NCT06591793
Recruiting
Phase 1
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
Overview
- Phase
- Phase 1
- Intervention
- AAVB-081
- Conditions
- Usher Syndrome, Type 1B
- Sponsor
- AAVantgarde Bio Srl
- Enrollment
- 15
- Locations
- 3
- Primary Endpoint
- To measure the number and severity of treatment related adverse events following treatment with AAVB-081
- Status
- Recruiting
- Last Updated
- last year
Overview
Brief Summary
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Molecular diagnosis of USB1B due to MYO7A mutation
- •Willingness to adhere to protocol per informed consent
Exclusion Criteria
- •Unwillingness to meet the requirements of the study
- •Participation in a clinical study with an Investigation Product in the past 6 months
- •Previous participation in another Gene Therapy trial
- •Any condition that would preclude subretinal surgery
- •Complicating ocular and/or systemic diseases
Arms & Interventions
Cohort 1
AAVB-081 dose level 1
Intervention: AAVB-081
Cohort 2
AAVB-081 dose level 2
Intervention: AAVB-081
Cohort 3
AAVB-081 dose level 3
Intervention: AAVB-081
Outcomes
Primary Outcomes
To measure the number and severity of treatment related adverse events following treatment with AAVB-081
Time Frame: 61 months
Secondary Outcomes
- Change from baseline in microperimetry following treatment with AAVB-081(61 months)
- Change from baseline in static perimetry following treatment with AAVB-081(61 months)
Study Sites (3)
Loading locations...
Similar Trials
Recruiting
Phase 1
Gene Therapy for RPGR Gene Mutation-associated X-linked Retinitis PigmentosaX-Linked Retinitis Pigmentosa (XLRP)NCT06492850Frontera Therapeutics32
Completed
Phase 1
A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112X-Linked Retinitis PigmentosaNCT03116113Biogen50
Completed
Phase 1
A Phase Ia Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Subcutaneously Administered CUG252 in Normal Healthy VolunteersSystemic Lupus ErythematosusNCT05328557Cugene Inc.32
Completed
Phase 1
Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis PigmentosaRetinitis PigmentosaRetinal DiseasesRetinal DegenerationNCT04919473Nanoscope Therapeutics Inc.11
Completed
Phase 2
Safety and Efficacy Study of RT001 to Treat Moderate to Severe Lateral Canthal LinesLateral Canthal LinesCrow's FeetFacial WrinklesNCT00968825Revance Therapeutics, Inc.73