PHOENIX (NCT05021536) is a Phase 3, randomized, placebo-controlled trial in more than 70 sites across the U.S. and Europe evaluating the safety and efficacy for AMX0035 (sodium phenylbutyrate/taurursodiol) in people living with amyotrophic lateral sclerosis (ALS). Recruiting for this trial is ongoing.
The primary objectives of the trial will be to further assess the safety and efficacy of AMX0035 as well as the impact of AMX0035 compared to placebo on disease progression over 48 weeks based on change from baseline of ALSFRS-R and survival. Additional measures that are critical to people with ALS, including change in slow vital capacity (SVC), serial assessments of patient reported outcomes, ventilation-free survival rates, and other outcomes, will also be assessed in PHOENIX.
Enrolling approximately 600 participants with definite or clinically probable ALS within 24 months of symptom onset, and with broader inclusion criteria than CENTAUR (NCT03127514), PHOENIX will be the first trial to include a global partnership between the Treatment Research Initiative to Cure ALS (TRICALS) and Northeast ALS (NEALS) consortium. Participants who complete the 48-week trial will have the option to receive AMX0035 after the trial if permitted by each regions’ regulatory guidance. This post-trial access to AMX0035 will also be dependent on regulatory and reimbursement milestones.