Ignite Creation Date:
2025-12-24 @ 5:51 PM
Ignite Modification Date:
2026-01-02 @ 1:11 PM
Study NCT ID:
NCT02994368
Status:
TERMINATED
Last Update Posted:
2022-01-06
First Post:
2016-12-13
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
"Natural History" Study of Choroideremia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015794', 'term': 'Choroideremia'}, {'id': 'D012162', 'term': 'Retinal Degeneration'}], 'ancestors': [{'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D015862', 'term': 'Choroid Diseases'}, {'id': 'D014603', 'term': 'Uveal Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D012164', 'term': 'Retinal Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D019370', 'term': 'Observation'}], 'ancestors': [{'id': 'D008722', 'term': 'Methods'}, {'id': 'D008919', 'term': 'Investigative Techniques'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 57}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2016-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-01', 'completionDateStruct': {'date': '2021-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2022-01-04', 'studyFirstSubmitDate': '2016-12-13', 'studyFirstSubmitQcDate': '2016-12-14', 'lastUpdatePostDateStruct': {'date': '2022-01-06', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-12-15', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2021-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Rate of progression of disease', 'timeFrame': '4 years', 'description': 'A variety of psychophysical, anatomical and image-based endpoints'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['retinal degeneration', 'eye', 'REP-1 gene'], 'conditions': ['Choroideremia']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to understand the rate of progression of all stages of choroideremia using a variety of assessments performed in the clinic including visual field measures, specialized photography of the eye and participant-reported visual problems'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Individuals with a clinical diagnosis of choroideremia', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria (Primary Cohort):\n\n* Healthy individuals (at least 14 years) with choroideremia (20/200 or better vision) willing to participate in an observational study and meeting the eligibility criteria.\n\nInclusion Criteria (Expansion Cohort):\n\n* Healthy individuals (at least 18 years) with choroideremia (20/200 or better vision in both eyes) willing to participate in an observational study and meeting the eligibility criteria.\n\nExclusion Criteria (Primary and Expansion Cohort):\n\n1. Prior therapy with an AAV vector-based treatment\n2. Pre-existing eye conditions that would: (1) preclude future planned treatment in a therapeutic intent clinical trial (i.e. intravitreal injection), (2) interfere with the interpretation of study endpoints, and/ or put patient at risk for surgical complications\n3. Complicating systemic diseases that would preclude future enrollment in a therapeutic intent clinical trial\n4. Any other condition that would not allow the potential subject to complete follow-up examinations during the course of the study and, in the opinion of the investigator, makes the potential subject unsuitable for the study.'}, 'identificationModule': {'nctId': 'NCT02994368', 'briefTitle': '"Natural History" Study of Choroideremia', 'organization': {'class': 'INDUSTRY', 'fullName': '4D Molecular Therapeutics'}, 'officialTitle': 'A Multicenter Prospective Observational "Natural History" Study in Patients With Choroideremia', 'orgStudyIdInfo': {'id': '4D-CHM-001-NH-0001'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Primary Cohort', 'description': 'No intervention', 'interventionNames': ['Other: Observation']}, {'label': 'Expansion Cohort', 'description': 'No intervention', 'interventionNames': ['Other: Observation']}], 'interventions': [{'name': 'Observation', 'type': 'OTHER', 'armGroupLabels': ['Expansion Cohort', 'Primary Cohort']}]}, 'contactsLocationsModule': {'locations': [{'zip': '75251', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Retina Foundation of the Southwest', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Baylor College of Medicine, Ophthalmology-Cullen Eye Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '84132', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'Moran Eye Center, University of Utah', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'city': 'Edmonton', 'state': 'Alberta', 'country': 'Canada', 'facility': 'University of Alberta', 'geoPoint': {'lat': 53.55014, 'lon': -113.46871}}], 'overallOfficials': [{'name': 'Schonmei Lee, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': '4D Molecular Therapeutics'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': '4D Molecular Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}