Study of ALK-001 on the Progression of Stargardt Disease (NCT07419334) | Clinical Trial Compass
RecruitingPhase 3
Study of ALK-001 on the Progression of Stargardt Disease
United States230 participantsStarted 2026-06
Plain-language summary
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)
Who can participate
Age range
8 Years – 45 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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 criteria
. Has taken disallowed items (supplements containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of randomization.
. 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.
. 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence
Timeframe: Month 6 to Month 24
Trial details
NCT IDNCT07419334
SponsorAlkeus Pharmaceuticals, Inc.
Sponsor typeINDUSTRY
Study typeINTERVENTIONAL
Primary completion2029-10
Contact for this trial
For trial questions: trials@alkeuspharma.com or 877-255-7476
. 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.