RecruitingPhase 1Phase 2NCT06591793

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


Sponsor

AAVantgarde Bio Srl

Enrollment

15 participants

Start Date

Jul 2, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

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

Min Age: 18 YearsMax Age: 50 Years

Inclusion Criteria2

  • Molecular diagnosis of USB1B due to MYO7A mutation
  • Willingness to adhere to protocol per informed consent

Exclusion Criteria5

  • 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

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Interventions

BIOLOGICALAAVB-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

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NCT06591793