Menu
Recruiting NCT06591793

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

Phase I / Phase II Interventional Usher Syndrome, Type 1B

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: AAVB-081.
Who it may be relevant to
Registry conditions: Usher Syndrome, Type 1B. Basic parameters: 18 years — 50 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Italy, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Biological AAVB-081
    Single subretinal administration

Primary outcome measures

  • To measure the number and severity of treatment related adverse events following treatment with AAVB-081 [Time frame: 61 months]
Secondary outcome measures (2)
  • Change from baseline in microperimetry following treatment with AAVB-081 [Time frame: 61 months]
  • Change from baseline in static perimetry following treatment with AAVB-081 [Time frame: 61 months]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United Kingdom · 2 centers
  • Moorfields Eye Hospital — London
  • Retina Clinic London — London
Italy · 1 center
  • University of Campania Luigi Vanvitelli — Naples

Identifiers

NCT: NCT06591793 · 081-101

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗