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)
|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: NCT02941991|
Recruitment Status : Completed
First Posted : October 21, 2016
Last Update Posted : December 10, 2020
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|Condition or disease||Intervention/treatment|
|Stargardt's Macular Dystrophy||Biological: hESC-RPE|
|Study Type :||Observational|
|Actual Enrollment :||12 participants|
|Official Title:||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)|
|Actual Study Start Date :||January 16, 2013|
|Actual Primary Completion Date :||October 2, 2019|
|Actual Study Completion Date :||October 2, 2019|
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
Other Name: ASP7316
- 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.
- 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).
- 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.
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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|
- 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.
- There are no exclusion criteria
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): NCT02941991
|Moorefields Eye Hospital NHS Foundation Trust|
|London, United Kingdom, EC1V2PD|
|Newcastle on Tyne NHS Foundation Trust|
|Newcastle upon Tyne, United Kingdom, NE7 7DN|
|Study Director:||Medical Director||Astellas Institute for Regenerative Medicine|
|Responsible Party:||Astellas Institute for Regenerative Medicine|
|Other Study ID Numbers:||
2012-002827-14 ( EudraCT Number )
ACT-hESC-RPE-SMD-01FU EU ( Other Identifier: Sponsor )
|First Posted:||October 21, 2016 Key Record Dates|
|Last Update Posted:||December 10, 2020|
|Last Verified:||December 2020|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Undecided|
Stargardt's Macular Dystrophy
juvenile macular dystrophy
Eye Diseases, Hereditary
Genetic Diseases, Inborn