RECRUITINGPhase 1 / Phase 2INTERVENTIONAL
Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
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
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
- Molecular diagnosis of USB1B due to MYO7A mutation
- Willingness to adhere to protocol per willing to sign a consent form
Who Should NOT Join This Trial:
- 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
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
BIOLOGICAL
AAVB-081
Single subretinal administration
Locations (3)
University of Campania Luigi Vanvitelli
Naples, Italy
Moorfields Eye Hospital
London, United Kingdom
Retina Clinic London
London, United Kingdom