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)
• 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.