Injecting AAVB-081 to treat Usher Syndrome Type IB
Official title: 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
Run by AAVantgarde Bio Srl · NCT06591793 · Data updated 2026-11-12
This study is testing a new injection for people with Usher Syndrome Type IB to see if it is safe and helps improve their vision.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 50 Years |
| Sex | All |
| Enrollment | 15 (estimated) |
| Sponsor | AAVantgarde Bio Srl Industry-sponsored |
| Locations | 3 sites (Naples and 2 other locations) |
| Status | Recruiting |
| Started | July 2, 2024 |
| Main results expected | July 2025 |
| Study ends | July 2029 |
| Registry record updated | May 2, 2025 |
| Trial ID | NCT06591793 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are individuals diagnosed with Usher Syndrome Type IB due to a MYO7A mutation.
✕Probably not a fit
Patients with complicating ocular or systemic diseases or those who have recently participated in other gene therapy trials may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This study evaluates the safety and tolerability of a single subretinal injection of AAVB-081 in patients with Usher Syndrome Type IB, specifically those with retinitis pigmentosa caused by a MYO7A gene mutation. The study aims to assess the initial efficacy of the treatment following administration. Participants will be monitored for any adverse effects and the overall response to the therapy.
Why it matters
Potential benefit: If successful, this treatment could improve vision and quality of life for patients with Usher Syndrome Type IB.
How similar studies have gone: Other studies involving gene therapy for retinal diseases have shown promising results, indicating potential for success in this approach.
Who can join: the full rules
Show full inclusion / exclusion 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
Where it's running
-
University of Campania Luigi Vanvitelli
Naples, Italy Recruiting -
Moorfields Eye Hospital
London, United Kingdom Recruiting -
Retina Clinic London
London, United Kingdom Recruiting
Study contacts
- Study coordinator: Clinical Operations Manager
- Email: clinicaltrials@aavantgarde.com
- Phone: +448000465680
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
-
AAVB-039 gene therapy for Stargardt disease
This study will test a single subretinal injection of AAVB-039 gene therapy in people with Stargardt disease caused by two ABCA4 mutations to see if it is safe and shows early benefit.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.