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)
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)
3 other identifiers
observational
12
1 country
2
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Jan 2013
Longer than P75 for all trials
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
January 16, 2013
CompletedFirst Submitted
Initial submission to the registry
October 20, 2016
CompletedFirst Posted
Study publicly available on registry
October 21, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 2, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
October 2, 2019
CompletedDecember 10, 2020
December 1, 2020
6.7 years
October 20, 2016
December 9, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Safety assessed by Adverse Events (AEs) of special interest in regards to the investigational product
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
Secondary Outcomes (2)
Incidence of graft failure or rejection
4 years
Number of patients with changes in ocular examinations or images
4 years
Study Arms (1)
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
Interventions
sub-retinal injection
Eligibility Criteria
The study population is by invitation only from previous participants in the core protocol.
You may qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Moorefields Eye Hospital NHS Foundation Trust
London, EC1V2PD, United Kingdom
Newcastle on Tyne NHS Foundation Trust
Newcastle upon Tyne, NE7 7DN, United Kingdom
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Astellas Institute for Regenerative Medicine
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 20, 2016
First Posted
October 21, 2016
Study Start
January 16, 2013
Primary Completion
October 2, 2019
Study Completion
October 2, 2019
Last Updated
December 10, 2020
Record last verified: 2020-12