Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa

Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-50
SponsorAAVantgarde Bio Srl

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.

Eligibility criteria

Qualifiers

Molecular diagnosis of USB1B due to MYO7A mutation

Willingness to adhere to protocol per informed consent

Disqualifiers

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

Trial design

Treatments tested in this trial

  • AAVB-081

Treatment groups

15 Participants
are divided into 3 treatment groups

Sponsors and collaborators