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


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Primary Outcome Measures:

  • 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.

  • 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.

  • 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.

  • 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.

  • 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

  • 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

  • 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

  • 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)

Participants will be contacted by the Patient Outreach Center (POC) on an annual basis to complete a questionnaire about the occurrence of safety events of special interest, 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 safety event of special interest, 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.