Clinical trial NCT03167203: A Safety Surveillance Study in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy
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
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE1
PHASE2
Conditions: Macular Degenerative Disease
Keywords: hESC-RPE
Macular Degenerative Disease
Lead sponsor: Astellas Pharma Inc
Sponsor class: INDUSTRY
First posted: 2017-05-25
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

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.

Primary outcomes:
measure: Number of adverse events (AEs) that are ophthalmologic, neurologic, infectious or hematologic
timeFrame: Up to 15 Years
measure: Number of new diagnoses of an immune-mediated disorder
timeFrame: Up to 15 Years
measure: Incidents of new cancer, irrespective of prior history
timeFrame: Up to 15 Years
measure: Incidents of hESC-RPE cell proliferation
timeFrame: Up to 15 Years
measure: Incidents of ectopic tissue (RPE or non-RPE) formation
timeFrame: Up to 15 Years
measure: Number of participant reported pregnancies or pregnancy of participant's partner
timeFrame: Up to 15 Years
measure: Number of reported pregnancy outcome(s)
timeFrame: Up to 15 Years
measure: All cause death
timeFrame: Up to 15 Years
Secondary outcomes:
Other outcomes:
Locations:
city: Kansas City
country: United States
facility: Private Practice
state: Missouri
city: London
country: United Kingdom
facility: Site GB44001
Study officials:
affiliation: Astellas Institute for Regenerative Medicine
name: Medical Monitor
role: STUDY_DIRECTOR
References: