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

Astellas

Status and phase: Invitation-only; Phase 2; Phase 1

Conditions: Macular Degenerative Disease

Treatments: Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells

Study type: Interventional

Funder types: Industry

Identifiers

NCT03167203

2016-005245-23 (EudraCT Number)

RPE-SSS-001 (Other Identifier)

7316-CL-0007

Details and patient eligibility

About

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.

Full 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.

Enrollment: 36 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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36 participants in 1 patient group

hESC-RPE cells

Experimental group

Description:

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

Treatment:

Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells