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 (Phoenix)

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

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase III

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

Clinicaltrials.gov ID (11 digit #):

NCT05021536

Neals Affiliated?

Yes

Coordinating Center Contact Information


Amylyx Trial Operations / .(JavaScript must be enabled to view this email address) / 857-320-6222
.(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 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:

08/31/2021

Estimated Study Completion Date:

03/01/2024

Posting Last Modified Date:

01/07/2022

Date Study Added to alsconsortium.org:

11/03/2021
  • More Information

    PHOENIX Clinical Trial Website: www.amylyxalstrial.com/

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Min Vital Capacity (% predicted normal):

    55

    Time since Symptom Onset:

    <24 months

    Time since Diagnosis:

    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 andmaintained 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

  • Site Contact Information

    Barrow Neurological Institute
    Phoenix, Arizona 85013
    United States

    University of Southern California
    Los Angeles, California 90033
    United States

    University of California Irvine
    Orange, California 92868
    United States

    California Pacific Medical Center Research Institute
    San Francisco, California 94109
    United States

    University of Colorado
    Aurora, Colorado 80045
    United States

    University of Florida
    Gainesville, Florida 32068
    United States

    University of South Florida
    Tampa, Florida 33612
    United States

    Emory University
    Atlanta, Georgia 30322
    United States

    Augusta University Neuroscience Center
    Augusta, Georgia 30912
    United States

    Northwestern University
    Chicago, Illinois 60611
    United States

    Johns Hopkins University
    Baltimore, Maryland 21287
    United States

    Healey & AMG Center for ALS Research at Massachusetts General Hospital
    Boston, Massachusetts 02114
    United States

    University of Massachusetts
    Worcester, Massachusetts 01655
    United States

    University of Michigan
    Ann Arbor, Michigan 48109
    United States

    Hennepin Healthcare Research Institute
    Minneapolis, Minnesota 55415
    United States

    Washington University School of Medicine
    Saint Louis, Missouri 63110
    United States

    Somnos Clinical Research
    Lincoln, Nebraska 68510
    United States

    Rutgers University
    New Brunswick, New Jersey 08901
    United States

    Columbia University
    New York, New York 10032
    United States

    University of North Carolina at Chapel Hill
    Chapel Hill, North Carolina 27514
    United States

    Wake Forest University Health Sciences
    Winston-Salem, North Carolina 27109
    United States

    The Ohio State University
    Columbus, Ohio 43210
    United States

    Lewis Katz School of Medicine at Temple University
    Philadelphia, Pennsylvania 19140
    United States

    University of Pennsylvania
    Philadelphia, Pennsylvania 19107
    United States

    Austin Neuromuscular Center
    Austin, Texas 78756
    United States

    Texas Neurology
    Dallas, Texas 75206
    United States

    Virginia Commonwealth University
    Henrico, Virginia 23233
    United States

    Swedish Neuroscience Institute
    Seattle, Washington 98122
    United States

    University of Washington
    Seattle, Washington 98195
    United States

    Providence St. Luke's Rehabilitation Medical Center
    Spokane, Washington 99202
    United States

    University of Wisconsin-Madison
    Madison, Wisconsin 53705
    United States

    University of Puerto Rico School of Medicine
    San Juan,
    Puerto Rico