What is the mechanism of action of this treatment?

Common treatments for Age-Related Macular Degeneration (AMD) often focus on reducing oxidative stress and preventing the accumulation of toxic byproducts in the retina. ALK-001, a modified form of vitamin A, aims to slow the progression of geographic atrophy by reducing the formation of toxic vitamin A dimers, which can damage retinal cells. This is crucial for AMD patients as it helps preserve vision by protecting retinal cells from degeneration. Other treatments, such as antioxidants, work by neutralizing free radicals, thereby preventing oxidative damage to the retina. These mechanisms are vital for maintaining retinal health and slowing the progression of AMD.

What side effects are associated with this type of intervention?

Common treatments for Age-Related Macular Degeneration (AMD), particularly those targeting the visual cycle like ALK-001, often include vitamin A derivatives and other pharmacological agents. Known side effects of these treatments can include dry eye, blurred vision, and potential retinal toxicity. In clinical trials, some patients have reported symptoms such as photophobia, decreased visual acuity, and ocular discomfort. It is important for patients to have regular ophthalmologic evaluations to monitor for these side effects and adjust treatment as necessary.

What prior FDA approvals exist?

The FDA has approved several treatments for Age-Related Macular Degeneration (AMD), particularly for its advanced form, known as geographic atrophy. One notable treatment is ALK-001, a modified form of vitamin A designed to slow the progression of geographic atrophy by reducing the formation of toxic vitamin A dimers in the retina. Other FDA-approved treatments for AMD include anti-VEGF (vascular endothelial growth factor) therapies such as ranibizumab (Lucentis), aflibercept (Eylea), and bevacizumab (Avastin), which work by inhibiting abnormal blood vessel growth and leakage in the retina. These treatments have significantly improved the management of wet AMD, although their mechanisms differ from that of ALK-001.

What other types of research exist?

Promising novel alternatives to ALK-001 for Age-Related Macular Degeneration (AMD) patients include: 1) Evinacumab, a monoclonal antibody targeting ANGPTL3, which has shown significant LDL-C reduction and potential cardiovascular benefits. 2) Photodynamic therapy with aminolevulinic acid (ALA) and visible blue light, which has been effective in treating actinic keratoses and may have applications in AMD. 3) Topical ophthalmic solutions like AL-8309B, which have been evaluated for their efficacy in reducing lesion enlargement in AMD. These treatments offer different mechanisms of action and potential benefits for AMD patients.

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Retinoid

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

Phase 3

Waitlist Available

Research Sponsored by Alkeus Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing a special type of vitamin A in people with vision loss due to Geographic Atrophy from age-related macular degeneration. The goal is to see if this vitamin can slow down the damage to the retina by reducing harmful clumps of vitamin A. There are no current treatments for this condition, making this study important for those affected.

Who is the study for?

This trial is for people with a condition called Geographic Atrophy (GA) that's due to dry age-related macular degeneration. Participants need to have at least one eye affected by GA. They shouldn't have any health issues that could mess with the study or stop them from following the trial procedures and staying in the study.

What is being tested?

The trial is testing ALK-001, an oral capsule, against a placebo (a pill without active medication). It's a phase 3 study where participants are randomly chosen to receive either ALK-001 or placebo, and neither they nor the researchers know who gets what—a method known as double-masking.

What are the potential side effects?

Specific side effects of ALK-001 aren't provided here, but common side effects for oral medications can include stomach upset, headache, dizziness, or allergic reactions. The safety profile will be closely monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have dry AMD causing vision loss in at least one eye.

Select...

I have vision loss in at least one eye due to dry AMD.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: ALK-001Experimental Treatment1 Intervention

Capsule

Group II: PlaceboPlacebo Group1 Intervention

Capsule

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ALK-001 oral capsule

2019

Completed Phase 3

~200

0 / 2Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Age-Related Macular Degeneration (AMD) often focus on reducing oxidative stress and preventing the accumulation of toxic byproducts in the retina. ALK-001, a modified form of vitamin A, aims to slow the progression of geographic atrophy by reducing the formation of toxic vitamin A dimers, which can damage retinal cells. This is crucial for AMD patients as it helps preserve vision by protecting retinal cells from degeneration. Other treatments, such as antioxidants, work by neutralizing free radicals, thereby preventing oxidative damage to the retina. These mechanisms are vital for maintaining retinal health and slowing the progression of AMD.

Treatment of geographic atrophy by the topical administration of OT-551: results of a phase II clinical trial.