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)

Study Purpose:

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

Study Status:

Recruiting

Disease:

Amyotrophic Lateral Sclerosis

Study Type:

Interventional

Type of Intervention:

N/A, Drug

Intervention Name:

Placebo, AMX0035

Placebo:

Yes

Phase:

Phase 3

Study Chair(s)/Principal Investigator(s):

N/A

Clinicaltrials.gov ID:

NCT05021536

Neals Affiliated?

Yes

Coordinating Center Contact Information

Amylyx Trial Operations / email hidden; JavaScript is required / 857 320 6222

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 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.

Study Sponsor:

Amylyx Pharmaceuticals Inc.

Participant Duration:

48 weeks

Estimated Enrollment:

600

Estimated Study Start Date:

10 / 28 / 2021

Estimated Study Completion Date:

03 / 01 / 2024

Posting Last Modified Date:

10 / 07 / 2022

Date Study Added to neals.org:

08 / 25 / 2021

PHOENIX Clinical Trial Website: www.amylyxalstrial.com/

Minimum Age:

18 Years

Maximum Age:

N/A

Can participants use Riluzole?

Yes

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

Barrow Neurological Institute | Active, not recruiting

Phoenix, Arizona 85013
United States

University of Southern California | Active, not recruiting

Los Angeles, California 90033
United States

University of California Irvine | Active, not recruiting

Orange, California 92868
United States

California Pacific Medical Center Research Institute | Active, not recruiting

San Francisco, California 94109
United States

University of Colorado | Active, not recruiting

Aurora, Colorado 80045
United States

University of Florida | Active, not recruiting

Gainesville, Florida 32068
United States

University of South Florida | Active, not recruiting

Tampa, Florida 33612
United States

Emory University | Active, not recruiting

Atlanta, Georgia 30322
United States

Augusta University Neuroscience Center | Active, not recruiting

Augusta, Georgia 30912
United States

Northwestern University | Active, not recruiting

Chicago, Illinois 60611
United States

Johns Hopkins University School of Medicine Outpatient Center | Active, not recruiting

Baltimore, Maryland 21287
United States

Healey & AMG Center for ALS Research at Massachusetts General Hospital | Active, not recruiting

Boston, Massachusetts 02114
United States

University of Massachusetts | Active, not recruiting

Worcester, Massachusetts 01655
United States

Hennepin Healthcare Research Institute | Active, not recruiting

Minneapolis, Minnesota 55415
United States

Washington University School of Medicine | Active, not recruiting

Saint Louis, Missouri 63110
United States

Somnos Clinical Research | Active, not recruiting

Lincoln, Nebraska 68510
United States

Rutgers University | Active, not recruiting

New Brunswick, New Jersey 08901
United States

Columbia University | Active, not recruiting

New York, New York 10032
United States

University of North Carolina at Chapel Hill | Active, not recruiting

Chapel Hill, North Carolina 27514
United States

Wake Forest University Health Sciences | Active, not recruiting

Winston-Salem, North Carolina 27109
United States

The Ohio State University | Active, not recruiting

Columbus, Ohio 43210
United States

University of Pennsylvania | Active, not recruiting

Philadelphia, Pennsylvania 19107
United States

Lewis Katz School of Medicine at Temple University | Active, not recruiting

Philadelphia, Pennsylvania 19140
United States

Austin Neuromuscular Center | Active, not recruiting

Austin, Texas 78756
United States

Texas Neurology | Active, not recruiting

Dallas, Texas 72506
United States

Virginia Commonwealth University | Active, not recruiting

Henrico, Virginia 23233
United States

Swedish Neuroscience Institute | Active, not recruiting

Seattle, Washington 98122
United States

University of Washington | Active, not recruiting

Seattle, Washington 98195
United States

University Hospitals Leuven | Recruiting

Leuven
Belgium

Hopital Gabriel Montpied Service de Neurologie | Not yet recruiting

Clermont-Ferrand
France

CHRU de Lille - Hôpital Roger Salengro | Recruiting

Lille
France

CHU de Limoges - Hôpital Dupuytren | Recruiting

Limoges
France

Hôpitaux Universitaires de Marseille Timone | Recruiting

Marseille
France

CHU de Montpellier | Recruiting

Montpellier
France

CHU Nice | Recruiting

Nice
France

Hôpital de la Salpêtrière | Recruiting

Paris
France

Le Centre Hospitalier Régional Universitaire de Tours | Recruiting

Tours
France

Hospices Civils de Lyon Hôpital Neurologique Pierre Wertheimer Cellule Mutualisée de Recherche Clinique (CMRC) | Not yet recruiting

Bron
France

Medizinische Fakultät Mannheim der Universität Heidelberg | Recruiting

Mannheim
Germany

Ulm University Medical Centre | Recruiting

Ulm
Germany

University Medical Center Rostock | Recruiting

Rostock
Germany

Charité - Universitätsmedizin Berlin | Recruiting

Berlin
Germany

Jena University Hospital | Recruiting

Jena
Germany

Hannover Medical School | Recruiting

Hannover
Germany

Uniklinikum Dresden | Recruiting

Dresden
Germany

Trinity College Dublin/Beaumont Hospital | Recruiting

Dublin
Ireland

University of Torino | Not yet recruiting

Turin
Italy

University of Padua | Recruiting

Padova
Italy

Università degli Studi della Campania Luigi Vanvitelli | Not yet recruiting

Napoli
Italy

Azienda Ospedaliero Universitaria Di Modena | Recruiting

Modena
Italy

University of Milan Medical School | Recruiting

Milan
Italy

IRCCS - Ospedale San Raffaele | Recruiting

Milan
Italy

Centro Clinico NEMO | Recruiting

Milan
Italy

Università degli Studi di Bari Aldo Moro | Recruiting

Bari
Italy

University Medical Center Utrecht | Recruiting

Utrecht
Netherlands

Centrum Medyczne Linden | Recruiting

Kraków
Poland

City Clinic Warsaw | Recruiting

Warsaw
Poland

Centro Hospitalar Universitário Lisboa-Norte | Recruiting

Lisbon
Portugal

Hospital Universitario y Politécnico La Fe | Recruiting

Valencia
Spain

Biodonostia Health Research Institute; Hospital Universitario Donostia | Not yet recruiting

San Sebastián
Spain

Hospital San Rafael | Recruiting

Madrid
Spain

Hospital Universitario de Basurto | Not yet recruiting

Bilbao
Spain

Hospital Universitari de Bellvitge-IDIBELL | Recruiting

Barcelona
Spain

Hospital del Mar | Recruiting

Barcelona
Spain

Karolinska Institutet | Recruiting

Stockholm
Sweden

Umeå University Hospital | Recruiting

Umeå
Sweden

The Walton Centre NHS Trust | Not yet recruiting

Liverpool
United Kingdom

King's College London | Not yet recruiting

London
United Kingdom

UCL Queen Square Institute of Neurology | Recruiting

London
United Kingdom

University of Plymouth | Recruiting

Plymouth
United Kingdom

Salford Royal Hospital Barnes Clinical Research Team | Not yet recruiting

Salford
United Kingdom

Sheffield Institute for Translational Neuroscience (SITraN) | Recruiting

Sheffield
United Kingdom