Stargardt disease is a rare, progressive, inherited retinal dystrophy affecting about 1 in 10,000 individuals, typically beginning in childhood or adolescence.

Progressive photoreceptor and Retinal Pigment Epithelium (RPE) degeneration in Stargardt disease results in irreversible loss of visual acuity, delayed dark adaptation, impaired color vision, and reduced contrast sensitivity, with many patients progressing to severe visual impairment or legal blindness.

Do you have patients diagnosed with Stargardt disease?

The NORTHSTAR Study is a clinical trial to evaluate a new investigational medication as a possible treatment for Stargardt disease.

Gildeuretinol (ALK-001) is being studied to evaluate its potential ability to reduce the accumulation of vitamin A dimers and slow the damage to retinal cells caused by Stargardt disease.

What is the purpose of the study?

The NORTHSTAR Study will assess how safe, effective, and tolerable oral investigational gildeuretinol may be in individuals diagnosed with Stargardt disease. Participation in the study includes nine in-person visits and scheduled phone calls by the study team, over the following.

Study duration:

Screening: Up to 45 days
Treatment period: 24 months
Follow-up: 30 days

Accumulation of toxic vitamin A dimers is a primary mechanism of Stargardt disease.

Stargardt Mechanism of Disease (MOD)

Stargardt disease is linked to the mutation of the ABCA4 gene, which plays a primary role in transporting vitamin A and its byproducts between the cells of the retina inside the eye.

In those with the condition, the retina has trouble clearing away the waste materials created as a byproduct of the visual cycle.

Oral Gildeuretinol Mechanism of Action (MOA)

Oral gildeuretinol is a new molecular entity designed to reduce the formation of toxic vitamin A dimers in the retina. Reducing the formation of toxic byproducts may potentially slow disease progression and loss of vision.

Gildeuretinol is a capsule taken once a day.

About the NORTHSTAR Study

The NORTHSTAR Study is a Phase 3, multicenter, double-masked, randomized, placebo-controlled study of the safety, tolerability, and efficacy of gildeuretinol acetate (ALK-001) on the progression of Stargardt disease.

Eligible participants will be randomized in a 1:1 ratio to receive either ALK-001 14 mg or placebo, both of which come in capsule form and are taken once daily during the 24-month treatment period.

Participants will have a follow-up study visit approximately 30 days after their last dose of ALK-001 or placebo.

Study Objectives

Primary Efficacy Objective:

To evaluate the efficacy of ALK-001 compared with placebo in slowing the progression of retinal atrophic lesion growth in participants with Stargardt disease (STGD).

Secondary Objectives:

To evaluate the efficacy of ALK-001 on visual acuity (LLVA) compared with placebo in participants with Stargardt disease (STGD).

Key Eligibility Criteria

To be eligible for the NORTHSTAR Study, participants must:

Be 8 to 45 years of age
Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy
Have provided a genetic report by any regional accredited organization that provides certified testing indicating the presence of ≥1 disease-causing mutation in the ABCA4 gene
Have at least one eye (“eligible eye[s]”) that displays a minimum of one well-demarcated area, ≥0.5 mm², of definitely decreased autofluorescence on a fundus autofluorescence image.
Have visual acuity of 20/200 or better for the eligible eye(s).

Other inclusion/exclusion criteria apply.

U.S. Trial Locations