Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment
NCT ID: NCT05021536
Last Updated: 2024-08-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
664 participants
INTERVENTIONAL
2021-10-28
2026-01-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating
Placebo
Matching Placebo Comparator
AMX0035
Placebo administered by mouth or via feeding tube for 48 weeks: once daily for first 3 weeks and then twice daily for remainder of study if participant tolerating
AMX0035
Proprietary formulation of taurursodiol and sodium phenylbutyrate
Interventions
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Placebo
Matching Placebo Comparator
AMX0035
Proprietary formulation of taurursodiol and sodium phenylbutyrate
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
18 Years
ALL
No
Sponsors
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Amylyx Pharmaceuticals Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Amylyx Study Director
Role: STUDY_DIRECTOR
Amylyx Pharmaceuticals
Locations
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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
Napoli, , Italy
University of Padua
Padua, , 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
Countries
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Other Identifiers
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A35-004
Identifier Type: -
Identifier Source: org_study_id
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