for people ages 12-60 (full criteria)
study started
estimated completion
Michael Gorin, MD, PhD(ucla)



The purpose of this study is to determine the long term safety and tolerability of ALK-001 (C20-D3-retinyl acetate), and to explore the effects of ALK-001 on the progression of Stargardt disease in patients between the ages of 12 and 60 years old. Funding Source - FDA OOPD

Official Title

A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease


This is a multicenter, randomized, double-masked, parallel group, placebo-controlled study evaluating the effects of ALK-001 administered daily by mouth in subjects with Stargardt disease (ABCA4-related).

Stargardt disease is a rare genetic disorder that leads to damage to the retina and results in legal blindness. The onset of symptoms usually occurs during one's teenage years, although symptoms can appear in children as young as 4 years old. There is currently no treatment for Stargardt.

Stargardt disease is caused by a defective ABCA4 gene, which affects the processing of vitamin A in the eye and leads to the formation of toxic vitamin A aggregates (called "vitamin A dimers") in the eye. Vitamin A dimers are thought to contribute to vision loss in Stargardt disease. ALK-001 is a chemically-modified vitamin A designed as a replacement of vitamin A. ALK-001 has been changed specifically to prevent the formation of toxic vitamin A dimers in the eye, without altering the normal processing of vitamin A to enable vision.

Trial participants will be randomly assigned to receive ALK-001 (30 subjects) or placebo (20 subjects) for one year. After one year of treatment, half of the participants (10 subjects) receiving placebo will be randomly crossed over to receive ALK-001 for the following 12 months, while the remaining 10 subjects will continue on the placebo. All subjects initially receiving ALK-001 will remain on the same treatment for 12 months.

Follow-up visits will take place 4 and 8 weeks after the initiation of treatment to assess safety and tolerability, then after 6, 9, 12, 15, 18, 21 and 24 months to assess the safety, tolerability and effects of ALK-001 on the progression of Stargardt disease.

The study is double-masked so that neither the participants, the clinical staff, nor the sponsor, are aware of the treatment allocation (ALK-001 or placebo). A Data Safety Monitoring Board (DSMB) will review safety and efficacy data throughout the study.


Stargardt Disease Stargardt Macular Degeneration Stargardt Macular Dystrophy Autosomal Recessive Stargardt Disease 1 (ABCA4-related) Macular Degeneration Vitamin A ALK-001


For people ages 12-60

Simplified Inclusion Criteria:

  • Male or female between 12 and 60 years old (inclusive), with any visual acuity
  • Has a clinical diagnosis of typical autosomal recessive Stargardt macular dystrophy (STGD1)
  • Has provided a genetic report indicating at least two ABCA4 disease-causing mutations. When only one ABCA4 disease-causing mutation is reported, sponsor's permission will be required.
  • At least one eye (called the "primary study eye") must have at least one well-demarcated area of significantly reduced autofluorescence as imaged by fundus autofluorescence (FAF)
  • Primary study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to permit good quality retinal imaging
  • Healthy as judged by investigator
  • Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 24-month study
  • Has signed and dated the informed consent forms (or assent where appropriate) to participate
  • Female of childbearing potential has signed the informed consent about birth defects or attestation on contraception requirements

Main Exclusion Criteria:

  • Has taken disallowed items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days
  • Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization
  • Has concurrent medical condition or history, which in the opinion of the investigator, is likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or study procedures
  • Has clinically significant abnormal laboratory result(s) at screening
  • Has active or historical acute or chronic liver disorder
  • Has active or historical ocular disorder in the primary study eye that, in the opinion of the investigator, may confound assessment of the retina morphologically or functionally (this could include for example cataract surgery within the past 6 months, choroidal neovascularization (CNV), glaucoma, recurring uveitis, diabetic retinopathy, other retinal disease, etc.)
  • Has had intraocular surgery or injections in the primary study eye within 90 days of the screening visit
  • Has a clinically significant abnormal electrocardiogram (ECG), or has a corrected QT interval (QTc) that is 450 ms or greater


  • University of California Los Angeles - Jules Stein Eye Institute accepting new patients
    Los Angeles California 90095 United States
  • University of Utah - Moran Eye Institute accepting new patients
    Salt Lake City Utah 84132 United States

Lead Scientist


accepting new patients
Start Date
Completion Date
Alkeus Pharmaceuticals, Inc.
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Phase 2
Study Type
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