Phase 3 Study of ALK-001 in Geographic Atrophy

Overview

This is a double-masked, multicenter, randomized, placebo-controlled clinical trial, evaluating the efficacy and safety of ALK-001 in participants with Geographic Atrophy (GA) secondary to age-related macular degeneration (AMD).

Up to 200 participants will receive ALK-001 while up to 100 participants will receive a placebo.

Full Title of Study: “A Phase 2/3 Multicenter, Randomized, Double-masked, Parallel-group, Placebo-controlled Study to Investigate the Safety, Pharmacokinetics, Tolerability, and Efficacy of ALK-001 in Geographic Atrophy Secondary to Age-related Macular Degeneration”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: December 31, 2021

Detailed Description

There is no treatment available for Geographic Atrophy secondary to AMD. AMD is characterized by an age-related degeneration of the retina.

The root cause for this degeneration or why some people develop AMD while others do not, is unknown. Over 20 years ago, it was hypothesized that the dimerization of vitamin A may be a significant contributor to the etiology of AMD. The eye indeed uses vitamin A as a cofactor to sense light, and a striking chemical signature of the aging and degenerating retina is the accumulation of vitamin A dimers in the retinal pigment epithelium (RPE) and the underlying Bruch's membrane. In rodent models, high levels of vitamin A dimers correlate with poor retinal health, and a variety of mechanisms have been proposed by which vitamin A dimers may induce retinal toxicity. It has been argued that these mechanisms participate in the development and progression of AMD.

ALK-001, the study drug, is a modified form of vitamin A. When taken once a day as a capsule, it replaces natural vitamin A in the body with one that forms vitamin A dimers more slowly. This study will measure the extent to which treatment with ALK-001 slows the progression of Geographic Atrophy.

Interventions

  • Drug: ALK-001 oral capsule
    • Daily administration for 24 months
  • Drug: Placebo oral capsule
    • Daily administration for 24 months

Arms, Groups and Cohorts

  • Experimental: ALK-001
    • Capsule
  • Placebo Comparator: Placebo
    • Capsule

Clinical Trial Outcome Measures

Primary Measures

  • Growth rate of GA lesions, as assessed by Fundus Autofluorescence (FAF)
    • Time Frame: Baseline to 24 months

Secondary Measures

  • Safety and tolerability, as assessed by evaluation of adverse events
    • Time Frame: Baseline to 24 months
  • Pharmacokinetics, as assessed by plasma concentrations of ALK-001 and metabolites
    • Time Frame: Baseline to 24 months
  • Incidence of choroidal neovascularization (CNV)
    • Time Frame: Baseline to 24 months
  • Changes in Visual Acuity
    • Time Frame: Baseline to 24 months
  • Changes in Reading Speed
    • Time Frame: Baseline to 24 months

Participating in This Clinical Trial

Major Inclusion Criteria:

  • At least one eye with geographic atrophy secondary to dry age-related macular degeneration (AMD)

Major Exclusion Criteria:

  • Medical condition, which may interfere with the progression of GA, prevent performance of study procedures, compliance with protocol, or continuous participation in the study

Gender Eligibility: All

Minimum Age: 60 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Alkeus Pharmaceuticals, Inc.
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Leonide Saad, PhD, Study Director, Alkeus Pharmaceuticals, Inc.
  • Overall Contact(s)
    • Leonide Saad, PhD, 800-287-2755, sagainfo@sagastudy.com

References

Kaufman Y, Ma L, Washington I. Deuterium enrichment of vitamin A at the C20 position slows the formation of detrimental vitamin A dimers in wild-type rodents. J Biol Chem. 2011 Mar 11;286(10):7958-65. doi: 10.1074/jbc.M110.178640. Epub 2010 Nov 12.

Mihai DM, Jiang H, Blaner WS, Romanov A, Washington I. The retina rapidly incorporates ingested C20-D₃-vitamin A in a swine model. Mol Vis. 2013 Jul 25;19:1677-83. Print 2013.

Saad L, Washington I. Can Vitamin A be Improved to Prevent Blindness due to Age-Related Macular Degeneration, Stargardt Disease and Other Retinal Dystrophies? Adv Exp Med Biol. 2016;854:355-61. doi: 10.1007/978-3-319-17121-0_47.

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