Don't get left behind! The modernized is coming. Check it out now.
Say goodbye to!
The new site is coming soon - go to the modernized
Working… Menu
Trial record 1 of 1 for:    alkeus | Geographic Atrophy
Previous Study | Return to List | Next Study

Phase 3 Study of ALK-001 in Geographic Atrophy (SAGA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03845582
Recruitment Status : Active, not recruiting
First Posted : February 19, 2019
Last Update Posted : February 16, 2022
Information provided by (Responsible Party):
Alkeus Pharmaceuticals, Inc.

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
Geographic Atrophy Age Related Macular Degeneration AMD Atrophy, Geographic Drug: ALK-001 oral capsule Drug: Placebo oral capsule 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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 300 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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 : August 31, 2023
Estimated Study Completion Date : September 30, 2023

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: ALK-001
Drug: ALK-001 oral capsule
Daily administration for 24 months
Other Names:
  • C20-D3-Retinyl Acetate
  • C20 Deuterated vitamin A

Placebo Comparator: Placebo
Drug: Placebo oral capsule
Daily administration for 24 months

Primary Outcome Measures :
  1. Growth rate of GA lesions, as assessed by Fundus Autofluorescence (FAF) [ Time Frame: Baseline to 24 months ]

Secondary Outcome Measures :
  1. Safety and tolerability, as assessed by evaluation of adverse events [ Time Frame: Baseline to 24 months ]
  2. Pharmacokinetics, as assessed by plasma concentrations of ALK-001 and metabolites [ Time Frame: Baseline to 24 months ]
  3. Incidence of choroidal neovascularization (CNV) [ Time Frame: Baseline to 24 months ]
  4. Changes in Visual Acuity [ Time Frame: Baseline to 24 months ]
  5. Changes in Reading Speed [ Time Frame: Baseline to 24 months ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   60 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

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 identifier (NCT number): NCT03845582

Layout table for location information
United States, Massachusetts
Coordinating Center
Somerville, Massachusetts, United States, 02144
Sponsors and Collaborators
Alkeus Pharmaceuticals, Inc.
Layout table for investigator information
Study Director: Leonide Saad, PhD Alkeus Pharmaceuticals, Inc.
Layout table for additonal information
Responsible Party: Alkeus Pharmaceuticals, Inc. Identifier: NCT03845582    
Other Study ID Numbers: ALK001-P3001
First Posted: February 19, 2019    Key Record Dates
Last Update Posted: February 16, 2022
Last Verified: February 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Geographic Atrophy
Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases
Pathological Conditions, Anatomical
Vitamin A
Retinol acetate
Physiological Effects of Drugs
Protective Agents
Anticarcinogenic Agents
Antineoplastic Agents
Adjuvants, Immunologic
Immunologic Factors