Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Hope for stargardt patients: new drug aims to slow blindness

NCT ID NCT07419334

Disease control Sponsor: Alkeus Pharmaceuticals, 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Sep 18, 2026 · Updated 6 times

Summary

This Phase 3 trial tests an oral drug called gildeuretinol acetate (ALK 001) in 230 people aged 8 to 45 with Stargardt disease, a genetic condition that causes progressive vision loss. Participants take the drug or a placebo daily for 24 months. The goal is to see if the drug can slow the growth of retinal damage and preserve eyesight.

What this could mean

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

Active substance
gildeuretinol acetate (ALK 001)
What this could lead to
If it works, this could slow the progression of Stargardt disease and help preserve vision longer.
What could go wrong
This is still an experimental drug. Even in Phase 3, it may not prove effective, and side effects are possible. Results may not apply to all patients.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 230 people

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

Started

Jun 2026

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

8 to 45 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 1. Male or female, 8 to 45 years of age (inclusive) on the day of screening. 2. Female participants of childbearing potential, as well as fertile male participants with female partners of childbearing potential, must be willing to comply with the protocol-defined methods of contraception from the time of consent until 90 days and 30 days, respectively, after the last dose of IMP. 3. Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy 4. Have provided a genetic report by any regional accredited organization that provides certified testing indicating the presence of disease-causing mutation. 5. Have signed and dated the informed consent forms (ICFs; or assent as appropriate) to participate. Exclusion Criteria 1. Has taken disallowed items (supplements containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of randomization. 2. Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization, or is planning to become pregnant during the course of study. A male participant who wishes to father a child during the course of the study. 3. Has ever participated in any gene therapy, cell therapy, or device study to treat STGD, unless documented confirmation of participation in the placebo arm with no surgery is provided. Has participated in a drug study to treat STGD within the past 6 months. 4. Has participated in any drug study to treat any other condition within 5 half-lives of the investigational drug prior to screening, unless documented confirmation of participation in the placebo arm is provided. Has participated in an investigational device study within 30 days prior to screening or longer if, in the Investigator's judgment, the device could affect study outcomes. 5. Anticipates participating in any other drug or device study within the duration of the study.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Stargardt disease are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

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

    14 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

Locations

  • Associated Retina Consultants

    RECRUITING

    Phoenix, Arizona, 85020, United States

  • Erie Retina Research

    RECRUITING

    Erie, Pennsylvania, 16505, United States

  • Foundation for Vision Research

    RECRUITING

    Grand Rapids, Michigan, 49546, United States

  • Medical Center Ophthalmology Associates

    RECRUITING

    San Antonio, Texas, 78240, United States

  • Mid Atlantic Retina

    NOT_YET_RECRUITING

    Philadelphia, Pennsylvania, 19073, United States

    Contact Email: •••••@•••••

  • Ohio State Havener Eye Institute

    RECRUITING

    Columbus, Ohio, 43212, United States

  • Retina Foundation of the Southwest

    RECRUITING

    Dallas, Texas, 75231, United States

  • Star Retina

    RECRUITING

    Fort Worth, Texas, 76132, United States

  • The Retina Group of Washington

    RECRUITING

    Fairfax, Virginia, 22031, United States

  • Tufts Medical Center - New England Eye Center

    NOT_YET_RECRUITING

    Boston, Massachusetts, 02116, United States

  • UPMC Vision Institute

    RECRUITING

    Pittsburgh, Pennsylvania, 15219, United States

    Contact Email: •••••@•••••

  • University of Colorado SOM, Sue-Anschutz-Rodgers Eye Center

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Vitreo Retinal Associates

    RECRUITING

    Gainesville, Florida, 32607, United States

  • Wagner Kapoor Research Institute

    RECRUITING

    Norfolk, Virginia, 23666, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.