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

January 29, 2024 updated by: Alkeus Pharmaceuticals, Inc.

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

300

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Somerville, Massachusetts, United States, 02144
        • Coordinating Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

58 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ALK-001
Capsule
Drug: ALK-001 oral capsule
Daily administration for 24 months
Other Names:
  • C20-D3-Retinyl Acetate
  • C20 Deuterated vitamin A
Placebo Comparator: Placebo
Capsule
Drug: Placebo oral capsule
Daily administration for 24 months

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alkeus Pharmaceuticals, Inc.

Investigators

  • Study Director: Leonide Saad, PhD, Alkeus Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 7, 2019

Primary Completion (Actual)

July 25, 2023

Study Completion (Estimated)

April 1, 2024

Study Registration Dates

First Submitted

February 15, 2019

First Submitted That Met QC Criteria

February 18, 2019

First Posted (Actual)

February 19, 2019

Study Record Updates

Last Update Posted (Actual)

January 30, 2024

Last Update Submitted That Met QC Criteria

January 29, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALK001-P3001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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