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 ALSStudy Status:
Not 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:
Neals Affiliated?
Yes
Coordinating Center Contact Information
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:
01 / 06 / 2023
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
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 School of Medicine Outpatient Center
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
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
University of Pennsylvania
Philadelphia, Pennsylvania
19107
United States
Lewis Katz School of Medicine at Temple University
Philadelphia, Pennsylvania
19140
United States
Austin Neuromuscular Center
Austin, Texas
78756
United States
Texas Neurology
Dallas, Texas
72506
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
University Hospitals Leuven
Leuven
Belgium
CHU de Montpellier
Montpellier
France
Le Centre Hospitalier Régional Universitaire de Tours
Tours
France
Hôpital de la Salpêtrière
Paris
France
CHU Nice
Nice
France
Hospices Civils de Lyon Hôpital Neurologique Pierre Wertheimer Cellule Mutualisée de Recherche Clinique (CMRC)
Bron
France
Hôpitaux Universitaires de Marseille Timone
Marseille
France
CHU de Limoges - Hôpital Dupuytren
Limoges
France
CHRU de Lille - Hôpital Roger Salengro
Lille
France
Hopital Gabriel Montpied Service de Neurologie
Clermont-Ferrand
France
Uniklinikum Dresden
Dresden
Germany
Hannover Medical School
Hannover
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
Charité - Universitätsmedizin Berlin
Berlin
Germany
Trinity College Dublin/Beaumont Hospital
Dublin
Ireland
Università degli Studi della Campania Luigi Vanvitelli
Napoli
Italy
University of Torino
Turin
Italy
University of Padua
Padova
Italy
University of Milan Medical School
Milan
Italy
Azienda Ospedaliero Universitaria Di Modena
Modena
Italy
Centro Clinico NEMO
Milan
Italy
Università degli Studi di Bari Aldo Moro
Bari
Italy
University Medical Center Utrecht
Utrecht
Netherlands
Centrum Medyczne Linden
Kraków
Poland
City Clinic Warsaw
Warsaw
Poland
Centro Hospitalar Universitário Lisboa-Norte
Lisbon
Portugal
Hospital Universitario y Politécnico La Fe
Valencia
Spain
Biodonostia Health Research Institute; Hospital Universitario Donostia
San Sebastián
Spain
Hospital San Rafael
Madrid
Spain
Hospital Universitari de Bellvitge-IDIBELL
Barcelona
Spain
Hospital del Mar
Barcelona
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