Hope for stargardt patients: new drug aims to slow blindness

NCT ID NCT07419334

Recruiting now Disease control Sponsor: Alkeus Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 29, 2026 · Updated 3 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.

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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

Contacts and locations

Locations

Associated Retina Consultants
RECRUITING

Phoenix, Arizona, 85020, United States
Erie Retina Research
RECRUITING

Erie, Pennsylvania, 16505, United States
Retina Foundation of the Southwest
RECRUITING

Dallas, Texas, 75231, United States
Star Retina
RECRUITING

Fort Worth, Texas, 76132, United States
Vitreo Retinal Associates
RECRUITING

Gainesville, Florida, 32607, United States

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