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 (CENTAUR)
Study Purpose:
The CENTAUR trial will be 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.
Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALSStudy Type:
Interventional TrialStudy Category:
Drug TrialStudy Status:
Active, currently recruitingPhase:
Phase IIStudy Chair(s)/Principal Investigator(s):
Study Director: Patrick Yeramian, MD, Amylyx Pharmaceuticals Inc.
Principal Investigator: Sabrina Paganoni, MD, Massachusetts General Hospital
Clinicaltrials.gov ID (11 digit #):
NCT03127514Neals Affiliated?
YesCoordinating Center Contact Information
Carly Doyle / .(JavaScript must be enabled to view this email address) / 855-437-4823
.(JavaScript must be enabled to view this email address) United States
Full Study Summary:
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.
Study Sponsor:
Amylyx Pharmaceuticals Inc.Participant Duration:
24 weeks