Actively Recruiting
Study of ALK-001 on the Progression of Stargardt Disease
Led by Alkeus Pharmaceuticals, Inc. · Updated on 2026-05-05
230
Participants Needed
3
Research Sites
243 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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)
CONDITIONS
Official Title
Study of ALK-001 on the Progression of Stargardt Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female, 8 to 45 years of age (inclusive) on the day of screening.
- Female participants of childbearing potential and fertile males with female partners of childbearing potential must agree to use protocol-defined contraception from consent until 90 days and 30 days after last dose, respectively.
- Clinical diagnosis of typical autosomal recessive Stargardt macular dystrophy.
- Provided a genetic report from an accredited organization showing disease-causing mutation.
- Signed and dated informed consent forms or assent as appropriate.
You will not qualify if you...
- Taken disallowed items (vitamin A or beta-carotene supplements, liver-based products, or prescription oral retinoid medications) within 30 days of randomization.
- Lactating, pregnant, positive pregnancy test at screening or randomization, or planning pregnancy during study. Males planning to father a child during study.
- Previously participated in gene therapy, cell therapy, or device study for Stargardt disease unless confirmed placebo arm with no surgery.
- Participated in a drug study for Stargardt disease within past 6 months.
- Participated in drug study for other conditions within 5 half-lives of investigational drug before screening unless confirmed placebo arm.
- Participated in investigational device study within 30 days prior to screening or longer if device might affect study outcomes.
- Plans to participate in any other drug or device study during this study.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 3 locations
1
Associated Retina Consultants
Phoenix, Arizona, United States, 85020
Actively Recruiting
2
Vitreo Retinal Associates
Gainesville, Florida, United States, 32607
Not Yet Recruiting
3
Erie Retina Research
Erie, Pennsylvania, United States, 16505
Actively Recruiting
Research Team
F
For trial questions: trials@alkeuspharma.com or 877-255-7476
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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