September 2016
Volume 57, Issue 12
Open Access
ARVO Annual Meeting Abstract  |   September 2016
TEASE: a phase 2 clinical trial assessing the tolerability and effects of oral once-a-day ALK-001 on Stargardt disease
Author Affiliations & Notes
  • Hendrik P Scholl
    Ophthalmology, Johns Hopkins University, Baltimore, Maryland, United States
  • Syed Mahmood Shah
    Ophthalmology, Johns Hopkins University, Baltimore, Maryland, United States
  • Christine Nichols Kay
    Vitreoretinal Associates, Gainesville, Florida, United States
  • Stephen H Tsang
    Harkness Eye Institute, Columbia University Medical Center, New York, New York, United States
  • Kimberly E Stepien
    Eye Institute, Medical College of Wisconsin, Milwaukee, Wisconsin, United States
  • Paul S Bernstein
    Moran Eye Center, University of Utah, Salt Lake City, Utah, United States
  • Byron L Lam
    Bascom Palmer Eye Institute, University of Miami, Miami, Florida, United States
  • Michael B Gorin
    Jules Stein Eye Institute, University of California Los Angeles, Los Angeles, California, United States
  • Ilyas Washington
    Harkness Eye Institute, Columbia University Medical Center, New York, New York, United States
  • Leonide Saad
    Alkeus Pharmaceuticals, Boston, Massachusetts, United States
  • Footnotes
    Commercial Relationships   Hendrik Scholl, None; Syed Shah, None; Christine Kay, None; Stephen Tsang, None; Kimberly Stepien, None; Paul Bernstein, None; Byron Lam, None; Michael Gorin, None; Ilyas Washington, Alkeus Pharmaceuticals (P), Alkeus Pharmaceuticals (C); Leonide Saad, Alkeus Pharmaceuticals (E), Alkeus Pharmaceuticals (S)
  • Footnotes
    Support  None
Investigative Ophthalmology & Visual Science September 2016, Vol.57, 2685. doi:
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      Hendrik P Scholl, Syed Mahmood Shah, Christine Nichols Kay, Stephen H Tsang, Kimberly E Stepien, Paul S Bernstein, Byron L Lam, Michael B Gorin, Ilyas Washington, Leonide Saad; TEASE: a phase 2 clinical trial assessing the tolerability and effects of oral once-a-day ALK-001 on Stargardt disease. Invest. Ophthalmol. Vis. Sci. 2016;57(12):2685.

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      © ARVO (1962-2015); The Authors (2016-present)

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Purpose : Stargardt disease (STGD1) is the leading cause of inherited juvenile macular degeneration. The disease stems from mutations in ABCA4, a gene that encodes a vitamin A transport protein in the retina. Defective vitamin A transport results in accelerated formation of vitamin A dimers, thought to cause retinal degenerations including STGD1. The tolerability and effects of ALK-001 in Stargardt disease (“TEASE”) study is the first of several prospective clinical trials to test the extent to which slowing the dimerization of vitamin A prevents the progression of STGD1.

Methods : ALK-001 is a vitamin A replacement: the chemical structure of ALK-001 is similar to that of vitamin A but 3 hydrogen atoms have been exchanged by deuterium. This change prevents ALK-001 from dimerizing. However, ALK-001 keeps all known functions of vitamin A. In preclinical evaluation, ALK-001 slows the rate of vitamin A dimerization approximately 4 to 5 fold but does not affect the visual cycle. TEASE is a 24-month, multicenter, randomized, double-masked, placebo-controlled study evaluating the effects of ALK-001 in up to 50 patients diagnosed with molecularly confirmed STGD1 aged between 12 and 60 years. Study subjects may have any visual acuity to enroll and must have a lesion of well-defined decreased signal on fundus autofluorescence imaging (FAF). TEASE subjects are randomly assigned to receive one of two doses of ALK-001 (30 subjects) or placebo (20 subjects) for one year. After one year, half of the subjects receiving placebo will be randomly crossed over to receive ALK-001 for the following 12 months, while all other subjects will continue receiving their initial treatment. A Data Monitoring Committee (DMC) reviews safety and efficacy data throughout the study.

Results : Enrollment is ongoing. As of late 2015, 15 subjects have been enrolled, with the longest treatment duration of about 3 months. The median best corrected visual acuity at baseline was 63 letters (range, 26-85) in the best eye. Median age was 44 years (range, 20-59) and median disease duration 8 years (range, 1-25). Median lesion size in the best eye on FAF was 1.80 mm2 (range, 0.25-17.74).

Conclusions : The TEASE study is one of the first FDA-regulated phase 2 clinical trials assessing a novel oral compound for retinal degenerative disease secondary to vitamin A dimerization, including STGD1.

This is an abstract that was submitted for the 2016 ARVO Annual Meeting, held in Seattle, Wash., May 1-5, 2016.


TEASE trial design

TEASE trial design


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