Injecting AAVB-081 to treat Usher Syndrome Type IB
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
PHASE1; PHASE2 · AAVantgarde Bio Srl · NCT06591793
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 |
| Enrollment | 15 (estimated) |
| Ages | 18 Years to 50 Years |
| Sex | All |
| Sponsor | AAVantgarde Bio Srl (industry) |
| Locations | 3 sites (Naples and 2 other locations) |
| Trial ID | NCT06591793 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are individuals diagnosed with Usher Syndrome Type IB due to a MYO7A mutation.
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.
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 performed: Other studies involving gene therapy for retinal diseases have shown promising results, indicating potential for success in this approach.
Eligibility criteria
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 this trial is running
Naples and 2 other locations
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Usher Syndrome, Type 1B, Usher Syndrome, MYO7A, USH1B, Retinitis Pigmentosa