200 Participants Needed

ALK-001 for Stargardt Disease

Recruiting at 8 trial locations
Age: Any Age
Sex: Any
Trial Phase: Phase 2
Sponsor: Alkeus Pharmaceuticals, Inc.
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

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.

What safety data exists for ALK-001 (Gildeuretinol) in humans?

Research on a similar compound, C20-D3-vitamin A, suggests it may slow disease progression in Stargardt disease without noted safety concerns in mice. However, specific human safety data for ALK-001 is not provided in the available research.12345

What makes the drug ALK-001 unique for treating Stargardt disease?

ALK-001 is unique because it is a modified form of vitamin A designed to slow the accumulation of toxic vitamin A byproducts in the retina, which is a novel approach since there are currently no approved treatments for Stargardt disease.678910

What is the purpose of this trial?

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.

Research Team

LS

Leonide Saad, PhD

Principal Investigator

Alkeus Pharmaceuticals, Inc.

Eligibility Criteria

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.
See 5 more

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
See 2 more

Timeline

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

Treatment Details

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

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

🇺🇸
Approved in United States as Gildeuretinol for:
  • Stargardt disease

Find a Clinic Near You

Who Is Running the Clinical Trial?

Alkeus Pharmaceuticals, Inc.

Lead Sponsor

Trials
4
Recruited
520+

Findings from Research

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.
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]
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.
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.
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]

References

Treatment with isotretinoin inhibits lipofuscin accumulation in a mouse model of recessive Stargardt's macular degeneration. [2018]
Isotretinoin treatment inhibits lipofuscin accumulation in a mouse model of recessive Stargardt's macular degeneration. [2013]
C20-D3-vitamin A slows lipofuscin accumulation and electrophysiological retinal degeneration in a mouse model of Stargardt disease. [2021]
Assessment of AAV Dual Vector Safety in theAbca4-/- Mouse Model of Stargardt Disease. [2021]
Total rod ERG suppression with high dose compassionate Fenretinide usage. [2021]
A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy. [2022]
Randomised study evaluating the pharmacodynamics of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease. [2022]
8.Russia (Federation)pubmed.ncbi.nlm.nih.gov
[New possibilities in the treatment of Stargardt disease]. [2020]
9.Czech Republicpubmed.ncbi.nlm.nih.gov
[Clinical Tests Testing New Therapies for Stargardt Disease]. [2019]
10.Russia (Federation)pubmed.ncbi.nlm.nih.gov
[Molecular genetic diagnosis of Stargardt disease]. [2019]
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top
Terms of Service·Privacy Policy·Cookies·Security