusion Criteria:

  • There are no exclusion criteria

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 ClinicalTrials.gov identifier (NCT number): NCT02445612

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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
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Study Director: Medical Director Astellas Institute for Regenerative Medicine
Additional Information:
Publications of Results:
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Responsible Party: Astellas Institute for Regenerative Medicine
ClinicalTrials.gov Identifier: NCT02445612    
Other Study ID Numbers: 7316-CL-0004
ACT MA09-hRPE 001(SMD)LTFU ( Other Identifier: Sponsor )
First Posted: May 15, 2015    Key Record Dates
Last Update Posted: November 13, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Access to anonymized individual participant level data will not be provided for this trial as it meets one or more of the exceptions described on www.clinicalstudydatarequest.com under "Sponsor Specific Details for Astellas."
Keywords provided by Astellas Pharma Inc ( Astellas Institute for Regenerative Medicine ):
fundus flavimaculatus
long term follow-up
juvenile macular dystrophy
Additional relevant MeSH terms:
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Macular Degeneration
Stargardt Disease
Retinal Degeneration
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn