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Vitamin A Derivative

ALK-001 for Stargardt Disease

Phase 2
Waitlist Available
Research Sponsored by Alkeus Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Has at least two ABCA4 disease-causing mutations, unless authorized by sponsor
Clinical diagnosis of Stargardt disease (STGD1)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
No Placebo-Only Group
All Individual Drugs Already Approved
Approved for 5 Other Conditions

Summary

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.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
While specific side effects aren't listed here, potential risks may include reactions related to vitamin A derivatives such as skin changes or vision disturbances since ALK-001 is a form of vitamin A. Regular monitoring will help identify any adverse effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have two mutations causing my ABCA4 disease, unless the sponsor has made an exception.
Select...
I have been diagnosed with Stargardt disease.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 5 Other Conditions
This treatment demonstrated efficacy for 5 other conditions.

Trial Design

1Treatment groups
Experimental Treatment
Group I: ALK-001Experimental Treatment1 Intervention

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Fundus Flavimaculatus, or Stargardt disease, is treated by targeting the accumulation of toxic vitamin A dimers in the retina. ALK-001 (C20-D3-retinyl acetate) works by slowing this accumulation, which is important because these dimers form lipofuscin, a substance that damages retinal cells. By reducing the buildup of these harmful compounds, such treatments aim to preserve retinal function and slow the progression of vision loss, offering patients a better quality of life.
Tissue engineering in age-related macular degeneration: a mini-review.Comparative Safety of Bevacizumab, Ranibizumab, and Aflibercept for Treatment of Neovascular Age-Related Macular Degeneration (AMD): A Systematic Review and Network Meta-Analysis of Direct Comparative Studies.AAV2 delivery of Flt23k intraceptors inhibits murine choroidal neovascularization.

Find a Location

Who is running the clinical trial?

Alkeus Pharmaceuticals, Inc.Lead Sponsor
3 Previous Clinical Trials
480 Total Patients Enrolled
2 Trials studying Stargardt Disease
180 Patients Enrolled for Stargardt Disease
Leonide Saad, PhDStudy DirectorAlkeus Pharmaceuticals, Inc.
3 Previous Clinical Trials
480 Total Patients Enrolled
2 Trials studying Stargardt Disease
180 Patients Enrolled for Stargardt Disease

Media Library

ALK-001 (Vitamin A Derivative) Clinical Trial Eligibility Overview. Trial Name: NCT04239625 — Phase 2
Stargardt Disease Research Study Groups: ALK-001
Stargardt Disease Clinical Trial 2023: ALK-001 Highlights & Side Effects. Trial Name: NCT04239625 — Phase 2
ALK-001 (Vitamin A Derivative) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04239625 — Phase 2
Stargardt Disease Patient Testimony for trial: Trial Name: NCT04239625 — Phase 2
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