PHOENIX // Trial of AMX0035 for ALS Treatment

Objectives

Primary objective – Treatment period

To assess the impact of AMX0035 treatment versus placebo on disease progression over a 48-week period, based on the change in ALSFRS-R from baseline, and on survival.

Secondary objectives – Treatment period

To evaluate the impact of AMX0035 versus placebo on Slow Vital Capacity (SVC) in adult patients with ALS over a 48-week period ;

To assess patient quality of life (QoL; using patient-reported outcomes [PROs] in the 40-item ALS Assessment Questionnaire [ALSAQ-40]) during treatment with AMX0035 versus placebo in adult ALS patients for 48 weeks ;

To evaluate AMX0035 versus placebo on time to any decline in King and MiToS stages, as derived from ALSFRS-R data over a 48-week period ;

To evaluate the impact of AMX0035 versus placebo on ventilation-free survival (defined as death, tracheostomy for respiratory distress or permanent non-invasive ventilation [>22 hours per day for 7 consecutive days]) in adult patients with ALS over a 48-week period ;

To evaluate AMX0035 versus placebo on patients' health status (using EQ-5D Questionnaire Descriptive System Patient Assessments (PRO) and EQ Visual Analog Scale [EQ VAS] during treatment with AMX0035 over a 48-week period.

Secondary objectives – Follow-up period

To assess the long-term overall survival of all-cause mortality beyond the 48-week treatment period in adult ALS patients treated with AMX0035 or placebo until death or study termination.

Safety objective

To evaluate the safety and tolerability of AMX0035 versus placebo over 48 weeks in adult patients with ALS.

Find out more : https://clinicaltrials.gov/ct2/show/NCT05021536

Patients who have completed the 48-week double-blind phase have the opportunity to enter the open-label extension (OLE) phase, during which all patients receive AMX0035. During this phase, efficacy and safety will be assessed for up to 108 weeks.

Find out more : https://classic.clinicaltrials.gov/ct2/show/NCT05619783