Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT05349721) looked at a drug called utreloxastat in people living with ALS (also known as motor neurone disease). The trial had three parts. In the first 24-week part, 222 people were assigned to utreloxastat and 114 to a placebo (a dummy treatment with no active ingredient). The main thing researchers were measuring was a combined score that took into account both survival and day-to-day functioning — using a standard ALS questionnaire called the ALSFRS-R, which rates 12 everyday activities on a scale where higher scores mean better function (maximum score of 48). The reported data shows that for the main measure — a ranking system where a higher number meant a better combined outcome of survival and functioning — the utreloxastat group scored an average rank of 172.22 and the placebo group scored 165.36, out of a possible range of 1 to 306. For the secondary measure of actual ALSFRS-R questionnaire scores at 24 weeks, the reported data shows the utreloxastat group averaged 29.4 and the placebo group averaged 31.4 (out of 48). For breathing capacity (measured as a percentage of what would be expected for a healthy person of similar age), the utreloxastat group showed a change of −14.62% and the placebo group −15.92% from the start of the trial. The reported data also shows that 174 out of 220 people in the utreloxastat group and 90 out of 114 in the placebo group experienced at least one adverse event (an untoward medical occurrence) during the trial. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 2 Motor Neurone Disease Trial, Terminated
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been diagnosed with ALS (also known as Lou Gehrig's disease), and your first symptom appeared no more than 24 months ago at the time of your first screening visit.
- Your ALS has been classified by a doctor as either "clinically definite" or "clinically probable" using a standard ALS rating system.
- Your ALS function score (a standard measure of daily abilities) is at least 34 out of 48 at the start of screening, meaning your physical function is relatively preserved.
- Your breathing capacity is at least 60% of the expected level, meaning you do not have significant breathing problems.
- Any regular medications or non-drug treatments you take (other than standard ALS medications) have been stable and unchanged for at least 14 days before screening, and you plan to keep them the same throughout the study.
- If you are female, you must have had a breast cancer imaging screening (such as a mammogram) with normal results within the 6 months before or during the screening period.
- If you are taking standard ALS medications (riluzole, edaravone, or sodium phenylbutyrate/taurursodiol), your dose has been stable and unchanged for at least 30 days before screening, and you plan to keep it the same throughout the study.
Who may not be able to join:
- You are pregnant, breastfeeding, or planning to become pregnant during the study.
- You have a significant ongoing health condition affecting organs such as the kidneys, liver, heart, lungs, or blood, or a serious psychiatric condition, that a doctor believes could put you at risk or affect the study results.
- You have any medical or mental health history that a doctor believes could make it unsafe or difficult for you to take part.
- You are currently taking part in another clinical trial using an experimental treatment, or you have done so within 30 days before screening (or within a longer period depending on how long the previous experimental drug stays in your body).
- You have previously received the experimental drug called PTC857.
- You are currently taking both edaravone and sodium phenylbutyrate/taurursodiol together as ALS treatments within 30 days before the start of screening.
- If you are female, you have any personal history of breast cancer (regardless of whether it is in remission), or any parent, sibling, or child who has had breast cancer.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
4 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Combined Assessment of Function (ALS Functional Rating Scale-Revised [ALSFRS-R]) and Survival (CAFS) Rank After 24 Weeks of Treatment (Intention-to-Treat [ITT] 1 Analysis Population)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 21 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.