Ophthalmology and Visual Sciences

 Purpose of study:
This research study is being done to see if a drug called ALK-001 is safe and well-tolerated in people with Stargardt disease, and to see what effects ALK-001 has on the progression of Stargardt disease over time.

What is Stargardt disease?
Stargardt disease is a rare genetic disorder affecting about 1 in 10,000 people in the United States. In people with Stargardt disease, toxic molecules build up in the retina, the light-sensing tissue at the back of the eye that allows you to see. These molecules damage the retina and cause symptoms such as wavy vision, blind spots, impaired color vision, and difficulty adapting to dim lighting. Vision loss in Stargardt disease is most intense in the macula (the center of the retina), affecting “straight ahead” tasks like reading or driving. Stargardt disease is the most common form of inherited juvenile macular degeneration, and often is diagnosed before age 20. Genetic testing is sometimes performed to confirm a diagnosis of Stargardt disease, and genetic counseling can often provide helpful information to families affected by Stargardt disease.

What is involved in the study?
The study will last about 2 years with up to 12 study visits at the Eye Institute. Fifty people will be asked to participate from across all study centers. Thirty people who participate in the study will be randomly assigned to receive a dose of ALK-001, and 20 people will receive a placebo. After 12 months, half of the participants receiving the placebo will be randomly assigned to receive ALK-001 for the remainder of the study. Participants will not be told which group they have been assigned to.

Compensation:
Research volunteers will receive a $50 gift card for each study visit that they complete.

You may be eligible to participate in this study if you meet these criteria.

1. An eye doctor has told you that you have Stargardt disease
2. You have had genetic testing performed by a certified lab to confirm ABCA4 mutation(s)
3. You are between the ages of 12 and 60 years old
4. You have no history of acute or chronic liver disorder
5. You are able to reliably administer oral medication by yourself or with help

More Information:

Additional information about the Alkeus study can be found at ClinicalTrials.gov.

RB Approval: WIRB Pr. No. 20151467 10/18/2015