Testing ALK-001 to Slow the Progression of Stargardt Disease
Tests ALK-001 against placebo in people with inherited Stargardt disease.
Plain-language title generated from the registry record
Registry title: Study of ALK-001 on the Progression of Stargardt Disease
Key facts
- Where
- Aurora, Boston, Columbus, Dallas, Erie, Fairfax, Fort Worth, Gainesville, Grand Rapids, Norfolk, Philadelphia, Phoenix, Pittsburgh, and San Antonio, United StatesSee the 14 sites
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 8 and 45 years old?
Source: NCT07419334, who can take part
Where in United States
- University of Colorado SOM, Sue-Anschutz-Rodgers Eye CenterAurora, United StatesTaking people
- Tufts Medical Center New England Eye CenterBoston, United StatesNot yet recruiting
- Ohio State Havener Eye InstituteColumbus, United StatesTaking people
- Retina Foundation of the SouthwestDallas, United StatesTaking people
- Erie Retina ResearchErie, United StatesTaking people
- The Retina Group of WashingtonFairfax, United StatesTaking people
- Star RetinaFort Worth, United StatesTaking people
- Vitreo Retinal AssociatesGainesville, United StatesTaking people
- Foundation for Vision ResearchGrand Rapids, United StatesTaking people
- Wagner Kapoor Research InstituteNorfolk, United StatesTaking people
- Mid Atlantic RetinaPhiladelphia, United StatesNot yet recruiting
- Associated Retina ConsultantsPhoenix, United StatesTaking people
- UPMC Vision InstitutePittsburgh, United StatesTaking people
- Medical Center Ophthalmology AssociatesSan Antonio, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Alkeus Pharmaceuticals, Inc.
- Trial code
- NCT07419334EU 2026-525296-60-00
- Record updated
- 18 September 2026
- Original description
This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)Read full original descriptionShow less
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 study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)
Inclusion: Male or female, 8 to 45 years of age (inclusive) on the day of screening; 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; Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy; Have provided a genetic report by any regional accredited organization that provides certified testing indicating the presence of disease-causing mutation; Have signed and dated the informed consent forms (ICFs; or assent as appropriate) to participate.
Exclusion: Has taken disallowed items (supplements containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of randomization; 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; 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; Anticipates participating in any other drug or device study within the duration of the study; 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.