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.
Recruitment for Phase 2 of this trial is now complete.
For more information visit clinicaltrials.gov
ALS News Today: Phase 1 Safety Study Clears Path to Test Utreloxastat in ALS Patients
