NCT03167203

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

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

• Status: Enrolling By Invitation
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sponsor: Astellas Institute for Regenerative Medicine · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Macular Degenerative Disease

Interventions

Type	Name	Description
BIOLOGICAL	Human 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

Timeline

• Start date: 2018-01-08
• Primary completion: 2029-03-31
• Completion: 2029-03-31
• First posted: 2017-05-25
• Last updated: 2026-04-16

Locations

2 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study