Cure My Disease

Don't miss your cure!

 Log in / Sign up

Modified vitamin a pill tested against inherited blindness

NCT ID NCT04239625

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 whether a daily pill called ALK-001 can slow the progression of Stargardt disease, a rare inherited condition that causes vision loss in children and young adults. The trial enrolls people aged 8 and older who have a clinical diagnosis of Stargardt disease and specific genetic mutations. Participants receive either ALK-001 or a placebo for up to 24 months, and researchers track safety, drug levels in the blood, and changes in vision.

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 taken as an oral pill
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 long-term vitamin A-based treatments carry unknown risks.

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 200 people

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

Started

Dec 2019

Expected to finish

Dec 2029

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: * Clinical diagnosis of Stargardt disease (STGD1) * Has at least two ABCA4 disease-causing mutations, unless authorized by sponsor * Has a best-corrected visual acuity (BCVA) greater than approximately 20/160 in at least one eye * 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 been invited to participate in this extension, and has signed and dated the informed consent forms (or assent where appropriate) to participate * Female of childbearing potential has signed the attestation on contraception requirements Simplified Exclusion Criteria: * Is lactating or pregnant * Has a medical condition likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or performance of study procedures * Has abnormal laboratory result(s) at screening * Has an ocular disorder that may confound ocular assessments * Has a history of ocular intervention within 90 days of screening

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Autosomal recessive Stargardt disease 1 (ABCA4-related) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

    Indianapolis, Indiana, 46202, United States

  • Alkeus Site

    Grand Rapids, Michigan, 49456, United States

  • Alkeus Site

    Westbury, New York, 11590, United States

  • Alkeus Site

    Silverdale, Washington, 98383, United States

More trials for these conditions

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