CardinALS-trial terminated after negative results
November 27, 2024
Yesterday, PTC Therapeutics announced the first results of the CardinALS study. Unfortunately, the analyses showed that on both the primary and secondary endpoints, the drug Utreloxastat shows no improving or slowing effect in people with ALS. The study has therefore been discontinued with immediate effect.
On 26 November, PTC Therapeutics announced the results of the CardinALS study. The analyses show that the drug shows no stabilizing or delaying effect in people with ALS. PTC looked at several outcome measures, including primary outcome measures such as score on ALSFRS, survival time and neurofilaments, a measure of neuronal nerve damage. Analysis of secondary outcome measures also showed no improvement. However, the study did show that taking the study drug was safe and well-tolerated by the participants. Due to these disappointing results, all study participants are being asked to stop taking the study drug immediately.
The CardinALS-study
The CardinALS trial is a phase 2-study that investigated whether the drug Utreloxastat, also known as PTC857, is effective for the treatment of ALS. It was hoped that the drug Utreloxastat could reduce oxidative stress, thus reducing nerve cell death in ALS. The study was placebo-controlled, meaning that the effect of taking the drug was compared with taking a placebo.
Reaction TRICALS
Leonard van den Berg, neurologist and co-chair of TRICALS:
Unfortunately, the results of the CardinALS study were negative. This means that we did not see the hoped-for effect when comparing the drug to placebo. We are disappointed that this study gave not the positive result we all wanted.
However, we do remain hopeful in our fight to make ALS a treatable disease. Despite the negative outcome of the CardinALS study, we can use the insights we have gained for future research. We would also like to extend our thanks to all the study participants and their families involved, without them this study could not have taken place.
