Tests AAVB-081 gene therapy in adults with Usher syndrome type 1B
Tests a single eye injection of AAVB-081 in adults with Usher syndrome type 1B.
Plain-language title generated from the registry record
Registry title: Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
Key facts
- Where
- Naples, ItalySee the site
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 50 years old?
Source: NCT06591793, who can take part
Where in Italy
- University of Campania "Luigi Vanvitelli"Naples, ItalyTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- AAVantgarde Bio Srl
- Trial code
- NCT06591793
- Record updated
- 2 May 2025
- Original description
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.Read full original descriptionShow less
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
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.
Inclusion: Molecular diagnosis of USB1B due to MYO7A mutation; Willingness to adhere to protocol per informed consent.
Exclusion: Previous participation in another Gene Therapy trial; Any condition that would preclude subretinal surgery; Complicating ocular and/or systemic diseases; Unwillingness to meet the requirements of the study; Participation in a clinical study with an Investigation Product in the past 6 months.