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Enrolling by invitation NCT03167203

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

Phase I / Phase II Interventional Macular Degenerative Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells.
Who it may be relevant to
Registry conditions: Macular Degenerative Disease. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • 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

Primary outcome measures

  • Number of adverse events (AEs) that are ophthalmologic, neurologic, infectious or hematologic [Time frame: Up to 15 Years]
  • Number of new diagnoses of an immune-mediated disorder [Time frame: Up to 15 Years]
  • Incidents of new cancer, irrespective of prior history [Time frame: Up to 15 Years]
  • Incidents of hESC-RPE cell proliferation [Time frame: Up to 15 Years]
  • Incidents of ectopic tissue (RPE or non-RPE) formation [Time frame: Up to 15 Years]
  • Number of participant reported pregnancies or pregnancy of participant's partner [Time frame: Up to 15 Years]
  • Number of reported pregnancy outcome(s) [Time frame: Up to 15 Years]
  • All cause death [Time frame: Up to 15 Years]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • Private Practice — Kansas City
United Kingdom · 1 center
  • Site GB44001 — London

Identifiers

NCT: NCT03167203 · 7316-CL-0007 · 2016-005245-23 · RPE-SSS-001

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗