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Gene therapy injection aims to halt blinding eye disease

NCT ID NCT06467344

Disease control Sponsor: Ascidian Therapeutics, Inc 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early study tests a new gene therapy called ACDN-01 for people with Stargardt disease, a genetic condition that leads to progressive vision loss. The therapy is given as a one-time injection under the retina. The main goals are to check safety and find the right dose, with a first look at whether it might help preserve sight.

What this could mean

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

Active substance
ACDN-01 (a gene therapy given as a one-time injection under the retina)
What this could lead to
If it works, this could slow or stop vision loss in people with Stargardt disease and related retinal conditions.
What could go wrong
This is a very early, first-in-human trial with only 15 participants. It primarily tests safety, not effectiveness. Gene therapies can cause inflammation or other side effects.

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 15 people

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

Started

Jun 2024

Expected to finish

Dec 2030

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

12 years and older

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.

Key Inclusion Criteria: * Presence of mutations in the ABCA4 gene * ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy) * Area of atrophy located in the macula of the study eye * BCVA of 20/50 (0.4 logMAR) or worse Key Exclusion Criteria: * The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy * Retinal disease other than ABCA4-related retinopathy * Presence of a medical condition (systemic or ophthalmic), psychiatric condition, including substance abuse disorder, or physical examination or laboratory finding that may in the opinion of the principal investigator and sponsor preclude adherence to the scheduled study visits, safe participation in the study, or affect the results of the study.

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Conditions

The condition(s) this trial relates to.

ABCA4-related retinopathy cone-rod dystrophy severe early-childhood-onset retinal dystrophy Stargardt disease

As listed by the trial registrant

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

Cone-rod dystrophy Juvenile macular degeneration Stargardt disease Stargardt disease 1

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

    10 sites. 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

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Bascom Palmer Eye Institute

    RECRUITING

    Miami, Florida, 33136, United States

  • Casey Eye Institute OHSU

    RECRUITING

    Portland, Oregon, 97239, United States

  • Cincinnati Eye Institute

    RECRUITING

    Cincinnati, Ohio, 45245, United States

  • Massachusetts Eye and Ear

    RECRUITING

    Boston, Massachusetts, 02114, United States

  • Retina Consultants of Texas

    RECRUITING

    Houston, Texas, 77401, United States

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75382, United States

  • University of Michigan Kellogg Eye Center

    RECRUITING

    Ann Arbor, Michigan, 48105, United States

  • University of San Francisco

    RECRUITING

    San Francisco, California, 94158, United States

  • Vitreo Retinal Associates

    RECRUITING

    Gainesville, Florida, 32607, United States

  • Wilmer Eye Institute at John Hopkins

    RECRUITING

    Baltimore, Maryland, 21218, United States

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