ALK-001 to slow progression of Stargardt disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Associated Retina Consultants
  Phoenix, Arizona, United States Recruiting
• University of Colorado SOM, Sue-Anschutz-Rodgers Eye Center
  Aurora, Colorado, United States Recruiting
• Vitreo Retinal Associates
  Gainesville, Florida, United States Recruiting
• Foundation for Vision Research
  Grand Rapids, Michigan, United States Recruiting
• Ohio State Havener Eye Institute
  Columbus, Ohio, United States Recruiting
• Erie Retina Research
  Erie, Pennsylvania, United States Recruiting

Study contacts

• Study coordinator: For trial questions: trials@alkeuspharma.com or 877-255-7476
• Email: trials@alkeuspharma.com
• Phone: 877-255-7476

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• ASP2020 eye injection safety in Stargardt-type macular dystrophy

  A small study will test whether a single ASP2020 eye injection is safe and may help people with Stargardt-type macular dystrophy.

  Recruiting at 4 sites Phase 1 Astellas Pharma Inc · Phoenix, Arizona and 3 other locations · NCT07734064

• C.001 for retinal degeneration: a first-in-human dose-escalation safety test

  A first-in-human study will test the safety and tolerability of a single subretinal injection of C.001 in adults with retinal diseases involving retinal pigment epithelium degeneration.

  Recruiting at 2 sites Phase 1 Cellio Therapeutics Inc · Beverly Hills, California and 2 other locations · NCT07594236

• Functional visual field and navigation testing for moderate-to-profound inherited retinal disease

  This project will test whether functional visual field and navigation tests can measure real-world vision and mobility in people with moderate-to-profound inherited retinal diseases.

  Recruiting Ray Therapeutics, Inc. · Irvine, California · NCT07502664

• Single intravitreal RTx-021 injection for Stargardt disease

  This will test whether a single injection of RTx-021 into one eye is safe and may help people aged 16 and older with Stargardt disease.

  Recruiting at 3 sites Phase 1/2 Ray Therapeutics, Inc. · Bakersfield, California and 2 other locations · NCT07439887

• Tracking how Stargardt-type macular dystrophy progresses in children and adults

  This project will follow children and adults with Stargardt-type macular dystrophies over time to see how their vision and eye findings change.

  Recruiting at 15 sites Astellas Pharma Inc · Phoenix, Arizona and 14 other locations · NCT07425574

• DC6001 tablets in healthy Chinese adults

  This trial will test single and repeated doses of DC6001 tablets in healthy Chinese adults to see how the drug is tolerated, how the body handles it, and whether food affects absorption.

  Recruiting Phase 1 Heronova Pharmaceuticals · Beijing, Beijing Municipality · NCT07417566

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.