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NCT Number: NCT06591793

Study of Subretinally Injected AAVB-081 in Patients 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.

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Key information

Conditions

Usher Syndrome, Type 1B Abnormalities, Multiple Blindness Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Deaf-Blind Disorders Deafness Ear Diseases Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Hearing Disorders Hearing Loss Hearing Loss, Sensorineural Nervous System Diseases Neurologic Manifestations Otorhinolaryngologic Diseases Pathological Conditions, Signs and Symptoms Retinal Degeneration Retinal Diseases Retinal Dystrophies Retinitis Pigmentosa Sensation Disorders Signs and Symptoms Usher Syndromes Vision Disorders

Age range

18 year–50 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

University of Campania Luigi Vanvitelli, Naples, Italy

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

AAVB-081

Biological

Single subretinal administration

Primary outcomes

  1. To measure the number and severity of treatment related adverse events following treatment with AAVB-081

    Time frame: 61 months

Secondary outcomes

  1. Change from baseline in microperimetry following treatment with AAVB-081

    Time frame: 61 months

  2. Change from baseline in static perimetry following treatment with AAVB-081

    Time frame: 61 months

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Operations Manager

CONTACT

[email protected]

+448000465680

Sponsors and collaborators

Lead sponsor

AAVantgarde Bio Srl

Industry

Registry information

Official study 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

Important dates

Study start
2024
Primary completion
2025
Study completion
2029
First posted
Sep 19, 2024
Registry last updated
May 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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