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
Interest of Measuring P2X4 Receptors on Blood Monocytes as a Diagnostic Marker in Amyotrophic Lateral Sclerosis: P2X4 as a Diagnostic Biomarker for ALS
Amyotrophic lateral sclerosis (ALS) is the most common form of motor neuron disease and is characterized by the degeneration of motor neurons leading to progressive paralysis and death within 3 to 5 years after diagnosis. To date, no key mechanism had been identified. Our associated laboratory has identified the P2X4 purinergic pathway that appears to be involved in the pathogenesis of ALS. Our goal is to verify these results at the human level in order to have a proof of concept of P2X4's role as a biomarker of the disease.
Translating Single-cell Vulnerability Into Novel ALS Biomarkers and Therapeutic Targets: Towards a Liquid Nerve Biopsy
The progress of ALS research and clinical practice is hampered by lack of effective biomarkers to monitor disease onset and progression. In response to this urgent need, we will integrate single-cell system biology approaches, histopathological and clinical data from precious human nerve biopsies collected from living ALS patients during the diagnostic workup and findings from innovative preclinical mouse models to unmask cell-specific molecular alterations that arise in the PNS tissue during the course of ALS pathology. This information will be used to select protein biomarkers of dysfunctional states associated with pre-manifest or early symptomatic stages of the disease, which will be further screened and validated in patient biofluids. Altogether, this project will lead to the discovery of novel, reliable and specific ALS biomarkers while providing insights into ALS mechanisms by leveraging an original "PNS perspective" on disease pathogenesis.