A Safety Surveillance Study in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy
- Conditions
- Macular Degenerative Disease
- Interventions
- Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells
- Registration Number
- NCT03167203
- Lead Sponsor
- Astellas Institute for Regenerative Medicine
- Brief Summary
The purpose of this study is to is to evaluate the occurrence of late onset (i.e., greater than 5 years after treatment) adverse events of special interest (AESI) in participants who have received sub-retinal transplant of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cells in an AIRM-sponsored clinical trial. The events of special interest are adverse events (AEs) that are presumed to have a potential causal relationship to the hESC-RPE cells.
- Detailed Description
Participants will be contacted by the Patient Outreach Center (POC) on an annual basis to complete a questionnaire about the occurrence of AESI, as defined in the Outcome Measure section.
The questionnaire will be completed directly by the participant (via a secure web based platform) or will be administered via telephone by a POC staff member. In the event of the occurrence of a AESI, study participant will be encouraged to contact the POC as soon as possible, rather than wait for the annual questionnaire completion. Follow up data is inclusive of the 1 year in the core trial, 4 years in the long term follow up trial and 10 years in this long term safety follow up. Participation for United Kingdom participants will be life-long.
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- All
- Target Recruitment
- 36
- Participant was treated with hESC-RPE cell therapy in an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial in macular degenerative disease.
- Participant is able to understand.
- There are no exclusion criteria.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description hESC-RPE cells Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells Participants previously enrolled into an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial and treated with Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial (hESC-RPE) cells
- Primary Outcome Measures
Name Time Method Incidents of hESC-RPE cell proliferation Up to 15 Years Occurrence of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cell proliferation will be participant self-reported via an annual questionnaire.
Number of adverse events (AEs) that are ophthalmologic, neurologic, infectious or hematologic Up to 15 Years Adverse events (AEs) will be coded using Medical Dictionary for Regulatory Activities (MedDRA). Participant self-reported via an annual questionnaire of adverse events that are ophthalmologic, neurologic, infectious or hematologic.
Number of new diagnoses of an immune-mediated disorder Up to 15 Years Any new diagnosis of an immune-mediated disorder will be participant self-reported via an annual questionnaire.
Incidents of new cancer, irrespective of prior history Up to 15 Years Any new cancer, irrespective of prior history, will be participant self-reported via an annual questionnaire.
Number of reported pregnancy outcome(s) Up to 15 Years Occurrence of pregnancy outcomes will be participant self-reported via an annual questionnaire
Incidents of ectopic tissue (RPE or non-RPE) formation Up to 15 Years Occurrence of ectopic tissue (retinal pigment epithelial \[RPE\] or non-RPE) will be participant self-reported via an annual questionnaire
Number of participant reported pregnancies or pregnancy of participant's partner Up to 15 Years Occurrence of pregnancy will be participant self-reported via an annual questionnaire
All cause death Up to 15 Years All causes of death will be collected via an annual questionnaire (through participant-designated secondary contacts)
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (2)
Site GB44001
🇬🇧London, United Kingdom
Private Practice
🇺🇸Kansas City, Missouri, United States