ALK-001 for Stargardt Disease
Trial Summary
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
What safety data exists for ALK-001 (Gildeuretinol) in humans?
What makes the drug ALK-001 unique for treating Stargardt disease?
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.
Research Team
Leonide Saad, PhD
Principal Investigator
Alkeus Pharmaceuticals, Inc.
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
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive ALK-001 to assess long-term safety, pharmacokinetics, and effects on Stargardt disease
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants continue to receive ALK-001 to assess long-term effects
Treatment Details
Interventions
- ALK-001
ALK-001 is already approved in United States for the following indications:
- Stargardt disease
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alkeus Pharmaceuticals, Inc.
Lead Sponsor