RecruitingPHASE1, PHASE2INTERVENTIONAL

A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)

This study, called 039-101, is looking into a new treatment called AAVB-039 for people who have Stargardt disease. Stargardt disease is an eye condition that affects vision. The main goal is to check how safe AAVB-039 is and if people can tolerate it well after a single injection into the eye. Researchers also want to get an early idea of whether the treatment helps improve or stabilize vision. Participants will need to have Stargardt disease caused by specific changes in a gene called ABCA4. This study is in its early stages (Phase 1/2), meaning it's the first time this treatment is being given to people to carefully monitor its effects.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

AAVantgarde Bio Srl

Enrolment target

Start

29 Sep 2025

Estimated completion

01 Jul 2032

Stargardt Disease

What is this study about?

This study is about a new treatment called AAVB-039 for an eye condition called Stargardt disease. Stargardt disease is an inherited condition that causes vision loss, usually starting in childhood or teenage years. It happens because of changes in a specific gene, often the ABCA4 gene, which affects how cells in the retina (the light-sensitive part at the back of your eye) work.

The main reasons for this study are to find out if AAVB-039 is safe for people and if their bodies can handle it well. It also aims to see if there are any early signs that the treatment is working. This is a very important step because AAVB-039 is a new investigational medicine, meaning it hasn't been approved for use yet. The study will look at how your body reacts to the treatment and if there’s any change in your vision after receiving it.

Researchers are using a special method for this treatment: a single injection directly into the back part of the eye, called the subretinal space. This allows the medicine to get right to the cells that need it most. By doing this, they hope to address the underlying cause of Stargardt disease for people whose condition is linked to specific changes in the ABCA4 gene.

Key takeaways

This study is testing a new gene therapy (AAVB-039) for Stargardt disease.
The main goals are to check its safety and early effectiveness.
It involves a single injection into the eye.
Eligibility requires Stargardt disease from specific ABCA4 gene changes.
Participants must be between 8 and 55 years old.
It's an early-stage (Phase 1/2) clinical trial.

Who may be eligible?

To join this study, you need to have a confirmed diagnosis of Stargardt disease that has been identified by genetic testing as being caused by changes in both copies of your ABCA4 gene. You also need to be willing to follow all the study's instructions and appointments carefully.

However, there are reasons why you might not be able to take part. For example, if you are currently involved in another medical study using a new, unapproved medicine, you won't be able to join this one. The same applies if you’ve previously participated in a study involving gene therapy or cell therapy. Also, certain other eye conditions or general health issues, or anything that would make eye surgery difficult or risky, might mean you can't be part of the study.

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.

Do I have a confirmed diagnosis of Stargardt disease?
Has genetic testing shown I have changes in both copies of my ABCA4 gene?
Am I between 8 and 55 years old?
Am I willing and able to attend all study appointments and follow instructions?
Am I currently not participating in another clinical study with a new, unapproved medicine?
Have I not had previous gene or cell therapy treatments?

Answer every question to see your result.

What does participation involve?

If you decide to participate in this study, you will receive a single injection of the study medicine, AAVB-039, into one of your eyes. This procedure involves minor eye surgery. After the injection, you will have several follow-up visits to the clinic. These visits will involve various eye examinations, vision tests, and possibly blood tests to monitor your health and the effects of the treatment. The research team will explain the full schedule of visits and assessments. The entire duration of your participation, including all follow-up appointments, will be clearly outlined to you before you make a decision to join.

Potential risks and benefits

Participating in a clinical trial like this could potentially offer benefits, such as gaining access to a new treatment before it's widely available and contributing to medical knowledge that might help others with Stargardt disease in the future. However, there are also potential risks, including those associated with eye surgery and the unknown side effects of a new, experimental medicine. It's important to understand that the treatment might not improve your condition, or it could even have negative effects. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (7)

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

Associated Retina Consultants
Verified postcode
Phoenix, United States· Recruiting
Retina Vitreous Associates Medical Group
Verified postcode
Los Angeles, United States· Recruiting
Mayo Clinic
Verified postcode
Rochester, United States· Recruiting
Retina Consultants of Texas
Verified postcode
Bellaire, United States· Recruiting
Retina Foundation of the Southwest
Verified postcode
Dallas, United States· Recruiting
Moorfields Eye Hospital NHS Foundation Trust
Verified postcode
London, United Kingdom· Recruiting
The Retina Clinic
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is Stargardt disease?

Stargardt disease is an inherited eye condition that causes progressive vision loss, usually starting in childhood or adolescence. It affects the macula, the central part of your retina responsible for sharp, detailed vision.

What is AAVB-039?

AAVB-039 is an experimental medicine being tested in this study. It's designed to deliver a working copy of the ABCA4 gene to the cells in the eye, which is hoped to address the cause of Stargardt disease.

How is the medicine given?

The medicine is given as a single injection directly into the back of your eye, in an area called the subretinal space. This is done through a surgical procedure.

What does a 'Phase 1/2' study mean?

Phase 1/2 refers to the early stages of a clinical trial. In these phases, researchers are mainly focused on checking if the new treatment is safe for people and what dose might be best. They also look for any early signs that it could be effective.

Can I join if I'm under 18?

Yes, the study is open to individuals aged 8 to 55 years old, so children and teenagers within this age range may be able to participate if they meet all other criteria.

How to find out more

AAVantgarde Clinical Operations Lead

clinicaltrials@aavantgarde.com +448000465680 Official trial record

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