Ignite Creation Date:
2025-12-26 @ 5:17 PM
Ignite Modification Date:
2026-01-01 @ 5:25 AM
Study NCT ID:
NCT01543906
Status:
COMPLETED
Last Update Posted:
2014-12-15
First Post:
2012-02-17
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D012174', 'term': 'Retinitis Pigmentosa'}], 'ancestors': [{'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D058499', 'term': 'Retinal Dystrophies'}, {'id': 'D012162', 'term': 'Retinal Degeneration'}, {'id': 'D012164', 'term': 'Retinal Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C009166', 'term': 'retinol acetate'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2012-02'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2014-12', 'completionDateStruct': {'date': '2014-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-12-11', 'studyFirstSubmitDate': '2012-02-17', 'studyFirstSubmitQcDate': '2012-03-01', 'lastUpdatePostDateStruct': {'date': '2014-12-15', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2012-03-05', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2014-07', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Visual field', 'timeFrame': '12 months'}], 'secondaryOutcomes': [{'measure': "Safety will be assessed by evaluating the following: adverse events, clinical laboratory results, ECG's and vital signs", 'timeFrame': '12 months'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ['Retinitis Pigmentosa (RP)']}, 'referencesModule': {'references': [{'pmid': '32573764', 'type': 'DERIVED', 'citation': 'Schwartz SG, Wang X, Chavis P, Kuriyan AE, Abariga SA. Vitamin A and fish oils for preventing the progression of retinitis pigmentosa. Cochrane Database Syst Rev. 2020 Jun 18;6(6):CD008428. doi: 10.1002/14651858.CD008428.pub3.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is:\n\n* To evaluate whether 7-day treatment with oral QLT091001 can improve visual function in RP subjects with an autosomal dominant mutation in RPE65.\n* To evaluate duration of visual function improvement (if observed) in RP subjects with an autosomal dominant mutation in RPE65 after 7-day treatment with oral QLT091001.\n* To evaluate the safety of oral QLT091001 administered once daily for 7 days in RP subjects with an autosomal dominant mutation in RPE65.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subjects will have RP caused by an autosomal dominant mutation in RPE65, as diagnosed by an ocular geneticist or ophthalmologist.\n* Subjects who have a best-corrected standard ETDRS visual acuity of 3 letters or better (20/800 Snellen equivalent) or visible photoreceptor outer segments on OCT/FAF.\n\nExclusion Criteria:\n\n* Subjects with any clinically important abnormal physical finding at Screening.\n* Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane/Roaccutane® or Soriatane/Neotigason®) within 6 months of Day 0 and subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure.\n* Subjects with a history of diabetes or chronic hyperlipidemia, hepatitis, pancreatitis, or cirrhosis.\n* Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60 days of Screening.'}, 'identificationModule': {'nctId': 'NCT01543906', 'briefTitle': 'Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)', 'organization': {'class': 'INDUSTRY', 'fullName': 'QLT Inc.'}, 'officialTitle': 'An Open-Label, Phase 1b, Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)', 'orgStudyIdInfo': {'id': 'RET RP 01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'QLT091001', 'description': 'oral QLT091001 administered once daily for 7 days', 'interventionNames': ['Drug: QLT091001']}], 'interventions': [{'name': 'QLT091001', 'type': 'DRUG', 'description': 'oral QLT091001 administered once daily for 7 days', 'armGroupLabels': ['QLT091001']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': "Montreal Children's Hospital, McGill University Health Centre", 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'city': 'Dublin', 'country': 'Ireland', 'facility': 'Royal Victoria Eye and Ear Hospital', 'geoPoint': {'lat': 53.33306, 'lon': -6.24889}}], 'overallOfficials': [{'name': 'Sushanta Mallick', 'role': 'STUDY_DIRECTOR', 'affiliation': 'QLT Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'QLT Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}