What is the mechanism of action of this treatment?

Fundus Flavimaculatus, or Stargardt disease, treatments like ALK-001 (C20-D3-retinyl acetate) work by slowing the accumulation of toxic byproducts in the retina, which are formed during the visual cycle. These toxic substances, such as lipofuscin, cause retinal cell damage and vision loss. By using a modified form of vitamin A, ALK-001 aims to reduce the formation of these harmful byproducts, potentially preserving retinal function and slowing disease progression. This mechanism is crucial for patients as it targets the root cause of retinal degeneration, offering hope for better long-term management of the disease.

What side effects are associated with this type of intervention?

Common treatments for Fundus Flavimaculatus, such as ALK-001 (a modified form of vitamin A), aim to slow the accumulation of toxic byproducts in the retina. Known side effects of similar treatments may include mild gastrointestinal discomfort, changes in liver enzyme levels, and potential teratogenic effects if taken in high doses. Patients should be monitored for these side effects, and treatment should be adjusted accordingly.

What prior FDA approvals exist?

Fundus Flavimaculatus, a variant of Stargardt disease, has limited FDA-approved treatments. The primary focus has been on therapies that slow the progression of retinal degeneration. ALK-001 (C20-D3-retinyl acetate) is a modified form of vitamin A designed to reduce toxic byproduct accumulation in the retina. Similar treatments include other forms of vitamin A derivatives and investigational drugs aimed at reducing retinal damage. However, specific FDA approvals for treatments directly targeting Fundus Flavimaculatus are sparse, and ongoing research continues to explore effective therapies.

What other types of research exist?

Promising novel alternatives to ALK-001 for Fundus Flavimaculatus patients include elamipretide, which has shown positive effects on visual function in intermediate AMD, and RNAi therapies like vutrisiran, which target transthyretin amyloidosis but may have potential applications in retinal diseases. Further studies and clinical trials are needed to confirm their efficacy and safety for Stargardt disease.

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Vitamin A Derivative

ALK-001 for Stargardt Disease (TEASE Trial)

Phase 2

Recruiting

Research Sponsored by Alkeus Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female between 8 and 70 years old (inclusive), with any visual acuity

Has a clinical diagnosis of typical autosomal recessive Stargardt macular dystrophy (STGD1)

Must not have

Has active or historical acute or chronic liver disorder

Has had intraocular surgery or injections in the primary study eye within 90 days of the screening visit

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to 24 months

Summary

This trial tests a modified vitamin A pill called ALK-001 for people with Stargardt disease. The drug aims to prevent harmful clumps of vitamin A in the eye that cause vision loss.

Who is the study for?

This trial is for individuals aged 8-70 with Stargardt disease, a genetic eye condition. Participants must have clear vision media and no allergies to eye dilation drops, be healthy overall, able to follow the study plan for two years, and females of childbearing age must agree to use contraception. Those with certain medical conditions or recent eye treatments are excluded.

What is being tested?

The trial tests ALK-001 (C20-D3-retinyl acetate) against a placebo to assess its safety over the long term and its impact on slowing down the progression of Stargardt disease. Patients will either receive ALK-001 or a placebo in this randomized study.

What are the potential side effects?

While specific side effects are not listed here, participants will be monitored for any adverse reactions due to ALK-001 as compared to those receiving a placebo. Side effects could range from minor issues at the site of administration to more systemic responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 8 and 70 years old and my ability to see is not limited.

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I have been diagnosed with Stargardt disease.

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My eyes can be clearly imaged and I'm not allergic to dilation drops.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a current or past liver condition.

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I have not had eye surgery or injections in my main eye within the last 3 months.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to 24 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to 24 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to 24 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Effects of ALK-001 on the progression of Stargardt disease

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ALK-001Experimental Treatment1 Intervention

Daily, oral administration of one capsule. See details below.

Group II: PlaceboPlacebo Group1 Intervention

Daily, oral administration of one capsule. See details below.

0 / 5Eligibility 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, treatments like ALK-001 (C20-D3-retinyl acetate) work by slowing the accumulation of toxic byproducts in the retina, which are formed during the visual cycle. These toxic substances, such as lipofuscin, cause retinal cell damage and vision loss. By using a modified form of vitamin A, ALK-001 aims to reduce the formation of these harmful byproducts, potentially preserving retinal function and slowing disease progression. This mechanism is crucial for patients as it targets the root cause of retinal degeneration, offering hope for better long-term management of the disease.

Emerging biological therapies for the treatment of age-related macular degeneration.AAV2 delivery of Flt23k intraceptors inhibits murine choroidal neovascularization.LAST II: Differential temporal responses of macular pigment optical density in patients with atrophic age-related macular degeneration to dietary supplementation with xanthophylls.