What side effects are associated with this type of intervention?

Common treatments for Stargardt disease, such as ALK-001 (C20-D3-retinyl acetate), aim to slow the accumulation of toxic vitamin A dimers in the retina. While specific side effects for ALK-001 are not detailed, treatments for retinal conditions often include potential side effects like ocular discomfort, dry eye, and blurred vision. In some cases, more severe reactions such as retinal damage or inflammation may occur, necessitating close monitoring by an ophthalmologist.

What prior FDA approvals exist?

Fundus Flavimaculatus, also known as Stargardt disease, is a genetic eye disorder that leads to progressive vision loss. Currently, there are no FDA-approved treatments specifically for slowing the accumulation of toxic vitamin A dimers in the retina for this condition. However, investigational drugs like ALK-001 (C20-D3-retinyl acetate) are being studied for their potential to slow disease progression by targeting these toxic dimers. Broader treatment approaches for retinal diseases often involve the use of anti-VEGF agents, though these are not specific to Stargardt disease.

What other types of research exist?

Promising novel alternatives to ALK-001 for Fundus Flavimaculatus (Stargardt disease) patients include gene therapy approaches, such as those targeting the ABCA4 gene mutations, and treatments aimed at reducing retinal oxidative stress or improving mitochondrial function. While specific alternatives were not detailed in the provided context, ongoing research in these areas holds potential for new therapeutic options in 2024.

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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 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

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

0 / 2Eligibility criteria met

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.

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