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Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

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ClinicalTrials.gov Identifier: NCT02402660
Recruitment Status : Recruiting
First Posted : March 30, 2015
Last Update Posted : March 3, 2022
Information provided by (Responsible Party):
Alkeus Pharmaceuticals, Inc.

Brief Summary:

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 8 and 70 years old.

Funding Source - FDA OOPD

Condition or disease Intervention/treatment Phase
Stargardt Disease Stargardt Macular Degeneration Stargardt Macular Dystrophy Autosomal Recessive Stargardt Disease 1 (ABCA4-related) Drug: ALK-001 Drug: Placebo Phase 2

Detailed Description:
This study evaluates the effects of orally-administered ALK-001 on the progression of Stargardt disease (ABCA4-related). Stargardt disease is a rare genetic disorder that leads to damage to the retina and results in legal blindness. 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, the investigational drug, is a chemically-modified vitamin A designed as a replacement of vitamin A to prevent the formation of toxic vitamin A dimers in the eye. Trial participants will receive either ALK-001 or placebo, and follow-up visits will take place periodically for up to 24 months. There is currently no treatment for Stargardt disease.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 140 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : August 2015
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : March 2025

Arm Intervention/treatment
Experimental: ALK-001
Daily, oral administration of one capsule. See details below.
Drug: ALK-001
Daily, oral administration for 24 months
Other Names:
  • C20-D3-Retinyl Acetate
  • C20 Deuterated vitamin A

Placebo Comparator: Placebo
Daily, oral administration of one capsule. See details below.
Drug: Placebo
Daily, oral administration for 24 months

Primary Outcome Measures :
  1. Safety and tolerability of 24 months of daily dosing of ALK-001 assessed by Incidence and/or clinically significant changes of a combination of ocular and non-ocular adverse events [ Time Frame: From baseline to 24 months ]

Secondary Outcome Measures :
  1. Effects of ALK-001 on the progression of Stargardt disease [ Time Frame: From baseline to 24 months ]
    Combination of changes in atrophic lesion size, best corrected visual acuity (BCVA), and ocular assessments.

  2. Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma [ Time Frame: Up to 24 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Simplified Inclusion Criteria:

  • 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)
  • 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), have decreased retinal sensitivity as measured by microperimetry, or have maculopathy expected to progress over the duration of the study
  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02402660

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Contact: Leonide Saad, PhD 800-287-2755 trials@alkeus.com

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United States, California
University of California Los Angeles - Jules Stein Eye Institute Recruiting
Los Angeles, California, United States, 90095
Principal Investigator: Michael Gorin, MD, PhD         
Sub-Investigator: Steven Nusinowitz, PhD         
United States, Florida
Vitreoretinal Associates Recruiting
Gainesville, Florida, United States, 32607
Principal Investigator: Christine Kay, MD         
Sub-Investigator: Jing Zhang, MD         
University of Miami - Bascom Palmer Eye Institute Recruiting
Miami, Florida, United States, 33136
Principal Investigator: Byron Lam, MD         
United States, Maryland
Johns Hopkins - Wilmer Eye Institute Active, not recruiting
Baltimore, Maryland, United States, 21287
United States, New York
Columbia University Medical Center - Harkness Eye Institute Recruiting
New York, New York, United States, 10032
Principal Investigator: Stephen Tsang, MD, PhD         
United States, Utah
University of Utah - Moran Eye Institute Completed
Salt Lake City, Utah, United States, 84132
United States, Wisconsin
Medical College of Wisconsin - Eye Institute Active, not recruiting
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Alkeus Pharmaceuticals, Inc.
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Study Director: Hendrik Scholl, MD University of Basel
Study Director: Leonide Saad, PhD Alkeus Pharmaceuticals, Inc.
Additional Information:

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Responsible Party: Alkeus Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02402660    
Other Study ID Numbers: ALK001-P1002
R01FD004098 ( U.S. FDA Grant/Contract )
R01FD006016 ( Other Grant/Funding Number: FDA OOPD )
First Posted: March 30, 2015    Key Record Dates
Last Update Posted: March 3, 2022
Last Verified: February 2022
Additional relevant MeSH terms:
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Macular Degeneration
Stargardt Disease
Retinal Degeneration
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Vitamin A
Retinol acetate
Physiological Effects of Drugs
Protective Agents
Anticarcinogenic Agents
Antineoplastic Agents
Adjuvants, Immunologic
Immunologic Factors