Macular Degenerative Disease
Conditions
Keywords
hESC-RPE, Macular Degenerative Disease
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.
Interventions
BIOLOGICALHuman Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells
Participants who have received sub-retinal transplant of hESC-RPE cells in an AIRM sponsored clinical trial
Sponsors
Astellas Institute for Regenerative Medicine
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* There are no
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
| 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 |
| Number of reported pregnancy outcome(s) | Up to 15 Years | Occurrence of pregnancy outcomes 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) |
Countries
United Kingdom, United States
Contacts
STUDY_DIRECTORMedical Monitor
Astellas Institute for Regenerative Medicine
Outcome results
None listed