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
Sex
ALL
Min age
18 Years
Max age
50 Years
Healthy volunteers
No
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
Primary outcome measure(s)
To measure the number and severity of treatment related adverse events following treatment with AAVB-081 — 61 months
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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