Phase 3 Study of ALK-001 in Geographic Atrophy

Age Related Macular Degeneration Clinical Trial

— SAGA  

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03845582
• Other study ID #: ALK001-P3001
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: May 7, 2019
• Est. completion date: April 1, 2024

Study information

• Verified date: January 2024
• Source: Alkeus Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 300
• Est. completion date: April 1, 2024
• Est. primary completion date: July 25, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 60 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Age Related Macular Degeneration
• AMD
• Atrophy
• Atrophy, Geographic
• Geographic Atrophy
• Macular Degeneration

Intervention

Drug:
• ALK-001 oral capsule
Daily administration for 24 months
• Placebo oral capsule
Daily administration for 24 months

Locations

Country	Name	City	State
United States	Coordinating Center	Somerville	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Alkeus Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

References & Publications (3)

Kaufman Y, Ma L, Washington I. Deuterium enrichment of vitamin A at the C20 position slows the formation of detrimental vitamin A dimers in wild-type rodents. J Biol Chem. 2011 Mar 11;286(10):7958-7965. doi: 10.1074/jbc.M110.178640. Epub 2010 Nov 12. — View Citation

Mihai DM, Jiang H, Blaner WS, Romanov A, Washington I. The retina rapidly incorporates ingested C20-D(3)-vitamin A in a swine model. Mol Vis. 2013 Jul 25;19:1677-83. Print 2013. — View Citation

Saad L, Washington I. Can Vitamin A be Improved to Prevent Blindness due to Age-Related Macular Degeneration, Stargardt Disease and Other Retinal Dystrophies? Adv Exp Med Biol. 2016;854:355-61. doi: 10.1007/978-3-319-17121-0_47. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Growth rate of GA lesions, as assessed by Fundus Autofluorescence (FAF)		Baseline to 24 months
Secondary	Safety and tolerability, as assessed by evaluation of adverse events		Baseline to 24 months
Secondary	Pharmacokinetics, as assessed by plasma concentrations of ALK-001 and metabolites		Baseline to 24 months
Secondary	Incidence of choroidal neovascularization (CNV)		Baseline to 24 months
Secondary	Changes in Visual Acuity		Baseline to 24 months
Secondary	Changes in Reading Speed		Baseline to 24 months