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Gene therapy injection aims to save sight in rare eye disease

NCT ID NCT07161544

Disease control Sponsor: AAVantgarde Bio Srl Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jul 31, 2026 · Updated 3 times

Summary

This study tests a gene therapy called AAVB-039 for Stargardt disease, an inherited condition that causes vision loss. About 75 adults with a specific gene mutation will receive a single injection under the retina. The main goal is to check safety, but researchers will also measure whether it slows retinal damage and preserves vision.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
AAVB-039 (a gene therapy given as a single injection under the retina)
What this could lead to
If it works, this could slow or stop vision loss in people with Stargardt disease, potentially preserving sight for years.
What could go wrong
This is an early (Phase 1/2) trial with only 75 participants, so results may not apply to everyone. Gene therapy under the retina carries risks like inflammation or retinal damage.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 75 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2025

Expected to finish

Jul 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

8 to 55 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Molecular diagnosis of Stargardt disease due to ABCA4 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 another Investigation Medicinal Product * Previous participation in another gene or cell therapy trial * Any condition that would preclude subretinal surgery * Complicating ocular and/or systemic diseases

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Conditions

The condition(s) this trial relates to.

Stargardt disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Stargardt disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    9 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Associated Retina Consultants

    RECRUITING

    Phoenix, Arizona, 85020, United States

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • Moorfields Eye Hospital NHS Foundation Trust

    RECRUITING

    London, England, EC1V 2PD, United Kingdom

  • Retina Consultants of Texas

    RECRUITING

    Bellaire, Texas, 77401, United States

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75231, United States

  • Retina Vitreous Associates Medical Group

    RECRUITING

    Los Angeles, California, 90211, United States

  • The Retina Clinic

    RECRUITING

    London, England, W1G 9AY, United Kingdom

    Contact Email: •••••@•••••

  • University of Florida Health Science Center

    RECRUITING

    Jacksonville, Florida, 32209, United States

  • University of Utah

    RECRUITING

    Salt Lake City, Utah, 84112, United States

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