Stargardt disease is an inherited progressive retinal disease leading to irreversible vision loss.
It is the most common form of inherited juvenile macular dystrophy, and there is currently no approved treatment available.
More than 95% of Stargardt disease cases are due to variants in the ABCA4 gene.
Is Stargardt disease changing how you see the world?
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 byproducts and slow the damage to retinal cells caused by Stargardt disease.
What is the purpose of the study?
NORTHSTAR will assess how safe, effective, and tolerable oral investigational gildeuretinol may be in individuals diagnosed with Stargardt disease.
Who can participate?
Individuals who meet these criteria may be able to join the study:
- Are 8 to 45 years old
- Diagnosed with typical autosomal recessive Stargardt disease
- Genetic test results showing at least one disease-causing variant in the ABCA4 gene
There may be additional criteria to be eligible.
Accumulation of toxic vitamin A byproducts is a primary mechanism of Stargardt disease.
What causes Stargardt disease?
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.
How does the study drug work?
Oral gildeuretinol is a new investigational medication designed to reduce the formation of toxic vitamin A byproducts, also referred to as dimers, in the retina. Lowering the formation of these waste materials may potentially slow disease progression and loss of vision.
Gildeuretinol is a capsule taken once a day.
About the NORTHSTAR Study
NORTHSTAR includes an initial screening period of up to 45 days followed by a 24-month (two-year) treatment period.
Participants are randomly selected to receive either the investigational drug or a placebo during the treatment period.
- Placebo contains no active ingredients.
- Study drug and placebo are capsule form.
- Taken by mouth once a day.
- 50% chance of receiving the study drug.
- No charge for treatment in the study, and support for travel expenses may be available.
About Clinical Studies
A clinical research study (also called a clinical trial) is carefully supervised medical research to test an investigational treatment.
- Clinical studies follow specific rules to protect the rights, safety, well-being, and privacy of participants.
- The results help government agencies determine if a medication is safe and effective and whether it should be made available to patients.
- Clinical studies are the only way to develop effective new medical treatments.
Associated Retina Consultants
Benjamin Bakall, MD, PhD
Contact: Mallory Mintert
hello@doctrials.com
Retina Foundation
Karl Csaky, MD, Phd
Contact: Jessica Watson
jclark@retinafoundation.org
214-363-3911 Ext 136
Erie Retina Research
David Almeida, MD, MBA, PhD
Contact: Joseph Sala
814-422-4852
j.sala.research@protonmail.com
Vitreo Retinal Associates
Christine Kay, MD
Contact: Hailey Ahrens
352-300-8412
hailey@vra-pa.com
Recruiting
RecruitingAssociated Retina Consultants
Benjamin Bakall, MD, PhD
Contact: Mallory Mintert
hello@doctrials.com
PENNSYLVANIA
Erie, PAErie Retina Research
David Almeida, MD, MBA, PhD
Contact: Joseph Sala
814-422-4852
j.sala.research@protonmail.com
Vitreo Retinal Associates
Christine Kay, MD
Contact: Hailey Ahrens
352-300-8412
hailey@vra-pa.com
TEXAS
Dallas, TXRetina Foundation
Karl Csaky, MD, PhD
Contact: Jessica Watson
214-363-3911 Ext 136
jclark@retinafoundation.org
Not Yet Recruiting
Not Yet Recruiting
FLORIDA
Miami, FL
MICHIGAN
Grand Rapids, MI
NEW YORK
Westbury, NY
TEXAS
Bellaire, TXFort Worth, TX
San Antonio, TX
VIRGINIA
Fairfax, VANorfolk, VA
FAQs
The NORTHSTAR Study is a placebo-controlled, blinded clinical study that will assess how safe, effective, and tolerable oral investigational gildeuretinol may be in individuals diagnosed with Stargardt disease.
Individuals who meet these criteria* may be able to join the study:
- Are 8 to 45 years old
- Diagnosed with typical autosomal recessive Stargardt disease
- Genetic test results showing at least one disease-causing variant in the ABCA4 gene
*Note there may be additional criteria to be eligible for the study.
Participation in the NORTHSTAR Study includes a screening of up to 45 days, a treatment period of 24 months and follow-up of 30 days after the last dose of treatment or placebo. It includes nine in-person visits and scheduled phone calls with the study team.
Oral gildeuretinol is a new investigational medication designed to reduce the formation of toxic vitamin A byproducts, also referred to as dimers, in the retina. Lowering the formation of these waste materials may potentially slow disease progression and loss of vision.
Gildeuretinol is a capsule taken once a day.
Gildeuretinol has been studied previously in Phase 2 trials (TEASE) with over 400 patients. More information about the safety data results from TEASE-1 study can be found on the news page of the Alkeus corporate website or by visiting the clinical trials TEASE study. Talk with your eye care provider to discuss whether the NORTHSTAR Study may be right for you.
Gildeuretinol has been studied previously in multiple Phase 2 trials (TEASE) with over 400 patients. More information about the safety data results from the TEASE-1 study can be found on the news page of the Alkeus corporate website or by visiting the clinical trials TEASE study.
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