Geographic AtrophyPhase 2/3SAGANCT03845582
Phase 2/3 Study of ALK-001 in Geographic Atrophy
A Phase 2/3 study testing ALK-001 oral capsule in people with Geographic Atrophy and Age Related Macular Degeneration, run by Alkeus Pharmaceuticals, Inc. at 24 sites in United States.
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). Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Geographic Atrophy, Age Related Macular Degeneration, AMD
- What is being tested?
- ALK-001 oral capsule
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 200 participants
- Where
- 24 sitesUnited States
- Study dates
- 7 May 2019to 30 Jun 2024
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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
Trial locations and contacts
24 sites in 1 country.
24 sites in 1 country
United States24 sites
Alkeus Site
Ashland, Oregon, 97520
Alkeus Site
Beverly Hills, California, 90211
Alkeus Site
Bloomington, Illinois, 61704
Alkeus Site
Chevy Chase, Maryland, 20815
Alkeus Site
Clearwater, Florida, 33761
Alkeus Site
Cleveland, Ohio, 44122
Alkeus Site
Edina, Minnesota, 55435
Alkeus Site
Fairfax, Virginia, 22031
Alkeus Site
Fort Myers, Florida, 33912
Alkeus Site
Gainesville, Florida, 32601
Alkeus Site
Grand Rapids, Michigan, 49546
Alkeus Site
Great Neck, New York, 11021
Alkeus Site
Hauppauge, New York, 11788
Alkeus Site
Houston, Texas, 77025
Alkeus Site
Lakewood, Colorado, 80228
Alkeus Site
Lexington, Kentucky, 40509
Alkeus Site
Northfield, New Jersey, 08225
Alkeus Site
Phoenix, Arizona, 85053
Alkeus Site-1
San Antonio, Texas, 78240
Alkeus Site-2
San Antonio, Texas, 78240
Alkeus Site
Silverdale, Washington, 98383
Alkeus Site
Tampa, Florida, 33609
Alkeus Site
Winter Haven, Florida, 33880
Alkeus Site
Worcester, Massachusetts, 01605
About this study
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
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.
Read the detailed description
There is no oral 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.
- Being tested
- ALK-001 oral capsule, Placebo oral capsule
- Medical subject headings
- Geographic Atrophy, Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALK-001 oral capsuleDrug
Also called C20-D3-Retinyl Acetate, C20 Deuterated vitamin A
Daily administration for 24 months
Placebo oral capsuleDrug
Daily administration for 24 months
| Study group | Role | What they receive |
|---|---|---|
| ALK-001 | Experimental | Capsule Receives: Drug: ALK-001 oral capsule |
| Placebo | Placebo comparator | Capsule Receives: Drug: Placebo oral capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Growth Rate of GA Lesions, as Assessed by Fundus Autofluorescence (FAF)
Measured: Baseline to 24 months
Secondary outcomes (2)
Number of Participants With Adverse Events
Measured: Baseline to 24 months
Changes in Visual Acuity
Changes in LLVA and BCVA
Measured: Baseline to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2019
- Study start7 May 2019
- Primary completion25 Jul 2023
- Study completion30 Jun 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Geographic AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 200 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alkeus Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03845582
- Sponsor's protocol ID
- ALK001-P3001
- Acronym
- SAGA
- Status last verified
- Nov 2024
- First submitted
- 15 Feb 2019
- Last updated
- 3 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 21075840Mihai 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.
PubMed 23914132Saad 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.
PubMed 26427432Boyer DS, Saad L, Washington I, Melamud A, Graham KB, O'Malley AE, DeBartolomeo GM, Jiang H, Harris LD, Kay CN, Ferrone PJ, Khan S; SAGA Study Team. Gildeuretinol Acetate versus Placebo for Geographic Atrophy Secondary to Age-Related Macular Degeneration: The Study of ALK-001 in Geographic Atrophy Randomized Clinical Trial. Ophthalmol Retina. 2026 Jun 3:S2468-6530(26)00268-X. doi: 10.1016/j.oret.2026.05.018. Online ahead of print.
PubMed 42242606Evans 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.
PubMed 37702300
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03845582
- Acronym / sponsor ID
- SAGA / ALK001-P3001
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 200 participants (actual)
- Lead sponsor
- Alkeus Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 7 May 2019 (actual)
- Primary completion
- 25 Jul 2023 (actual)
- Study completion
- 30 Jun 2024 (actual)
- First posted
- 19 Feb 2019 (actual)
- Results
- Posted 16 Oct 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 oral 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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ALK-001 | Experimental | Drug: ALK-001 oral capsuleCapsule |
| Placebo | Placebo comparator | Drug: Placebo oral capsuleCapsule |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALK-001 oral capsuleAlso: C20-D3-Retinyl Acetate, C20 Deuterated vitamin ADaily administration for 24 months | Drug | ALK-001 |
| Placebo oral capsuleDaily administration for 24 months | Drug | Placebo |
Primary outcome
Growth Rate of GA Lesions, as Assessed by Fundus Autofluorescence (FAF)
Time frame: Baseline to 24 months
2 secondary outcomes
Number of Participants With Adverse Events
Time frame: Baseline to 24 months
Changes in Visual Acuity
Time frame: Baseline to 24 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 21075840Kaufman 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.
- Background: PubMed 23914132Mihai 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.
- Background: PubMed 26427432Saad 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.
- Derived from this study: PubMed 42242606Boyer DS, Saad L, Washington I, Melamud A, Graham KB, O'Malley AE, DeBartolomeo GM, Jiang H, Harris LD, Kay CN, Ferrone PJ, Khan S; SAGA Study Team. Gildeuretinol Acetate versus Placebo for Geographic Atrophy Secondary to Age-Related Macular Degeneration: The Study of ALK-001 in Geographic Atrophy Randomized Clinical Trial. Ophthalmol Retina. 2026 Jun 3:S2468-6530(26)00268-X. doi: 10.1016/j.oret.2026.05.018. Online ahead of print.
- Derived from this study: PubMed 37702300Evans 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.
- Individual participant dataSharing planned: No
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RecruitingPhase 3
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RecruitingPhase 2/3
A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
Geographic Atrophy, Macular Degeneration
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
24 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing ALK-001 oral capsule in people with Geographic Atrophy and Age Related Macular Degeneration, run by Alkeus Pharmaceuticals, Inc. at 24 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Oct 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Growth Rate of GA Lesions, as Assessed by Fundus Autofluorescence (FAF) (time frame: Baseline to 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03845582, which also lists 5 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2025.