NCT03127514

AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)

Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for the Treatment of ALS

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 137 (actual)
• Sponsor: Amylyx Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The CENTAUR trial was a 2:1 (active:placebo) randomized, double-blind, placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for the 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. The trial will also assess the effects of AMX0035 on muscle strength, vital capacity, and biomarkers of ALS including markers of neuronal death and neuroinflammation.

Conditions

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Neuromuscular Diseases
• Neurodegenerative Diseases
• Spinal Cord Diseases
• TDP-43 Proteinopathies
• Nervous System Diseases
• Central Nervous System Diseases

Interventions

Type	Name	Description
DRUG	AMX0035	AMX0035
OTHER	Placebo	Matching Placebo Comparator

Timeline

• Start date: 2017-06-22
• Primary completion: 2019-09-25
• Completion: 2019-11-24
• First posted: 2017-04-25
• Last updated: 2024-05-16
• Results posted: 2021-08-11

Locations

25 sites across 1 country: United States

Regulatory

• FDA-regulated drug study