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

NCT ID: NCT06591793

Last Updated: 2025-05-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-07-02

Study Completion Date

2029-07-31

Brief Summary

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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.

Detailed Description

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Conditions

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Usher Syndrome, Type 1B

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1

AAVB-081 dose level 1

Group Type EXPERIMENTAL

AAVB-081

Intervention Type BIOLOGICAL

Single subretinal administration

Cohort 2

AAVB-081 dose level 2

Group Type EXPERIMENTAL

AAVB-081

Intervention Type BIOLOGICAL

Single subretinal administration

Cohort 3

AAVB-081 dose level 3

Group Type EXPERIMENTAL

AAVB-081

Intervention Type BIOLOGICAL

Single subretinal administration

Interventions

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AAVB-081

Single subretinal administration

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

50 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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AAVantgarde Bio Srl

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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University of Campania Luigi Vanvitelli

Naples, , Italy

Site Status RECRUITING

Moorfields Eye Hospital

London, , United Kingdom

Site Status RECRUITING

Retina Clinic London

London, , United Kingdom

Site Status RECRUITING

Countries

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Italy United Kingdom

Central Contacts

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Clinical Operations Manager

Role: CONTACT

+448000465680

Facility Contacts

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Francesca Simonelli

Role: primary

Michel Michaelides

Role: primary

Paulo E Stanga

Role: primary

Other Identifiers

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081-101

Identifier Type: -

Identifier Source: org_study_id

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