What the trial was testing
The CENTAUR enrolled 137 patients with als. The study was sponsored by Amylyx Pharmaceuticals Inc. and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
People on AMX0035 lost function 25% slower than those on the dummy treatment.
The New England journal of medicine · 2020 · NCT03127514
These findings — that patients on AMX0035 had 25% slower loss of daily function over six months — were published in the The New England journal of medicine and represent the headline result of the study.
Researchers tracked outcomes across 137 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with als, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
AMX0035 (now marketed as Relyvrio) was FDA-approved for ALS in 2022 based on this and later studies. However, the manufacturer withdrew it from the market in 2024 after a larger follow-up study showed it did not extend survival or slow disease progression. Talk to your doctor about other approved ALS treatments like riluzole or edaravone.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open als trials
Evaluation of the Combined Therapy of EH-301 and N-acetylcysteine Together With Riluzole in Amyotrophic Lateral Sclerosis (ALS)
This study is designed to evaluate whether a combination of N-acetylcysteine (NAC) and EH-301 can slow down or improve symptoms of amyotrophic lateral sclerosis (ALS). Researchers will assess changes in disease progression using the ALS Functional Rating Scale-Revised (ALSFRS-R), a standard tool for measuring daily functioning in people with ALS. The main question is whether taking NAC together with EH-301 can prevent symptom worsening and possibly improve existing ALS symptoms. Participants will be randomly assigned to receive either the active combination (NAC + EH-301) or matching placebos for 6 months. During this period, they will attend regular clinic visits for evaluations, tests, and safety monitoring. After completing the initial 6-month phase, all participants may choose to join a 6-month open-label extension, where everyone receives the active treatment regardless of their original group.
UPenn Observational Research Repository on Neurodegenerative Disease
The aim of this study is to create a repository of both cross-sectional and longitudinal data, including cognitive, linguistic, imaging and biofluid biological specimens, for neurodegenerative disease research and treatment.