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)
|ClinicalTrials.gov Identifier: NCT02941991|
Recruitment Status : Active, not recruiting
First Posted : October 21, 2016
Last Update Posted : December 28, 2017
|Condition or disease||Intervention/treatment|
|Stargardt's Macular Dystrophy||Biological: hESC-RPE|
|Study Type :||Observational|
|Actual Enrollment :||11 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|
|Estimated Primary Completion Date :||December 2019|
|Estimated Study Completion Date :||December 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
- 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.
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|