Recruiting

AAVB-081 for Usher Syndrome Type IB Retinitis Pigmentosa

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What is being tested

AAVB-081

Biological
Who is being recruted

Congenital Abnormalities+22

+ Abnormalities, Multiple

+ Blindness

From 18 to 50 Years
+7 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 1 & 2
Interventional
Study Start: July 2024
See protocol details

Summary

Principal SponsorAAVantgarde Bio Srl
Study ContactClinical Operations Manager
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: July 2, 2024

Actual date on which the first participant was enrolled.

This study aims to explore a new treatment for individuals with Usher Syndrome Type IB, a genetic condition that leads to progressive vision loss known as retinitis pigmentosa. The study specifically targets those with a mutation in the MYO7A gene. Researchers are investigating the safety and effectiveness of a single subretinal injection of a therapy called AAVB-081. The goal is to find out if this treatment can be tolerated well by patients and if it can show some initial signs of improving or stabilizing vision. Participants in this study will receive the treatment directly into the retina through an injection. The study does not specify the exact outcomes being measured, but it focuses on monitoring the safety and any early signs of effectiveness of the treatment. By participating, patients may help in advancing potential therapies that could eventually lead to improved vision care for those affected by this challenging condition.

Official TitleA 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
NCT06591793
Principal SponsorAAVantgarde Bio Srl
Study ContactClinical Operations Manager
Last updated: January 28, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

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

15 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 50 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Congenital AbnormalitiesAbnormalities, MultipleBlindnessDeafnessEar DiseasesEye DiseasesHearing DisordersHearing Loss, SensorineuralCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNeurologic ManifestationsOtorhinolaryngologic DiseasesRetinal DegenerationRetinal DiseasesRetinitis PigmentosaSensation DisordersSigns and SymptomsPathological Conditions, Signs and SymptomsVision DisordersEye Diseases, HereditaryGenetic Diseases, InbornHearing LossUsher SyndromesDeaf-Blind DisordersRetinal Dystrophies

Criteria

2 inclusion criteria required to participate
Molecular diagnosis of USB1B due to MYO7A mutation
Willingness to adhere to protocol per informed consent
5 exclusion criteria prevent from participating
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
Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

3 intervention groups are designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
AAVB-081 dose level 1

Group II

Experimental
AAVB-081 dose level 2

Group III

Experimental
AAVB-081 dose level 3

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 3 locations

Recruiting

University of Campania Luigi Vanvitelli

Naples, ItalyOpen University of Campania Luigi Vanvitelli in Google Maps
Recruiting

Moorfields Eye Hospital

London, United Kingdom
Recruiting

Retina Clinic London

London, United Kingdom
Recruiting
3 Study Centers