RecruitingPHASE1, PHASE2INTERVENTIONAL

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

This research study is looking into a new treatment called AAVB-081 for a rare inherited condition called Usher Syndrome type 1B (USH1B). This condition can cause both hearing and vision loss, specifically a type of progressive sight loss known as retinitis pigmentosa. The study focuses on people aged 18 to 50 whose Usher Syndrome type 1B is caused by a change in a gene known as MYO7A. The main goals are to find out if a single injection of AAVB-081, given into the back of the eye (subretinal), is safe and well-tolerated by participants. Researchers will also be looking closely to see if the treatment shows any early signs of helping with vision. This is a crucial early-stage study (Phase 1/2) to understand the potential of this new genetic therapy.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

AAVantgarde Bio Srl

Enrolment target

Start

02 Jul 2024

Estimated completion

01 Jul 2029

Usher Syndrome, Type 1B

What is this study about?

This clinical trial is designed to investigate a new treatment, known as AAVB-081, for individuals living with Usher Syndrome type 1B (USH1B). Usher Syndrome is a genetic condition that typically affects both hearing and vision. Specifically, this study focuses on the vision problems, which are often a progressive loss of sight called retinitis pigmentosa. This type of Usher Syndrome is linked to a specific change in the MYO7A gene.

The main purpose of this study is to carefully assess the safety and how well people tolerate a single dose of AAVB-081. This treatment is delivered directly into the back of the eye using a special injection technique. Additionally, the researchers will be watching for any early signs to see if the treatment is effective in improving or stabilising vision.

This is an early-stage study, meaning it's one of the first times this treatment is being tested in people. The information gathered from this trial will be vital for understanding if AAVB-081 could be a promising future treatment option for people with Usher Syndrome type 1B and retinitis pigmentosa.

Key takeaways

This is an early-stage study for Usher Syndrome type 1B (USH1B) related vision loss.
The treatment, AAVB-081, is a gene therapy delivered by eye injection.
The study aims to check safety, tolerability, and early signs of vision improvement.
Participants must have USH1B due to a MYO7A gene mutation and be aged 18-50.
Close monitoring will be in place after the treatment.

Who may be eligible?

To be considered for this study, you must be between 18 and 50 years old. You need to have a confirmed diagnosis of Usher Syndrome type 1B that is specifically caused by a mutation in the MYO7A gene. It's also important that you are willing and able to follow all the study's instructions and attend all required appointments.

There are certain reasons why you might not be able to join. For example, if you've taken part in another clinical trial involving an investigational medicine in the last six months, or if you've ever had a gene therapy trial before, you wouldn't be eligible. Also, if you have any eye conditions or other health issues that would make eye surgery difficult or risky, or if you simply don't wish to meet the study's requirements, you wouldn't be able to participate.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you between 18 and 50 years old?
Do you have a confirmed diagnosis of Usher Syndrome type 1B due to a MYO7A gene change?
Are you willing and able to follow all study instructions and attend appointments?
Have you *not* participated in another gene therapy trial before?
Do you have no other eye or health conditions that would make eye surgery difficult or risky?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive the AAVB-081 treatment through a single injection into the back of one of your eyes. This is a specialised surgical procedure. Following the injection, you would have a series of regular visits to the clinic so the research team can closely monitor your health, check how your eye is healing, and assess your vision.

These visits will involve various eye tests and general health checks. The exact number and frequency of visits, as well as the total duration of your participation, would be explained in detail by the study team. This ongoing monitoring is crucial to understand the safety and potential effects of the treatment over time.

Potential risks and benefits

Taking part in this study offers the potential benefit of receiving a new, experimental treatment that might help with your vision loss related to Usher Syndrome type 1B. However, as with any new treatment and surgical procedure, there are potential risks, including discomfort, infection, or other complications related to the eye injection. The study team will explain all known and potential risks in detail. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (3)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

University of Campania Luigi Vanvitelli
Verified postcode
Naples, Italy· Recruiting
Moorfields Eye Hospital
Verified postcode
London, United Kingdom· Recruiting
Retina Clinic London
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is Usher Syndrome type 1B?

It's a rare genetic condition that affects both hearing and vision. The vision loss is a progressive condition called retinitis pigmentosa, specific to a MYO7A gene change in this type.

What is AAVB-081?

AAVB-081 is an experimental gene therapy designed to address the underlying genetic cause of vision loss in Usher Syndrome type 1B.

How is the treatment given?

The treatment is given as a single injection directly into the back of one of your eyes, which is a specialised surgical procedure.

What are the main goals of the study?

The study aims to understand if AAVB-081 is safe and well-tolerated, and to look for early signs that it might help improve or stabilise vision.

Can I leave the study if I change my mind?

Yes, you are free to withdraw from the study at any time, for any reason, and your decision will not affect your medical care.

How to find out more

Clinical Operations Manager

clinicaltrials@aavantgarde.com +448000465680 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.