CENTAUR (NCT03127514) was a Phase 2 multicenter trial in adults with amyotrophic lateral sclerosis (ALS), encompassing both a 6-month randomized placebo-controlled phase and an open-label long-term follow-up phase. This trial included 137 participants in the U.S. and evaluated safety and efficacy of AMX0035 — a fixed-dose coformulation of 2 active compounds, sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine). The trial completed in 2019 and showed positive results.1
The primary objectives of the randomized phase of CENTAUR were to assess the safety and tolerability of AMX0035 as well as the impact of AMX0035 on disease progression, as measured by the Revised ALS Functional Rating Scale (ALSFRS-R). Additional measures that are critical to people with ALS, including muscle strength, lung vital capacity and biomarkers of neuronal degeneration, were also assessed in CENTAUR.1
The CENTAUR trial enrolled participants between 18 and 80 years old with definite ALS (as described by revised El Escorial criteria) — and within 18 months of symptom onset — and allowed edaravone and/or riluzole use.1