NORTHSTAR

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) - NORTHSTAR

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Eye disorders
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All genders
person-wave 8-45
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Overview
Principal Investigator: Michelle Liang, MD

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).

Study details
Inclusion Criteria
  • Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy.
  • Have 20/200 or better in the eligible eye(s)
  • 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
Exclusion Criteria
  • 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 the 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 any history or existence of a medical condition that causes poor absorption of fat-soluble vitamins, such as vitamin A.
Study Requirements

The total length of the study is 2 years and 2.5 months including the screening period and safety follow-up. There will be 9 in person study visits. In addition there will be weekly phone calls from weeks 1-11 which will switch to monthly for months 4 to the end. The in person visits will include a few blood draws, urine collection, reading speed tests, imaging, ECG, ophthalmic examination, visual acuity, and questionnaires.