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ACTIVE NOT RECRUITING

NCT05021536

PHASE3

Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment

Sponsor: Amylyx Pharmaceuticals Inc.

View on ClinicalTrials.gov

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

Official title: 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)

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

664

Start Date

2021-10-28

Completion Date

2026-01-01

Last Updated

2024-08-14

Healthy Volunteers

Conditions

Amyotrophic Lateral Sclerosis

Interventions

OTHER

Placebo

Matching Placebo Comparator

DRUG

AMX0035

Proprietary formulation of taurursodiol and sodium phenylbutyrate

Inclusion Criteria: * Male or female, at least 18 years of age * Diagnosis of ALS (definite or clinically probable) * Time since onset of first symptom of ALS should be \<24 months prior to randomization; * If the participant is to be treated with riluzole and/or edaravone during the course of the trial, then treatment with riluzole and/or edaravone was, at the time of the screening visit, started and maintained at a stable regimen for at least 14 days for riluzole and/or for a full treatment cycle for edaravone; * Capable of providing informed consent * Capable and willing to follow trial procedures including visits to the trial clinic and visit requirements; * Women of child bearing potential (e.g. not post-menopausal for at least one year or surgically sterile) must agree to use adequate birth control for the duration of the study and 3 months after last dose of study drug. Women must not be planning to become pregnant for the duration of the study and 3 months after last dose of study drug * Men must agree to practice contraception for the duration of the study and 3 months after last dose of study drug. Men must not plan to father a child or provide for sperm donation for the duration of the study and 3 months after last dose of study drug Exclusion Criteria: * Presence of tracheostomy or permanent assisted ventilation(PAV) * Slow Vital Capacity (SVC) less than 55% * History of known allergy to phenyl butyrate or bile salts * Abnormal liver function defined as bilirubin levels and/or aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \> 5 times the upper limit of the normal (obtained within 12 weeks from first dose) * Renal insufficiency as defined by eGFR \<60 mL/min/1.73m\^2 (obtained within 12 weeks from first dose) * Pregnant women (confirmed by a pregnancy test within 7 days of first dose) or women currently breastfeeding * Current severe biliary disease which may result in the Investigator medical judgement in biliary obstruction including for example active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gangrene of the gallbladder, abscess of the gallbladder * History of Class III/IV heart failure (per New York Heart Association - NYHA) * Participant under severe salt restriction where the added salt intake due to treatment would put the participant at risk, in the Investigator clinical judgment * Presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the participant to provide informed consent, according to Investigator judgment * Clinically significant unstable medical condition (other than ALS) (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, severe laboratory test anomaly or clinically significant electrocardiogram \[ECG\] changes) that would pose a risk to the participant if he/she were to participate in the trial, according to Investigator judgment * Previous treatment for ALS with cellular therapies or gene therapies * Currently enrolled in another trial involving use of an investigational therapy * Previous treatment with PB or taurursodiol within 30 days from Screening * Implantation of Diaphragm Pacing System (DPS) * Currently or previously treated within the last 30 days or planned exposure to any prohibited medications listed in Section 6.8 of the protocol

Locations (69)

Barrow Neurological Institute

Phoenix, Arizona, United States

University of Southern California

Los Angeles, California, United States

University of California Irvine

Orange, California, United States

California Pacific Medical Center Research Institute

San Francisco, California, United States

University of Colorado

Aurora, Colorado, United States

University of Florida

Gainesville, Florida, United States

University of South Florida

Tampa, Florida, United States

Emory University

Atlanta, Georgia, United States

Augusta University Neuroscience Center

Augusta, Georgia, United States

Northwestern University

Chicago, Illinois, United States

Johns Hopkins University School of Medicine Outpatient Center

Baltimore, Maryland, United States

Healey & AMG Center for ALS Research at Massachusetts General Hospital

Boston, Massachusetts, United States

University of Massachusetts

Worcester, Massachusetts, United States

Hennepin Healthcare Research Institute

Minneapolis, Minnesota, United States

Washington University School of Medicine

St Louis, Missouri, United States

Somnos Clinical Research

Lincoln, Nebraska, United States

Rutgers University

New Brunswick, New Jersey, United States

Columbia University

New York, New York, United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Wake Forest University Health Sciences

Winston-Salem, North Carolina, United States

The Ohio State University

Columbus, Ohio, United States

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Lewis Katz School of Medicine at Temple University

Philadelphia, Pennsylvania, United States

Austin Neuromuscular Center

Austin, Texas, United States

Texas Neurology

Dallas, Texas, United States

Virginia Commonwealth University

Henrico, Virginia, United States

Swedish Neuroscience Institute

Seattle, Washington, United States

University of Washington

Seattle, Washington, United States

University Hospitals Leuven

Leuven, Belgium

Hospices Civils de Lyon Hôpital Neurologique Pierre Wertheimer Cellule Mutualisée de Recherche Clinique (CMRC)

Bron, France

Hopital Gabriel Montpied Service de Neurologie

Clermont-Ferrand, France

CHRU de Lille - Hôpital Roger Salengro

Lille, France

CHU de Limoges - Hôpital Dupuytren

Limoges, France

Hôpitaux Universitaires de Marseille Timone

Marseille, France

CHU de Montpellier

Montpellier, France

CHU Nice

Nice, France

Hôpital de la Salpêtrière

Paris, France

Le Centre Hospitalier Régional Universitaire de Tours

Tours, France

Charité - Universitätsmedizin Berlin

Berlin, Germany

Uniklinikum Dresden

Dresden, Germany

Hannover Medical School

Hanover, Germany

Jena University Hospital

Jena, Germany

Medizinische Fakultät Mannheim der Universität Heidelberg

Mannheim, Germany

University Medical Center Rostock

Rostock, Germany

Ulm University Medical Centre

Ulm, Germany

Trinity College Dublin/Beaumont Hospital

Dublin, Ireland

Università degli Studi di Bari Aldo Moro

Bari, Italy

Centro Clinico NEMO

Milan, Italy

University of Milan Medical School

Milan, Italy

Azienda Ospedaliero Universitaria Di Modena

Modena, Italy

Università degli Studi della Campania Luigi Vanvitelli

Naples, Italy

University of Padua

Padova, Italy

University of Torino

Turin, Italy

University Medical Center Utrecht

Utrecht, Netherlands

Centrum Medyczne Linden

Krakow, Poland

City Clinic Warsaw

Warsaw, Poland

Centro Hospitalar Universitário Lisboa-Norte

Lisbon, Portugal

Hospital del Mar

Barcelona, Spain

Hospital Universitari de Bellvitge-IDIBELL

Barcelona, Spain

Hospital San Rafael

Madrid, Spain

Biodonostia Health Research Institute; Hospital Universitario Donostia

San Sebastián, Spain

Hospital Universitario y Politécnico La Fe

Valencia, Spain

Karolinska Institutet

Stockholm, Sweden

Umeå University Hospital

Umeå, Sweden

King's College London

London, United Kingdom

UCL Queen Square Institute of Neurology

London, United Kingdom

University of Plymouth

Plymouth, United Kingdom

Salford Royal Hospital Barnes

Salford, United Kingdom

Sheffield Institute for Translational Neuroscience (SITraN)

Sheffield, United Kingdom