Ignite Creation Date:
2025-12-24 @ 7:38 PM
Ignite Modification Date:
2026-02-24 @ 8:50 AM
Study NCT ID:
NCT03167203
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-10-20
First Post:
2017-05-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Safety Surveillance Study in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 36}}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2018-01-08', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-10', 'completionDateStruct': {'date': '2029-03-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-10-17', 'studyFirstSubmitDate': '2017-05-24', 'studyFirstSubmitQcDate': '2017-05-24', 'lastUpdatePostDateStruct': {'date': '2025-10-20', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2017-05-25', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-03-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of adverse events (AEs) that are ophthalmologic, neurologic, infectious or hematologic', 'timeFrame': 'Up to 15 Years', 'description': '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.'}, {'measure': 'Number of new diagnoses of an immune-mediated disorder', 'timeFrame': 'Up to 15 Years', 'description': 'Any new diagnosis of an immune-mediated disorder will be participant self-reported via an annual questionnaire.'}, {'measure': 'Incidents of new cancer, irrespective of prior history', 'timeFrame': 'Up to 15 Years', 'description': 'Any new cancer, irrespective of prior history, will be participant self-reported via an annual questionnaire.'}, {'measure': 'Incidents of hESC-RPE cell proliferation', 'timeFrame': 'Up to 15 Years', 'description': 'Occurrence of human embryonic stem cell derived - retinal pigment epithelial (hESC-RPE) cell proliferation will be participant self-reported via an annual questionnaire.'}, {'measure': 'Incidents of ectopic tissue (RPE or non-RPE) formation', 'timeFrame': 'Up to 15 Years', 'description': 'Occurrence of ectopic tissue (retinal pigment epithelial \\[RPE\\] or non-RPE) will be participant self-reported via an annual questionnaire'}, {'measure': "Number of participant reported pregnancies or pregnancy of participant's partner", 'timeFrame': 'Up to 15 Years', 'description': 'Occurrence of pregnancy will be participant self-reported via an annual questionnaire'}, {'measure': 'Number of reported pregnancy outcome(s)', 'timeFrame': 'Up to 15 Years', 'description': 'Occurrence of pregnancy outcomes will be participant self-reported via an annual questionnaire'}, {'measure': 'All cause death', 'timeFrame': 'Up to 15 Years', 'description': 'All causes of death will be collected via an annual questionnaire (through participant-designated secondary contacts)'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['hESC-RPE', 'Macular Degenerative Disease'], 'conditions': ['Macular Degenerative Disease']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.\n\nThe 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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participant was treated with hESC-RPE cell therapy in an Astellas Institute for Regenerative Medicine (AIRM) sponsored clinical trial in macular degenerative disease.\n* Participant is able to understand.\n\nExclusion Criteria:\n\n* There are no exclusion criteria.'}, 'identificationModule': {'nctId': 'NCT03167203', 'briefTitle': 'A Safety Surveillance Study in Subjects With Macular Degenerative Disease Treated With Human Embryonic Stem Cell-derived Retinal Pigment Epithelial Cell Therapy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Astellas Pharma Inc'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': '7316-CL-0007'}, 'secondaryIdInfos': [{'id': '2016-005245-23', 'type': 'EUDRACT_NUMBER'}, {'id': 'RPE-SSS-001', 'type': 'OTHER', 'domain': 'Sponsor'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'hESC-RPE cells', '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', 'interventionNames': ['Biological: Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells']}], 'interventions': [{'name': 'Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial Cells', 'type': 'BIOLOGICAL', 'otherNames': ['ASP7316'], 'description': 'Participants who have received sub-retinal transplant of hESC-RPE cells in an AIRM sponsored clinical trial', 'armGroupLabels': ['hESC-RPE cells']}]}, 'contactsLocationsModule': {'locations': [{'zip': '64133', 'city': 'Kansas City', 'state': 'Missouri', 'country': 'United States', 'facility': 'Private Practice', 'geoPoint': {'lat': 39.09973, 'lon': -94.57857}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'Site GB44001', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Medical Monitor', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Astellas Institute for Regenerative Medicine'}]}, 'ipdSharingStatementModule': {'url': 'https://www.clinicaltrials.astellas.com/transparency/', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'CSR'], 'timeFrame': 'Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data.', 'ipdSharing': 'YES', 'description': "Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as products terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.", 'accessCriteria': 'Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement.'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Astellas Institute for Regenerative Medicine', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}