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
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 50
Healthy Volunteers: f
View:
• Molecular diagnosis of USB1B due to MYO7A mutation
• Willingness to adhere to protocol per informed consent
Locations
Other Locations
Italy
University of Campania Luigi Vanvitelli
RECRUITING
Naples
United Kingdom
Moorfields Eye Hospital
RECRUITING
London
Retina Clinic London
RECRUITING
London
Contact Information
Primary
Clinical Operations Manager
clinicaltrials@aavantgarde.com
+448000465680
Time Frame
Start Date: 2024-07-02
Estimated Completion Date: 2029-07
Participants
Target number of participants: 15
Treatments
Experimental: Cohort 1
AAVB-081 dose level 1
Experimental: Cohort 2
AAVB-081 dose level 2
Experimental: Cohort 3
AAVB-081 dose level 3
Related Therapeutic Areas
Sponsors
Leads: AAVantgarde Bio Srl