A Safety Surveillance Study in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Safety Surveillance Study of Events of Special Interest Occurring in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy

Conditions
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Macular Degenerative Disease

Intervention / Treatment
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* Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells


Participation Criteria
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Eligibility Criteria
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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 exclusion criteria.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: hESC-RPE cells<br>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 | Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells<br>* Participants who have received sub-retinal transplant of hESC-RPE cells in an AIRM sponsored clinical trial<br>* Other names: ASP7316;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | 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. | 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 | 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 | Up to 15 Years | 
| Number of reported pregnancy outcome(s) | Occurrence of pregnancy outcomes will be participant self-reported via an annual questionnaire | Up to 15 Years | 
| All cause death | All causes of death will be collected via an annual questionnaire (through participant-designated secondary contacts) | Up to 15 Years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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hESC-RPE, Macular Degenerative Disease