Last updated on February 2018

Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease


Brief description of study

The purpose of this study is to determine the long term safety and tolerability of ALK-001 (C20-D3-retinyl acetate), and to explore the effects of ALK-001 on the progression of Stargardt disease in patients between the ages of 12 and 60 years old.

Funding Source - FDA OOPD

Detailed Study Description

This is a multicenter, randomized, double-masked, parallel group, placebo-controlled study evaluating the effects of ALK-001 administered daily by mouth in subjects with Stargardt disease (ABCA4-related).

Stargardt disease is a rare genetic disorder that leads to damage to the retina and results in legal blindness. The onset of symptoms usually occurs during one's teenage years, although symptoms can appear in children as young as 4 years old. There is currently no treatment for Stargardt.

Stargardt disease is caused by a defective ABCA4 gene, which affects the processing of vitamin A in the eye and leads to the formation of toxic vitamin A aggregates (called "vitamin A dimers") in the eye. Vitamin A dimers are thought to contribute to vision loss in Stargardt disease. ALK-001 is a chemically-modified vitamin A designed as a replacement of vitamin A. ALK-001 has been changed specifically to prevent the formation of toxic vitamin A dimers in the eye, without altering the normal processing of vitamin A to enable vision.

Trial participants will be randomly assigned to receive ALK-001 (30 subjects) or placebo (20 subjects) for one year. After one year of treatment, half of the participants (10 subjects) receiving placebo will be randomly crossed over to receive ALK-001 for the following 12 months, while the remaining 10 subjects will continue on the placebo. All subjects initially receiving ALK-001 will remain on the same treatment for 12 months.

Follow-up visits will take place 4 and 8 weeks after the initiation of treatment to assess safety and tolerability, then after 6, 9, 12, 15, 18, 21 and 24 months to assess the safety, tolerability and effects of ALK-001 on the progression of Stargardt disease.

The study is double-masked so that neither the participants, the clinical staff, nor the sponsor, are aware of the treatment allocation (ALK-001 or placebo). A Data Safety Monitoring Board (DSMB) will review safety and efficacy data throughout the study.

Clinical Study Identifier: NCT02402660

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Recruitment Status: Open


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