ALK-001 for Stargardt Disease

Recruiting in Palo Alto (17 mi)

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Alkeus Pharmaceuticals, Inc.

No Placebo Group

Prior Safety Data

Approved in 1 Jurisdiction

Trial Summary

What is the purpose of this trial?This trial tests a special type of vitamin A called ALK-001 to see if it can safely slow down vision loss in people with Stargardt disease by reducing harmful substances in the eye. ALK-001 is designed to reduce the accumulation of toxic substances in the retina, which are associated with vision loss in Stargardt disease.

Eligibility Criteria

This trial is for individuals at least 8 years old with a clinical diagnosis of Stargardt disease, who have two specific gene mutations (unless the sponsor says otherwise), and vision better than approximately 20/160 in one eye. Participants must be healthy overall, able to follow the study plan for 24 months, not pregnant or breastfeeding, and without recent ocular interventions or conditions that could affect study results.

Inclusion Criteria

Healthy as judged by investigator

Has a best-corrected visual acuity (BCVA) greater than approximately 20/160 in at least one eye

I have two mutations causing my eye condition and can see better than 20/160 with correction in one eye.

+5 more

Exclusion Criteria

I had eye surgery or treatment within the last 3 months.

I do not have any health issues that would stop me from following the study rules or taking the study drug.

Is lactating or pregnant

+2 more

Participant Groups

The TEASE trial tests ALK-001's long-term safety and its impact on slowing down Stargardt disease progression. It's an extension of a previous study (NCT02402660) where participants are invited to continue treatment. The drug's behavior in the body over time will also be studied.

1Treatment groups

Experimental Treatment

Group I: ALK-001Experimental Treatment1 Intervention

ALK-001 is already approved in United States for the following indications:

🇺🇸 Approved in United States as Gildeuretinol for: