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

NCT03167203 | Enrolling By Invitation Phase 1 FDA Drug

• 3 other identifiers: 7316-CL-00072016-005245-23RPE-SSS-001
• Study Type: interventional
• Target: 36
• Locations: 2 countries
• Sites: 2

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.

Conditions

Macular Degenerative Disease

Keywords

hESC-RPE; Macular Degenerative Disease

Outcome Measures

Primary Outcomes (8)

• 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

Study Arms (1)

hESC-RPE cells

EXPERIMENTAL

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

Interventions

Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells BIOLOGICAL

Participants who have received sub-retinal transplant of hESC-RPE cells in an AIRM sponsored clinical trial

Also known as: ASP7316

hESC-RPE cells

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

Sponsors & Collaborators

• Astellas Institute for Regenerative Medicine: lead

Study Sites (2)

Private Practice

Kansas City, Missouri, 64133, United States

Location

Site GB44001

London, United Kingdom

Location

Study Officials

• Medical Monitor

  Astellas Institute for Regenerative Medicine

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 24, 2017
• First Posted: May 25, 2017
• Study Start: January 8, 2018
• Primary Completion (Estimated): March 31, 2029
• Study Completion (Estimated): March 31, 2029
• Last Updated: April 16, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data.
• Access Criteria: Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement.

Locations

US (1); GB (1)