Ignite Creation Date:
2025-12-25 @ 1:06 AM
Ignite Modification Date:
2025-12-27 @ 11:28 AM
Study NCT ID:
NCT06591793
Status:
RECRUITING
Last Update Posted:
2025-05-02
First Post:
2024-08-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C536485', 'term': 'Usher syndrome, type 1B'}, {'id': 'D052245', 'term': 'Usher Syndromes'}, {'id': 'D012174', 'term': 'Retinitis Pigmentosa'}], 'ancestors': [{'id': 'D054062', 'term': 'Deaf-Blind Disorders'}, {'id': 'D003638', 'term': 'Deafness'}, {'id': 'D034381', 'term': 'Hearing Loss'}, {'id': 'D006311', 'term': 'Hearing Disorders'}, {'id': 'D004427', 'term': 'Ear Diseases'}, {'id': 'D010038', 'term': 'Otorhinolaryngologic Diseases'}, {'id': 'D006319', 'term': 'Hearing Loss, Sensorineural'}, {'id': 'D012678', 'term': 'Sensation Disorders'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D001766', 'term': 'Blindness'}, {'id': 'D014786', 'term': 'Vision Disorders'}, {'id': 'D058499', 'term': 'Retinal Dystrophies'}, {'id': 'D012162', 'term': 'Retinal Degeneration'}, {'id': 'D012164', 'term': 'Retinal Diseases'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D000015', 'term': 'Abnormalities, Multiple'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D015785', 'term': 'Eye Diseases, Hereditary'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 15}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-07-02', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2029-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-05-01', 'studyFirstSubmitDate': '2024-08-16', 'studyFirstSubmitQcDate': '2024-09-09', 'lastUpdatePostDateStruct': {'date': '2025-05-02', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-09-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To measure the number and severity of treatment related adverse events following treatment with AAVB-081', 'timeFrame': '61 months'}], 'secondaryOutcomes': [{'measure': 'Change from baseline in microperimetry following treatment with AAVB-081', 'timeFrame': '61 months'}, {'measure': 'Change from baseline in static perimetry following treatment with AAVB-081', 'timeFrame': '61 months'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Usher Syndrome', 'MYO7A', 'USH1B', 'Retinitis Pigmentosa'], 'conditions': ['Usher Syndrome, Type 1B']}, 'descriptionModule': {'briefSummary': 'The purpose of the 081-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-081 in USH1B patients with retinitis pigmentosa due to a mutation in the MYO7A gene. The study will also assess the initial efficacy following AAVB-081 administration.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '50 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Molecular diagnosis of USB1B due to MYO7A mutation\n* Willingness to adhere to protocol per informed consent\n\nExclusion Criteria:\n\n* Unwillingness to meet the requirements of the study\n* Participation in a clinical study with an Investigation Product in the past 6 months\n* Previous participation in another Gene Therapy trial\n* Any condition that would preclude subretinal surgery\n* Complicating ocular and/or systemic diseases'}, 'identificationModule': {'nctId': 'NCT06591793', 'briefTitle': 'Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa', 'organization': {'class': 'INDUSTRY', 'fullName': 'AAVantgarde Bio Srl'}, 'officialTitle': 'A Phase 1/2 Multicenter, Open-label, Dose Escalation, Safety and Efficacy Study of Subretinal Administration of Dual AAV8.MYO7A, AAVB-081 in Subjects With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa', 'orgStudyIdInfo': {'id': '081-101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1', 'description': 'AAVB-081 dose level 1', 'interventionNames': ['Biological: AAVB-081']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': 'AAVB-081 dose level 2', 'interventionNames': ['Biological: AAVB-081']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 3', 'description': 'AAVB-081 dose level 3', 'interventionNames': ['Biological: AAVB-081']}], 'interventions': [{'name': 'AAVB-081', 'type': 'BIOLOGICAL', 'description': 'Single subretinal administration', 'armGroupLabels': ['Cohort 1', 'Cohort 2', 'Cohort 3']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Naples', 'status': 'RECRUITING', 'country': 'Italy', 'contacts': [{'name': 'Francesca Simonelli', 'role': 'CONTACT'}], 'facility': 'University of Campania Luigi Vanvitelli', 'geoPoint': {'lat': 40.85216, 'lon': 14.26811}}, {'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Michel Michaelides', 'role': 'CONTACT'}], 'facility': 'Moorfields Eye Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'London', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'name': 'Paulo E Stanga', 'role': 'CONTACT'}], 'facility': 'Retina Clinic London', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'centralContacts': [{'name': 'Clinical Operations Manager', 'role': 'CONTACT', 'email': 'clinicaltrials@aavantgarde.com', 'phone': '+448000465680'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'AAVantgarde Bio Srl', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}