ALK-001 to slow progression of Stargardt disease

A Phase 3 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, and Efficacy of Gildeuretinol Acetate (ALK 001) on the Progression of Stargardt Disease (STGD)

This trial will test whether ALK-001 can slow vision loss in people aged 8 to 45 with ABCA4-related Stargardt disease.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 230 (estimated)
• Ages: 8 Years to 45 Years
• Sex: All
• Sponsor: Alkeus Pharmaceuticals, Inc. (Industry-sponsored)
• Locations: 3 sites (Phoenix, Arizona and 2 other locations)
• Trial ID: NCT07419334 on ClinicalTrials.gov

What this trial studies

This Phase 3 interventional trial enrolls participants aged 8–45 with clinical signs of autosomal recessive Stargardt disease and a genetic report confirming disease-causing ABCA4 mutations. Participants will receive either ALK-001 or a placebo and will be followed for safety and changes in vision and retinal findings over the treatment period. The protocol requires contraception for people of childbearing potential and excludes recent use of vitamin A, beta-carotene, liver-based products, or oral retinoids. Study visits are conducted at several U.S. retina centers.

Who should consider this trial

Good fit: Ideal candidates are people aged 8 to 45 with a clinical diagnosis of autosomal recessive Stargardt disease and a genetic report confirming ABCA4 disease-causing mutations who can comply with contraception and medication restrictions.

Not a fit: People who are pregnant or lactating, recently took disallowed vitamin A–containing or retinoid products, or who lack a confirmed ABCA4 mutation are unlikely to be eligible or to benefit from this trial.

Why it matters

Potential benefit: If successful, ALK-001 could slow progression of vision loss and help preserve central vision in people with Stargardt disease.

How similar studies have performed: Modifying vitamin A chemistry to reduce toxic byproducts has been explored in earlier-phase studies with some promising signals, but definitive large Phase 3 evidence is still limited.

Eligibility criteria

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.

Where this trial is running

Phoenix, Arizona and 2 other locations

• Associated Retina Consultants — Phoenix, Arizona, United States (Recruiting)
• Vitreo Retinal Associates — Gainesville, Florida, United States (Not_yet_recruiting)
• Erie Retina Research — Erie, Pennsylvania, United States (Recruiting)

Study contacts

• Study coordinator: For trial questions: [e-mail] or 877-255-7476

Conditions Stargardt Disease; Stargardt Macular Degeneration; Stargardt Macular Dystrophy; Autosomal Recessive Stargardt Disease 1