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

Description

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.

Study Start Date

August 2015

Estimated Completion Date

March 2018

Interventions

  • Drug: ALK-001
  • Drug: Placebo

Specialties

  • Ophthalmology: Retina
  • Physician Assistant: Ophthalmology

MeSH Terms

  • Eye Diseases
  • Macular Degeneration
  • Retinal Degeneration

Study ID

Alkeus Pharmaceuticals, Inc. -- ALK001-P1002

Status

Unknown

Trial ID

NCT02402660

Study Type

Interventional

Trial Phase

Phase 2

Enrollment Quota

50

Sponsor

Alkeus Pharmaceuticals, Inc.

Main

Inclusion Criteria

  • Male or female between 12 and 60 years old (inclusive)
  • 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 atrophy as imaged by fundus autofluorescence (FAF) with a minimum area of 0.5 square millimeter, confirmed by the investigator, or a minimum area of 0.1 square millimeter upon approval by the sponsor
  • All atrophy lesions of the primary study eye summed together are less than or equal to 12 square millimeter as confirmed by the investigator
  • Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) of the primary study eye is between 45 and 70 letters at screening (between 20/40 and 20/125 Snellen). Visual acuity as low as 35 letters at baseline (20/200) acceptable upon sponsor approval
  • Has at least one documented FAF photograph acquired up to 18 months prior to the day of screening or randomization. For the primary study eye, the FAF image taken at screening should show a measurably larger atrophic lesion than the historical documented FAF, as confirmed by the investigator.
  • Primary study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to permit good quality retinal imaging as assessed by the investigator
  • Healthy as judged by investigator
  • Able and willing to comply with study requirements, restrictions and instructions (contraception requirements, scheduled visits, study procedures, compliance with study drug intake, compliance with supplements restrictions, compliance with vitamin A restrictions), and is likely to complete the 24-month study as judged by the investigator
  • 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 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

Gender

Both

Ages

12 Years to 60 Years

Accepts Healthy Volunteers

No

Study Locations and Contact Information (7)

Study Location Distance Name Phone Email
Columbia University Medical Center Harkness Eye Institute - New York, New York 44.8 miles None None None
Johns Hopkins Wilmer Eye Institute - Baltimore, Maryland 219.6 miles None None None
Medical College of Wisconsin Eye Institute - Milwaukee, Wisconsin 769.3 miles None None None
Vitreoretinal Associates - Gainesville, Florida 952.8 miles None None None
University of Miami Bascom Palmer Eye Institute - Miami, Florida 1,141.0 miles None None None
University of Utah Moran Eye Institute - Salt Lake City, Utah 2,001.2 miles None None None
University of California Los Angeles Jules Stein Eye Institute - Los Angeles, California 2,499.3 miles None None None

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