ALK-001 for Stargardt Disease

Not currently recruiting at 8 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: Alkeus Pharmaceuticals, Inc.

Disqualifiers: Pregnancy, Lactation, Ocular disorder, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: Long-term

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. ^{1 2 3 4 5}

Why 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. ^{1 2 5 6 7}

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

Healthy as judged by investigator

Has a best-corrected visual acuity (BCVA) greater than approximately 20/160 in at least one eye

I have two mutations causing my eye condition and can see better than 20/160 with correction in one eye.

Exclusion Criteria

I had eye surgery or treatment within the last 3 months.

I do not have any health issues that would stop me from following the study rules or taking the study drug.

Is lactating or pregnant

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ALK-001 to assess long-term safety, pharmacokinetics, and effects on Stargardt disease

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive ALK-001 to assess long-term effects

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• ALK-001

Trial Overview

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.

How Is the Trial Designed?

Group I: ALK-001 Experimental Treatment 1 Intervention

ALK-001 is already approved in United States for the following indications:

Approved in United States as Gildeuretinol for:

• Stargardt disease

Who Is Running the Clinical Trial?

Alkeus Pharmaceuticals, Inc.

Lead Sponsor

Trials

4

Recruited

520+

Published Research Related to This Trial

Isotretinoin effectively inhibited the accumulation of toxic lipofuscin pigments in a mouse model of recessive Stargardt's macular degeneration, suggesting it could delay visual loss in affected patients.

The treatment not only blocked the formation of A2E, a key component in lipofuscin biosynthesis, but also showed no significant visual loss in treated mice, indicating a potential safe therapeutic option for this inherited blinding disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment with isotretinoin inhibits lipofuscin accumulation in a mouse model of recessive Stargardt's macular degeneration. Radu, RA., Mata, NL., Nusinowitz, S., et al.[2018]

Stargardt disease, caused by mutations in the ABCA4 gene, currently lacks effective treatments that have successfully completed clinical trials.

Research is ongoing in various areas, including drugs to reduce harmful vitamin A dimers, stem cell therapies for retinal regeneration, and gene therapy aimed at delivering a functional ABCA4 gene directly into the eye.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[New possibilities in the treatment of Stargardt disease]. Zhorzholadze, NV., Sheremet, NL., Tanas, AS., et al.[2020]

High doses of Fenretinide (800 mg daily for 1 out of every 3 weeks) can significantly suppress rod function in children, as evidenced by elevated dark adaptation thresholds and absent rod ERG responses, but cone function remains normal.

After stopping Fenretinide, rod function appears to recover quickly, with normal or near-normal thresholds and ERG responses, suggesting that the effects on rod function are reversible and that cone function is largely unaffected by the drug's impact on retinol availability.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Total rod ERG suppression with high dose compassionate Fenretinide usage. Marmor, MF., Jain, A., Moshfeghi, D.[2021]

Genetic screening for Stargardt disease using a panel of 5 common ABCA4 mutations identified mutations in 50% of patients, while more comprehensive massive parallel sequencing confirmed a diagnosis in 84% of patients tested.

The majority of mutations found were in the ABCA4 gene, highlighting its significance in Stargardt disease, with specific mutations like p.L541P, p.A1038V, and p.G1961E being particularly prevalent.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Molecular genetic diagnosis of Stargardt disease]. Sheremet, NL., Zhorzholadze, NV., Ronzina, IA., et al.[2019]

Stargardt disease (STGD) is caused by mutations in the ABCR gene, which is crucial for retinal function and is expressed primarily in rod photoreceptors.

The study identified 19 different mutations in the ABCR gene among STGD families, confirming its role as the causal gene for this autosomal recessive retinal disorder.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy. Allikmets, R., Singh, N., Sun, H., et al.[2022]

In a study involving 23 subjects with Stargardt disease, emixustat demonstrated a dose-dependent effect on rod b-wave recovery rates, with the 10 mg dose showing near-complete suppression, indicating its potential efficacy in targeting RPE65.

The safety profile of emixustat was consistent with previous studies, primarily involving ocular adverse events, which suggests it may be a viable treatment option for Stargardt disease as it moves into a longer phase 3 trial.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Randomised study evaluating the pharmacodynamics of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease. Kubota, R., Birch, DG., Gregory, JK., et al.[2022]

There are currently eight ongoing clinical trials for Stargardt disease, focusing on various treatment strategies including gene therapy, neuroprotection, and stem cell therapy.

These trials aim to address the disease through different mechanisms, such as inhibiting toxic vitamin A dimers and restoring the function of the ABCA4 gene, which raises hope for effective treatments in the future.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical Tests Testing New Therapies for Stargardt Disease]. Kousal, B., Ďuďáková, Ľ., Hlavatá, L., et al.[2019]

Isotretinoin effectively inhibited the accumulation of toxic lipofuscin pigments, specifically A2E, in a mouse model of recessive Stargardt's macular degeneration, suggesting a potential therapeutic strategy for this inherited blinding disease.

The treatment with isotretinoin did not result in significant visual loss in the treated mice, indicating its safety and potential efficacy in delaying visual decline associated with Stargardt's disease and possibly other retinal degenerations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Isotretinoin treatment inhibits lipofuscin accumulation in a mouse model of recessive Stargardt's macular degeneration. Radu, RA., Mata, NL., Nusinowitz, S., et al.[2013]

Long-term treatment with deuterium-enriched vitamin A (C20-D(3)-vitamin A) in a mouse model of Stargardt disease showed potential to reduce lipofuscin accumulation in the retinal pigment epithelium, which is linked to vision loss.

The study suggests that preventing the formation of vitamin A dimers may slow down retinal damage and vision loss associated with Stargardt disease, indicating a possible new therapeutic approach for patients with ABCA4 genetic defects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

C20-D3-vitamin A slows lipofuscin accumulation and electrophysiological retinal degeneration in a mouse model of Stargardt disease. Ma, L., Kaufman, Y., Zhang, J., et al.[2021]

The overlapping adeno-associated viral (AAV) dual vector system for delivering the ABCA4 gene showed no signs of toxicity in Abca4-/- mice, indicating it is safe for potential use in humans.

Both structural and functional assessments of the retina, including optical coherence tomography and electroretinography, confirmed that the dual vector system did not cause any adverse effects compared to sham injections, supporting its advancement to clinical trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Assessment of AAV Dual Vector Safety in theAbca4-/- Mouse Model of Stargardt Disease. McClements, ME., Barnard, AR., Charbel Issa, P., et al.[2021]

Citations

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alkeuspharma.com

alkeuspharma.com/presentation-of-gildeuretinol-data-at-aao-2025/

October 9, 2025

The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol ...

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT02402660

Phase 2 Tolerability and Effects of ALK-001 on Stargardt ...

This study evaluates the effects of orally-administered ALK-001 on the progression of Stargardt disease (ABCA4-related).

3.

firstwordpharma.com

firstwordpharma.com/story/5983376

Alkeus Pharmaceuticals Announces Gildeuretinol Data ...

The Tolerability and Effects of ALK-001 on Stargardt diseasE (TEASE) studies consist of four independent clinical studies of oral gildeuretinol ...

4.

iovs.arvojournals.org

iovs.arvojournals.org/article.aspx?articleid=2804409

Safety and Efficacy of Oral Gildeuretinol for Stargardt Disease

Daily oral gildeuretinol significantly slowed the growth of atrophic retinal lesions in Stargardt disease. The treatment was well-tolerated over the duration ...

5.

ophthalmologytimes.com

ophthalmologytimes.com/view/alkeus-pharmaceuticals-receives-fda-rare-pediatric-disease-and-fast-track-designations-for-gildeuretinol-as-treatment-for-stargardt-disease

Alkeus Pharmaceuticals receives FDA Rare Pediatric ...

Gildeuretinol (ALK-001) received FDA Rare Pediatric Disease and Fast Track designations for Stargardt disease, highlighting its potential as a ...

6.

glance.eyesoneyecare.com

glance.eyesoneyecare.com/stories/2025-05-09/favorable-safety-data-supports-alkeus-oral-gildeuretinol-for-dry-amd/

Favorable safety data supports Alkeus' oral gildeuretinol for ...

As such: These Vitamin A dimers may lead to vision loss, particularly in degenerative retinal diseases such as GA and Stargardt disease—two ...

7.

alkeuspharma.com

alkeuspharma.com/presentation-of-positive-interim-tease-3-study-data-in-early-stage-stargardt-disease-patients-treated-with-oral-gildeuretinol/

Alkeus Pharmaceuticals Announces Presentation of ...

Alkeus Pharmaceuticals Announces Presentation of Positive Interim TEASE-3 Study Data in Early-Stage Stargardt Disease Patients Treated with Oral ...