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

May 1, 2025 updated by: AAVantgarde Bio Srl

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

15

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Italy
      • Naples, Italy
        • Recruiting
        • University of Campania Luigi Vanvitelli
        • Contact:
          • Francesca Simonelli
  • United Kingdom
      • London, United Kingdom
        • Recruiting
        • Moorfields Eye Hospital
        • Contact:
          • Michel Michaelides
      • London, United Kingdom
        • Recruiting
        • Retina Clinic London
        • Contact:
          • Paulo E Stanga

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1
AAVB-081 dose level 1
Biological: AAVB-081
Single subretinal administration
Experimental: Cohort 2
AAVB-081 dose level 2
Biological: AAVB-081
Single subretinal administration
Experimental: Cohort 3
AAVB-081 dose level 3
Biological: AAVB-081
Single subretinal administration

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
To measure the number and severity of treatment related adverse events following treatment with AAVB-081
Time Frame: 61 months
61 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in microperimetry following treatment with AAVB-081
Time Frame: 61 months
61 months
Change from baseline in static perimetry following treatment with AAVB-081
Time Frame: 61 months
61 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

AAVantgarde Bio Srl

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 2, 2024

Primary Completion (Estimated)

July 1, 2025

Study Completion (Estimated)

July 1, 2029

Study Registration Dates

First Submitted

August 16, 2024

First Submitted That Met QC Criteria

September 9, 2024

First Posted (Actual)

September 19, 2024

Study Record Updates

Last Update Posted (Actual)

May 2, 2025

Last Update Submitted That Met QC Criteria

May 1, 2025

Last Verified

May 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 081-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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