Protocol Number: ALK001-P1002
Primary Investigator: Liliya Sutherland, D.O.
The purpose of this study is to evaluate how ALK-001 affects the vision of those with Stargardt disease. ALK-001 is a molecule that resembles vitamin A. When taken orally, ALK-001 replaces the majority of vitamin A in the body. ALK-001 slows the formation of vitamin A “dimers” in the eye. Vitamin A dimers are toxic molecules believed to contribute to Stargardt disease. We hope that slowing vitamin A dimer formation can slow vision loss associated with Stargardt disease. The use of ALK-001 is investigational. The word “investigational” means that ALK-001 is not approved for sale or marketing by the Food and Drug Administration (FDA). The FDA is allowing the use of ALK001 in this study. A research study is not the same as going to the doctor to receive treatment with a commercial drug. Rather, it is a scientific approach to generate new knowledge and test if a study drug may be approved by FDA as a safe and effective treatment.
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