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

Launched by AAVANTGARDE BIO SRL · Sep 9, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called AAVB-081 for patients with Usher Syndrome Type 1B (USH1B), a condition that can cause vision loss due to a genetic mutation. The main goal of the study is to see if a single injection of this treatment, given directly into the eye, is safe and well-tolerated by participants. Researchers will also look at how effective the treatment is in improving vision after it has been administered.

To be eligible for this trial, participants must have a confirmed diagnosis of USH1B linked to a specific genetic mutation in the MYO7A gene. They should be willing to follow the study's guidelines and provide informed consent. However, individuals who have participated in another gene therapy trial recently or have certain eye or health conditions that could interfere with the injection process are not eligible. If someone joins the trial, they can expect to undergo a thorough evaluation and be monitored closely for any side effects or changes in their vision after receiving the treatment.

Gender

ALL

Eligibility 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