- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02463344
Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Patients With AMD
November 10, 2020 updated by: Astellas Institute for Regenerative Medicine
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 Advanced Dry AMD
The purpose of his study is to evaluate the long term safety and tolerability of MA09-hRPE cellular therapy in patients with advanced dry Age-Related Macular Degeneration (AMD) from one to five years following the surgical procedure to implant the MA09-hRPE cells.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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 AMD 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 transplanted (three patients), 100,000 MA09-hRPE cells transplanted (three patients), 150,000 MA09-hRPE cells (three patients) and increasing to a maximum dose of 200,000 MA09-hRPE cells transplanted (one patient).
Three patients with severe to moderate 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 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 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.
Study Type
Observational
Enrollment (Actual)
11
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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California
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Los Angeles, California, United States, 90095
- Jules Stein Eye Institute, UCLA School of Medicine
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Florida
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Miami, Florida, United States, 33136
- Bascom Palmer Eye Institute
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Massachusetts
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Boston, Massachusetts, United States, 02114-3002
- Mass Eye and Ear
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19107
- Wills Eye Institute-Mid Atlantic Retina
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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
53 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 for 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 |
---|---|
MA09-hRPE
Experimental: Subretinal injection of 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 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 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).
|
4 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of patients with changes in ocular examinations or images
Time Frame: 4 years
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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.
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4 years
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Incidence of graft failure or rejection
Time Frame: 4 years
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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).
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4 years
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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.
General Publications
- Schwartz SD, Hubschman JP, Heilwell G, Franco-Cardenas V, Pan CK, Ostrick RM, Mickunas E, Gay R, Klimanskaya I, Lanza R. Embryonic stem cell trials for macular degeneration: a preliminary report. Lancet. 2012 Feb 25;379(9817):713-20. doi: 10.1016/S0140-6736(12)60028-2. Epub 2012 Jan 24.
- Schwartz SD, Regillo CD, Lam BL, Eliott D, Rosenfeld PJ, Gregori NZ, Hubschman JP, Davis JL, Heilwell G, Spirn M, Maguire J, Gay R, Bateman J, Ostrick RM, Morris D, Vincent M, Anglade E, Del Priore LV, Lanza R. Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies. Lancet. 2015 Feb 7;385(9967):509-16. doi: 10.1016/S0140-6736(14)61376-3. Epub 2014 Oct 15.
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)
February 25, 2013
Primary Completion (Actual)
August 19, 2019
Study Completion (Actual)
August 19, 2019
Study Registration Dates
First Submitted
May 13, 2015
First Submitted That Met QC Criteria
June 2, 2015
First Posted (Estimate)
June 4, 2015
Study Record Updates
Last Update Posted (Actual)
November 13, 2020
Last Update Submitted That Met QC Criteria
November 10, 2020
Last Verified
November 1, 2020
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 7316-CL-0005
- ACT MA09-hRPE AMD -001 LTFU (Other Identifier: Sponsor)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
No
IPD 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."
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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