Phase 3 Study of ALK-001 in Geographic Atrophy (SAGA)
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|ClinicalTrials.gov Identifier: NCT03845582|
Recruitment Status : Recruiting
First Posted : February 19, 2019
Last Update Posted : June 13, 2019
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|
|Geographic Atrophy Age Related Macular Degeneration AMD Atrophy, Geographic||Drug: ALK-001 oral capsule Drug: Placebo oral capsule||Phase 3|
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 Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||300 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||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|
|Actual Study Start Date :||May 7, 2019|
|Estimated Primary Completion Date :||December 31, 2021|
|Estimated Study Completion Date :||March 31, 2022|
Drug: ALK-001 oral capsule
Daily administration for 24 months
Placebo Comparator: Placebo
Drug: Placebo oral capsule
Daily administration for 24 months
- Growth rate of GA lesions, as assessed by Fundus Autofluorescence (FAF) [ Time Frame: Baseline to 24 months ]
- 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 ]
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): NCT03845582
|Contact: Leonide Saad, PhDfirstname.lastname@example.org|
|United States, Massachusetts|
|Somerville, Massachusetts, United States, 02144|
|Study Director:||Leonide Saad, PhD||Alkeus Pharmaceuticals, Inc.|