Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
- Registration Number
- NCT06591793
- Lead Sponsor
- AAVantgarde Bio Srl
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 15
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Cohort 2 AAVB-081 AAVB-081 dose level 2 Cohort 3 AAVB-081 AAVB-081 dose level 3 Cohort 1 AAVB-081 AAVB-081 dose level 1
- Primary Outcome Measures
Name Time Method To measure the number and severity of treatment related adverse events following treatment with AAVB-081 61 months
- Secondary Outcome Measures
Name Time Method 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
Trial Locations
- Locations (3)
University of Campania Luigi Vanvitelli
🇮🇹Naples, Italy
Moorfields Eye Hospital
🇬🇧London, United Kingdom
Retina Clinic London
🇬🇧London, United Kingdom