NCT05021536

Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment

A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of AMX0035 Versus Placebo for 48-week Treatment of Adult Patients With Amyotrophic Lateral Sclerosis (ALS)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 664 (actual)
• Sponsor: Amylyx Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The Phoenix Trial is a randomized double blind placebo controlled Phase III trial to evaluate the safety and efficacy of AMX0035 for treatment of ALS

Detailed description

AMX0035 is a combination therapy designed to reduce neuronal death through blockade of key cellular death pathways originating in the mitochondria and endoplasmic reticulum (ER). This clinical trial is designed to demonstrate that treatment is safe, tolerable, and able to slow decline in function as measured by the ALSFRS-R and survival over 48 week. The trial will also assess the effects of AMX0035 on slow vital capacity, quality of life and plasma biomarkers of ALS.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
OTHER	Placebo	Matching Placebo Comparator
DRUG	AMX0035	Proprietary formulation of taurursodiol and sodium phenylbutyrate

Timeline

• Start date: 2021-10-28
• Primary completion: 2024-01-19
• Completion: 2026-01-01
• First posted: 2021-08-25
• Last updated: 2024-08-14

Locations

69 sites across 12 countries: United States, Belgium, France, Germany, Ireland, Italy, Netherlands, Poland, Portugal, Spain, Sweden, United Kingdom

Regulatory

• FDA-regulated drug study