SAGA: Phase 3 Study of ALK-001 in Geographic Atrophy
Study Details
Study Description
Brief Summary
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: ALK-001 Capsule |
Drug: ALK-001 oral capsule
Daily administration for 24 months
Other Names:
|
| Placebo Comparator: Placebo Capsule |
Drug: Placebo oral capsule
Daily administration for 24 months
|
Outcome Measures
Primary Outcome Measures
- Growth rate of GA lesions, as assessed by Fundus Autofluorescence (FAF) [Baseline to 24 months]
Secondary Outcome Measures
- Safety and tolerability, as assessed by evaluation of adverse events [Baseline to 24 months]
- Pharmacokinetics, as assessed by plasma concentrations of ALK-001 and metabolites [Baseline to 24 months]
- Incidence of choroidal neovascularization (CNV) [Baseline to 24 months]
- Changes in Visual Acuity [Baseline to 24 months]
- Changes in Reading Speed [Baseline to 24 months]
Eligibility Criteria
Criteria
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Coordinating Center | Somerville | Massachusetts | United States | 02144 |
Sponsors and Collaborators
- Alkeus Pharmaceuticals, Inc.
Investigators
- Study Director: Leonide Saad, PhD, Alkeus Pharmaceuticals, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
- 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):7958-7965. 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.
- ALK001-P3001