ALK-001 for Stargardt Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests an experimental treatment called ALK-001 to determine if it can slow the progression of Stargardt disease, a genetic eye condition affecting vision. Researchers aim to understand the long-term safety and pharmacokinetics of the drug. It is suitable for individuals diagnosed with Stargardt disease who have specific genetic mutations linked to it and can see better than approximately 20/160 in at least one eye. Participants should be generally healthy and able to adhere to the study requirements over 24 months. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in Stargardt disease treatment.
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.
Is there any evidence suggesting that ALK-001 is likely to be safe for humans?
Research has shown that ALK-001, also known as oral gildeuretinol acetate, was safe in earlier studies for Stargardt disease. These studies found that patients generally tolerated ALK-001 well, with no major safety issues reported and no serious side effects experienced.
Currently, ALK-001 is being tested in a Phase 2 trial. This indicates that it has shown initial safety in earlier studies, but researchers are still collecting more detailed safety information. Overall, current data suggests that ALK-001 is safe for use at this stage of the research.12345Why do researchers think this study treatment might be promising?
Unlike the standard of care for Stargardt Disease, which typically involves managing symptoms and protecting the retina with supplements like vitamin A, ALK-001 presents a novel approach. Researchers are excited about ALK-001 because it introduces a new active ingredient, a modified form of vitamin A that aims to slow down the toxic buildup in the retina, a key factor in Stargardt Disease. This unique mechanism of action targets the root cause rather than just alleviating symptoms, offering hope for more effective disease management.
What evidence suggests that ALK-001 might be an effective treatment for Stargardt disease?
Research has shown that ALK-001, also known as gildeuretinol, may help treat Stargardt disease. Studies have found that daily use of ALK-001 can significantly slow the progression of vision loss in patients with this condition. Patients have tolerated this treatment well, experiencing few uncomfortable side effects. The FDA has recognized ALK-001's potential and granted it special designations to expedite its availability to patients. Overall, evidence suggests that ALK-001 could effectively slow the progression of Stargardt disease.12567
Who Is on the Research Team?
Leonide Saad, PhD
Principal Investigator
Alkeus Pharmaceuticals, Inc.
Are You a Good Fit for This Trial?
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 for a Trial Participant
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
What Are the Treatments Tested in This Trial?
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