CARDINALS

Utreloxastat

Nerve cells that control muscle movement, called motor neurons, are lost in ALS, leading to symptoms including muscle weakness. One mechanism involved with motor neuron loss is ferroptosis. Ferroptosis is a form of cell-death due to accumulation of toxic levels of iron that results from a key enzymatic pathway that becomes dysfunctional in ALS. Utreloxastat (also known as PTC857) is an oral small molecule with a mechanism of action that targets and inhibits activity of that key enzyme and is believed to decrease nerve cell damage in people living with ALS.

Study design

If you qualify and agree to be in the study, you will be in the study for about 7 months or a little longer than a year if you decide to take part in an extension study. Participants are randomly, double-blind placed into 1 of 2 study groups:

• Group 1 takes the study drug (utreloxastat)
• Group 2 takes the placebo

Both the study drug and the placebo are liquid that is swallowed 2 times a day. The placebo looks like the study drug, but it contains no active ingredient. Two-thirds of participants will take the study drug, and one-third will take the placebo.

There are 4 parts to this study:

• Screening: Lasts about 8 weeks with at least 1 visit to the study clinic and 1 telephone call visit. You will receive tests and exams to see if you qualify to take part in the study.
• Treatment Part A: Lasts 24 weeks, with around 3 visits occurring at the study clinic and 5 over the telephone. A home health provider will come to your home up to 5 times to take vital signs and draw blood. You will take the assigned study drug or the placebo during this part.
• Treatment Part B (optional): Continues for 28 weeks with 3 visits occurring at the study clinic and 3 over the telephone. In-home vital signs and blood draws will occur 3 times. All participants who agree to continue in the study will take the study drug.
• Follow-up: All participants will have a final follow-up visit by telephone 4 weeks after they stop taking the study drug or placebo.

Can I participate in the study?

The main criteria to participate in this study are:

• You are 18-80 years old;
• Began having symptom(s) less than 2 years ago that led to your ALS diagnosis;
• Are able to swallow the liquid study drug;

Note: This list is not exhaustive, but these are the main criteria.

Registration and more information

If you are interested in participating in the CARDINALS study and fit the above profile, please contact one of the participating tricals centres below (contact details can be found by clicking on ‘read more’). They can provide you with more information and answer any questions you may have. If you experience difficulty contacting a centre, you may reach out directly to the TRICALS operational team at info@tricals.org for support in this process. More information about the study can be found at https://classic.clinicaltrials.gov/ct2/show/NCT05349721?term=NCT05349721&rank=1