Phase 3 Study of ALK-001 in Geographic Atrophy

Phase 3

Completed

• Conditions: Age Related Macular Degeneration; Geographic Atrophy; Atrophy, Geographic; AMD
• Interventions: Drug: Placebo oral capsule; Drug: ALK-001 oral capsule

• Registration Number: NCT03845582
• Lead Sponsor: 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.

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 Timeline

• Study First Submitted: 2019-02-15
• Study First Submitted QC: 2019-02-18
• Study First Posted: 2019-02-19
• Study Start: 2019-05-07
• Primary Completion: 2023-07-25
• Study Completion: 2024-06-30
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-11
• Last Update Posted: 2024-11-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 200

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo oral capsule	Capsule
ALK-001	ALK-001 oral capsule	Capsule

Primary Outcome Measures

Name	Time	Method
Growth rate of GA lesions, as assessed by Fundus Autofluorescence (FAF)	Baseline to 24 months

Secondary Outcome Measures

Name	Time	Method
Safety and tolerability, as assessed by evaluation of adverse events	Baseline to 24 months
Changes in Visual Acuity	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 Reading Speed	Baseline to 24 months

Trial Locations

Locations (1)

Coordinating Center; 🇺🇸 Somerville, Massachusetts, United States