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

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.

Study Overview

• Status: Enrolling by invitation
• Conditions: Macular Degenerative Disease
• Intervention / Treatment: Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial 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.

• Study Type: Interventional
• Enrollment (Estimated): 36
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United Kingdom
  • London, United Kingdom
    • Site GB44001
• United States
  • Missouri
    • Kansas City, Missouri, United States, 64133
      • Private Practice

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: hESC-RPE cells; 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; Participants who have received sub-retinal transplant of hESC-RPE cells in an AIRM sponsored clinical trial; Other Names: ASP7316

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Astellas Institute for Regenerative Medicine
• Investigators: Study Director: Medical Monitor, Astellas Institute for Regenerative Medicine

Study record dates

Study Major Dates

• Study Start (Actual): January 8, 2018
• Primary Completion (Estimated): March 31, 2029
• Study Completion (Estimated): March 31, 2029

Study Registration Dates

• First Submitted: May 24, 2017
• First Submitted That Met QC Criteria: May 24, 2017
• First Posted (Actual): May 25, 2017

Study Record Updates

• Last Update Posted (Actual): May 15, 2026
• Last Update Submitted That Met QC Criteria: May 14, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: hESC-RPE; Macular Degenerative Disease
• Other Study ID Numbers: 7316-CL-0007; 2016-005245-23 (EudraCT Number); RPE-SSS-001 (Other Identifier: Sponsor)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as products terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.
• IPD Sharing 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.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No