MND Australia

CardinALS – PTC857 (utreloxastat)

What does PTC857/utreloxastat do?

A key mechanism that causes motor neuron loss in MND is a process called oxidative stress, that leads to cell damage. Oxidative stress increases the production of a particular enzyme called 15-LO. An increase in 15-LO then triggers several processes that are known to contribute to motor neuron death. Utreloxastat is designed to block the action of 15-LO in an attempt to reduce oxidative stress and prevent motor neuron death, therefore slowing or preventing disease progression.  


This study is intended to assess the efficacy, safety and tolerability of PTC857 in adult male and female subjects diagnosed with ALS.  The study will also look at how the body interacts with the substance and will look for any effects on biomarkers. A biomarker refers to a measurable substance within the body, such as in the blood, urine or cerebral spinal fluid. 

The CardinALS study consists of 4 periods. These are screening (8 weeks), treatment (24 weeks), long-term treatment extension (28 weeks) and a follow-up phase (4 weeks). Following completion of the LTE period, participants who enter the Continued LTE Period will receive open-label PTC857 for an additional 108 weeks.

Eligible subjects will be randomised to receive either PTC857 orally twice a day or placebo. A placebo drug contains no active ingredient. 

How can I take part in this trial?

Trial sites and eligibility criteria are listed below. In order to take part in a clinical trial, you should first speak to your neurologist or MND Clinic.  

Eligibility criteria

  • ALS with preserved function, defined as:
    1. Onset of the first symptom leading to the diagnosis of ALS ≤24 months at the time of the initial Screening Visit
    2. Revised EL Escorial criteria of either;(i) Clinically definite ALS (ii) Clinically probable ALS
  • A total ALSFRS-R score of at least 34 at the start of the Screening Period
  • No significant respiratory compromise as evidenced by slow vital capacity ≥60% at the start of the Screening Period
QLDRoyal Brisbane and Women's Hospital

Gold Coast Hospital, Southport
VICCalvary Health Care Bethlehem

Austin Health, Heidelberg

Want to know more? 

For more information visit

ALS News Today: Phase 1 Safety Study Clears Path to Test Utreloxastat in ALS Patients