Recruiting NCT07419334
Study of ALK-001 on the Progression of Stargardt Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ALK-001, Placebo.
- Who it may be relevant to
- Registry conditions: Stargardt Disease. Basic parameters: 8 years — 45 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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)
Overview
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)
Interventions
- Drug ALK-001
Daily oral administration for 24 months - Drug Placebo
Daily oral administration for 24 months
Primary outcome measures
- Annualized growth rate of retinal atrophic lesions measured by Fundus Autofluorescence [Time frame: Month 6 to Month 24]
Secondary outcome measures (1)
- Changes in Low Luminance Visual Acuity from baseline to Month 24 [Time frame: baseline to Month 24]
Eligibility criteria
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.
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 5 centers
- Associated Retina Consultants — Phoenix
- Vitreo Retinal Associates — Gainesville
- Erie Retina Research — Erie
- Retina Foundation of the Southwest — Dallas
- Star Retina — Fort Worth
Identifiers
NCT: NCT07419334 · ALK001-P1004 · 2026-525296-60-00