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NCT Number: NCT03845582

Phase 2/3 Study of ALK-001 in Geographic Atrophy

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.

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Key information

Conditions

Geographic Atrophy AMD Age Related Macular Degeneration Atrophy, Geographic Eye Diseases Macular Degeneration Retinal Degeneration Retinal Diseases

Age range

60 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Alkeus Site, Phoenix, Arizona, United States

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About this study

There is no oral 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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ALK-001 oral capsule

Drug

Daily administration for 24 months

Other names: C20-D3-Retinyl Acetate, C20 Deuterated vitamin A

Placebo oral capsule

Drug

Daily administration for 24 months

Primary outcomes

  1. Growth Rate of GA Lesions, as Assessed by Fundus Autofluorescence (FAF)

    Time frame: Baseline to 24 months

Secondary outcomes

  1. Number of Participants With Adverse Events

    Time frame: Baseline to 24 months

  2. Changes in Visual Acuity

    Time frame: Baseline to 24 months

    Changes in LLVA and BCVA

Sponsors and collaborators

Lead sponsor

Alkeus Pharmaceuticals, Inc.

Industry

Registry information

Official study 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

Acronym: SAGA

Important dates

Study start
2019
Primary completion
2023
Study completion
2024
First posted
Feb 19, 2019
Registry last updated
Nov 3, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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