NCT03845582

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
200

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started May 2019

Longer than P75 for phase_2

Geographic Reach
1 country

24 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 15, 2019

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 19, 2019

Completed
3 months until next milestone

Study Start

First participant enrolled

May 7, 2019

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 25, 2023

Completed
11 months until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2024

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

October 16, 2025

Completed
Last Updated

November 3, 2025

Status Verified

November 1, 2024

Enrollment Period

4.2 years

First QC Date

February 15, 2019

Results QC Date

September 30, 2025

Last Update Submit

October 16, 2025

Conditions

Outcome Measures

Primary Outcomes (1)

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

    Baseline to 24 months

Secondary Outcomes (2)

  • Number of Participants With Adverse Events

    Baseline to 24 months

  • Changes in Visual Acuity

    Baseline to 24 months

Study Arms (2)

ALK-001

EXPERIMENTAL

Capsule

Drug: ALK-001 oral capsule

Placebo

PLACEBO COMPARATOR

Capsule

Drug: Placebo oral capsule

Interventions

Daily administration for 24 months

Also known as: C20-D3-Retinyl Acetate, C20 Deuterated vitamin A
ALK-001

Daily administration for 24 months

Placebo

Eligibility Criteria

Age60 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • \- At least one eye with geographic atrophy secondary to dry age-related macular degeneration (AMD)

You may not qualify if:

  • \- Medical condition, which may interfere with the progression of GA, prevent performance of study procedures, compliance with protocol, or continuous participation in the study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (24)

Alkeus Site

Phoenix, Arizona, 85053, United States

Location

Alkeus Site

Beverly Hills, California, 90211, United States

Location

Alkeus Site

Lakewood, Colorado, 80228, United States

Location

Alkeus Site

Clearwater, Florida, 33761, United States

Location

Alkeus Site

Fort Myers, Florida, 33912, United States

Location

Alkeus Site

Gainesville, Florida, 32601, United States

Location

Alkeus Site

Tampa, Florida, 33609, United States

Location

Alkeus Site

Winter Haven, Florida, 33880, United States

Location

Alkeus Site

Bloomington, Illinois, 61704, United States

Location

Alkeus Site

Lexington, Kentucky, 40509, United States

Location

Alkeus Site

Chevy Chase, Maryland, 20815, United States

Location

Alkeus Site

Worcester, Massachusetts, 01605, United States

Location

Alkeus Site

Grand Rapids, Michigan, 49546, United States

Location

Alkeus Site

Edina, Minnesota, 55435, United States

Location

Alkeus Site

Northfield, New Jersey, 08225, United States

Location

Alkeus Site

Great Neck, New York, 11021, United States

Location

Alkeus Site

Hauppauge, New York, 11788, United States

Location

Alkeus Site

Cleveland, Ohio, 44122, United States

Location

Alkeus Site

Ashland, Oregon, 97520, United States

Location

Alkeus Site

Houston, Texas, 77025, United States

Location

Alkeus Site-1

San Antonio, Texas, 78240, United States

Location

Alkeus Site-2

San Antonio, Texas, 78240, United States

Location

Alkeus Site

Fairfax, Virginia, 22031, United States

Location

Alkeus Site

Silverdale, Washington, 98383, United States

Location

Related Publications (4)

  • 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.

    PMID: 21075840BACKGROUND
  • 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.

    PMID: 23914132BACKGROUND
  • 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.

    PMID: 26427432BACKGROUND
  • Evans JR, Lawrenson JG. Antioxidant vitamin and mineral supplements for slowing the progression of age-related macular degeneration. Cochrane Database Syst Rev. 2023 Sep 13;9(9):CD000254. doi: 10.1002/14651858.CD000254.pub5.

MeSH Terms

Conditions

Geographic AtrophyMacular Degeneration

Condition Hierarchy (Ancestors)

Retinal DegenerationRetinal DiseasesEye Diseases

Results Point of Contact

Title
Meena Venugopal
Organization
Alkeus Pharmaceuticals, Inc.

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 15, 2019

First Posted

February 19, 2019

Study Start

May 7, 2019

Primary Completion

July 25, 2023

Study Completion

June 30, 2024

Last Updated

November 3, 2025

Results First Posted

October 16, 2025

Record last verified: 2024-11

Data Sharing

IPD Sharing
Will not share

Locations