Ignite Creation Date:
2025-12-24 @ 5:05 PM
Ignite Modification Date:
2025-12-27 @ 12:52 PM
Study NCT ID:
NCT06492850
Status:
RECRUITING
Last Update Posted:
2024-07-09
First Post:
2024-07-01
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 32}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-04-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-07', 'completionDateStruct': {'date': '2026-02-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-07-01', 'studyFirstSubmitDate': '2024-07-01', 'studyFirstSubmitQcDate': '2024-07-01', 'lastUpdatePostDateStruct': {'date': '2024-07-09', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-07-09', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-08-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To evaluate the safety and tolerance of FT-002', 'timeFrame': 'from FT-002 administration through up to 1 years', 'description': 'Incidence and severity of AEs'}], 'secondaryOutcomes': [{'measure': 'To evaluate the efficacy of FT-002', 'timeFrame': 'from FT-002 administration through up to 1 years', 'description': 'Changes in visual sensitivity /FST/BCVAfrom baseline'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['X-Linked Retinitis Pigmentosa (XLRP)', 'FT-002'], 'conditions': ['X-Linked Retinitis Pigmentosa (XLRP)']}, 'descriptionModule': {'briefSummary': 'The aim of this study was to evaluate the safety, tolerability, and efficacy of one-time subretinal injection of FT-002 in male subjects (8-45 years of age) with RPGR (Retinitis Pigmentosa GTPase Regulator) gene mutation-associated X-linked retinitis pigmentosa, of XLRP. This study includes Phase I (dose escalation phase) and Phase II (dose expansion phase).'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '45 Years', 'minimumAge': '8 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Subjects that are willing and able to follow study procedures including scheduled visits, treatment plan, and laboratory tests, and sign a written informed consent form;\n* Age: Phase I dose escalation stage, 18-45 years old male (including boundary value) at the time of signing the ICF; Phase II dose extension stage, males 8-45 years old (including boundary values) at the time of signing the ICF;\n* Clinically diagnosed XLRP, the main symptoms include but are not limited to night blindness, visual field loss, vision loss, etc.;\n\nExclusion Criteria:\n\n* Have other retinal degenerative diseases, such as retinal degeneration caused by other known Inherited retinal disease gene variants or previously received an gene therapy product.'}, 'identificationModule': {'nctId': 'NCT06492850', 'briefTitle': 'Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa', 'organization': {'class': 'INDUSTRY', 'fullName': 'Frontera Therapeutics'}, 'officialTitle': 'A Phase I/II Dose-escalation and Dose-expansion Study to Evaluate the Safety and Efficacy of FT-002 Subretinal Injection in Subjects With RPGR Gene Mutation-associated X-linked Retinitis Pigmentosa.', 'orgStudyIdInfo': {'id': 'FT002-C101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'FT-002 dose 1', 'description': 'Low dose FT-002', 'interventionNames': ['Genetic: FT-002']}, {'type': 'EXPERIMENTAL', 'label': 'High dose FT-002', 'description': 'High dose FT-002', 'interventionNames': ['Genetic: FT-002']}], 'interventions': [{'name': 'FT-002', 'type': 'GENETIC', 'description': 'Intraocular injection of a single dose', 'armGroupLabels': ['FT-002 dose 1', 'High dose FT-002']}]}, 'contactsLocationsModule': {'locations': [{'zip': '100142', 'city': 'Beijing', 'state': 'Beijing Municipality', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Ruifang Sui, Professor', 'role': 'CONTACT'}], 'facility': 'Peking Union Medical College Hospital', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}], 'centralContacts': [{'name': 'Xinyan Li', 'role': 'CONTACT', 'email': 'xinyan.li@fronteratherapeutics.com', 'phone': '+86-021-58206061'}, {'name': 'Minghui Xue', 'role': 'CONTACT', 'email': 'minghui.xue@fronteratherapeutics.com', 'phone': '+86-021-58206061'}], 'overallOfficials': [{'name': 'Ruifang Sui', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Peking Union Medical College Hospital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Frontera Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}