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Enrolling by Invitation

NCT Number: NCT03167203

A Safety Surveillance Study 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.

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Key information

Conditions

Macular Degenerative Disease

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Site GB44001, London, United Kingdom

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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

Other names: ASP7316

Primary outcomes

  1. Number of adverse events (AEs) that are ophthalmologic, neurologic, infectious or hematologic

    Time frame: Up to 15 Years

    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.

  2. Number of new diagnoses of an immune-mediated disorder

    Time frame: Up to 15 Years

    Any new diagnosis of an immune-mediated disorder will be participant self-reported via an annual questionnaire.

  3. Incidents of new cancer, irrespective of prior history

    Time frame: Up to 15 Years

    Any new cancer, irrespective of prior history, will be participant self-reported via an annual questionnaire.

  4. Incidents of hESC-RPE cell proliferation

    Time frame: Up to 15 Years

    Occurrence of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cell proliferation will be participant self-reported via an annual questionnaire.

  5. Incidents of ectopic tissue (RPE or non-RPE) formation

    Time frame: Up to 15 Years

    Occurrence of ectopic tissue (retinal pigment epithelial [RPE] or non-RPE) will be participant self-reported via an annual questionnaire

  6. Number of participant reported pregnancies or pregnancy of participant's partner

    Time frame: Up to 15 Years

    Occurrence of pregnancy will be participant self-reported via an annual questionnaire

  7. Number of reported pregnancy outcome(s)

    Time frame: Up to 15 Years

    Occurrence of pregnancy outcomes will be participant self-reported via an annual questionnaire

  8. All cause death

    Time frame: Up to 15 Years

    All causes of death will be collected via an annual questionnaire (through participant-designated secondary contacts)

Sponsors and collaborators

Lead sponsor

Astellas Institute for Regenerative Medicine

Industry

Registry information

Official study 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

Important dates

Study start
2018
Primary completion
2029
Study completion
2029
First posted
May 25, 2017
Registry last updated
Jul 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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