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Vitamin a derivative eyed to halt inherited blindness

NCT ID NCT02402660

NEW 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 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 21, 2026 · Last updated Sep 21, 2026

Summary

Researchers are testing a daily oral drug called ALK-001 in people aged 8 to 70 with Stargardt disease, an inherited condition that causes progressive vision loss. The trial compares ALK-001 to a placebo over 24 months to see if the drug is safe and can slow damage to the retina. Participants must have a genetic diagnosis of Stargardt disease with two ABCA4 mutations.

What this could mean

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

Active substance
a modified form of vitamin A called ALK-001
What this could lead to
If ALK-001 works, it could become the first treatment to slow vision loss in Stargardt disease, a condition with no approved therapy.
What could go wrong
This is a phase 2 trial, so researchers do not yet know if ALK-001 helps. The drug may not slow vision loss, and daily high doses of a vitamin A derivative could cause 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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 160 people

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

Started

Aug 2015

Expected to finish

Nov 2028

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 70 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.

Simplified Inclusion Criteria: * Male or female between 8 and 70 years old (inclusive), with any visual acuity * Has a clinical diagnosis of typical autosomal recessive Stargardt macular dystrophy (STGD1) * Has provided a genetic report indicating at least two ABCA4 disease-causing mutations. When only one ABCA4 disease-causing mutation is reported, sponsor's permission will be required. * At least one eye (called the "primary study eye") must have at least one well-demarcated area of significantly reduced autofluorescence as imaged by fundus autofluorescence (FAF), have decreased retinal sensitivity as measured by microperimetry, or have maculopathy expected to progress over the duration of the study * Primary study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to permit good quality retinal imaging * Healthy as judged by investigator * Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 24-month study * Has signed and dated the informed consent forms (or assent where appropriate) to participate * Female of childbearing potential has signed the informed consent about birth defects or attestation on contraception requirements Main Exclusion Criteria: * Has taken disallowed items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days * Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization * Has concurrent medical condition or history, which in the opinion of the investigator, is likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or study procedures * Has clinically significant abnormal laboratory result(s) at screening * Has active or historical acute or chronic liver disorder * Has active or historical ocular disorder in the primary study eye that, in the opinion of the investigator, may confound assessment of the retina morphologically or functionally (this could include for example cataract surgery within the past 6 months, choroidal neovascularization (CNV), glaucoma, recurring uveitis, diabetic retinopathy, other retinal disease, etc.) * Has had intraocular surgery or injections in the primary study eye within 90 days of the screening visit * Has a clinically significant abnormal electrocardiogram (ECG), or has a corrected QT interval (QTc) that is 450 ms or greater

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Conditions

The condition(s) this trial relates to.

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.

Autosomal recessive Stargardt disease 1 (ABCA4-related) Stargardt disease Stargardt macular degeneration Stargardt macular dystrophy

Contacts and locations

Locations

  • Alkeus Site

    Phoenix, Arizona, 85020, United States

  • Alkeus Site

    Los Angeles, California, 90095, United States

  • Alkeus Site

    Aurora, Colorado, 80045, United States

  • Alkeus Site

    Gainesville, Florida, 32601, United States

  • Alkeus Site

    Miami, Florida, 33136, United States

  • Alkeus Site

    Indianapolis, Indiana, 46202, United States

  • Alkeus Site

    Baltimore, Maryland, 21287, United States

  • Alkeus Site

    Grand Rapids, Michigan, 49546, United States

  • Alkeus Site

    New York, New York, 10032, United States

  • Alkeus Site

    Westbury, New York, 11590, United States

  • Alkeus Site

    Houston, Texas, 77025, United States

  • Alkeus Site

    Salt Lake City, Utah, 84132, United States

  • Alkeus Site

    Silverdale, Washington, 98383, United States

  • Alkeus Site

    Milwaukee, Wisconsin, 53226, United States

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