A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
December 9, 2020 updated by: Astellas Institute for Regenerative Medicine
Follow-up to 5 Years of 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 (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)
The purpose of this study is to evaluate the long term safety and tolerability of hESC-RPE cellular therapy in patients with advanced SMD from 1 to 5 years following the surgical procedure to implant the hESC-RPE cells.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
This study is a long-term, follow- up of a Phase I/II, open-label, non-randomized, 4-cohort, dose escalation, multi-center clinical trial (referred to as the core trial or core protocol) in which a maximum of 12 SMD patients were transplanted with sequential doses of hESC-RPE cells, starting at a dose of 50,000 hESC-RPE cells transplanted and increasing to a maximum dose of 200,000 hESC-RPE cells transplanted.
All patients who participate in the core protocol are eligible for participation in the follow-up protocol.
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.
Informed consent for this follow-up protocol will be obtained at the first visit which will occur at the 12-month visit of the core protocol.
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 Outcome.
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 life-long annual health survey, under a separate protocol, to further monitor long-term safety.
Study Type
Observational
Enrollment (Actual)
12
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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-
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London, United Kingdom, EC1V2PD
- Moorefields Eye Hospital NHS Foundation Trust
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Newcastle upon Tyne, United Kingdom, NE7 7DN
- Newcastle on Tyne NHS Foundation Trust
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
The study population is by invitation only from previous participants in the core protocol.
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Cohort
- Time Perspectives: Prospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
|---|---|
|
uniocular subretinal injection of hESC-RPE cells
Cohort 1. 50,000 cells transplanted; Cohort 2. 100,000 cells transplanted; Cohort 3. 150,000 cells transplanted; Cohort 4. 200,000 cells transplanted
|
sub-retinal injection
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
|
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 Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, any Adverse Event (AE) that causes the subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer (irrespective of prior history), ectopic or proliferative cell growth (Retinal pigment epithelium (RPE) or non-RPE) with adverse clinical consequence, unexpected, clinically significant AE possibly related to the cell transplant procedure or the investigational product (hESC-RPE cells), Pregnancy in a female subject or the partner of a male subject and pregnancy outcome.
|
4 years
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
|---|---|---|
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Incidence of graft failure or rejection
Time Frame: 4 years
|
Evidence of graft failure or rejection may consist of: Presence of retinal edema, cystoid macular edema, retinal white dots, retinal hemorrhage, serous retinal detachment, sub-retinal exudates, sub-retinal fibrosis, ocular vascular and/or optic disc leakage, and 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).
|
4 years
|
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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.
|
4 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
January 16, 2013
Primary Completion (Actual)
October 2, 2019
Study Completion (Actual)
October 2, 2019
Study Registration Dates
First Submitted
October 20, 2016
First Submitted That Met QC Criteria
October 20, 2016
First Posted (Estimate)
October 21, 2016
Study Record Updates
Last Update Posted (Actual)
December 10, 2020
Last Update Submitted That Met QC Criteria
December 9, 2020
Last Verified
December 1, 2020
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 7316-CL-0006
- 2012-002827-14 (EudraCT Number)
- ACT-hESC-RPE-SMD-01FU EU (Other Identifier: Sponsor)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Undecided
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
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