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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
36
Participants who have received sub-retinal transplant of hESC-RPE cells in an AIRM sponsored clinical trial
Private Practice
Kansas City, Missouri, United States
Site GB44001
London, United Kingdom
Number of adverse events (AEs) that are ophthalmologic, neurologic, infectious or hematologic
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.
Time frame: Up to 15 Years
Number of new diagnoses of an immune-mediated disorder
Any new diagnosis of an immune-mediated disorder will be participant self-reported via an annual questionnaire.
Time frame: Up to 15 Years
Incidents of new cancer, irrespective of prior history
Any new cancer, irrespective of prior history, will be participant self-reported via an annual questionnaire.
Time frame: Up to 15 Years
Incidents of hESC-RPE cell proliferation
Occurrence of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cell proliferation will be participant self-reported via an annual questionnaire.
Time frame: Up to 15 Years
Incidents of ectopic tissue (RPE or non-RPE) formation
Occurrence of ectopic tissue (retinal pigment epithelial \[RPE\] or non-RPE) will be participant self-reported via an annual questionnaire
Time frame: Up to 15 Years
Number of participant reported pregnancies or pregnancy of participant's partner
Occurrence of pregnancy will be participant self-reported via an annual questionnaire
Time frame: Up to 15 Years
Number of reported pregnancy outcome(s)
Occurrence of pregnancy outcomes will be participant self-reported via an annual questionnaire
Time frame: Up to 15 Years
All cause death
All causes of death will be collected via an annual questionnaire (through participant-designated secondary contacts)
Time frame: Up to 15 Years
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