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Quick Info
Status
Currently Recruiting
Phase
3
Trial Type
Interventional
Treatment Type
Drug Trials
Randomization
Not Available
Enrollment
600
Start Date
10/28/2021
Contact Information
    Contact information unknown.
Locations
Belgium, Other
University Hospitals Leuven, Leuven, Belgium
France, Other
Hospices Civils de Lyon Hôpital Neurologique Pierre Wertheimer Cellule Mutualisée de Recherche Clini, 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
Germany, Other
Charité - Universitätsmedizin Berlin, Berlin, Germany
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
Ireland, Other
Trinity College Dublin/Beaumont Hospital, Dublin, Ireland
Italy, Other
Università degli Studi di Bari Aldo Moro, Bari, Italy
Centro Clinico NEMO, Milan, Italy
IRCCS - Ospedale San Raffaele, 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, Padova, Italy
University of Torino, Turin, Italy
Netherlands, Other
University Medical Center Utrecht, Utrecht, Netherlands
Poland, Other
Centrum Medyczne Linden, Kraków, Poland
City Clinic Warsaw, Warsaw, Poland
Portugal, Other
Centro Hospitalar Universitário Lisboa-Norte, Lisbon, Portugal
Spain, Other
Hospital del Mar, Barcelona, Spain
Hospital Universitari de Bellvitge-IDIBELL, Barcelona, Spain
Hospital Universitario de Basurto, Bilbao, Spain
Hospital San Rafael, Madrid, Spain
Biodonostia Health Research Institute; Hospital Universitario Donostia, San Sebastián, Spain
Hospital Universitario y Politécnico La Fe, Valencia, Spain
Sweden, Other
Karolinska Institutet, Stockholm, Sweden
Umeå University Hospital, Umeå, Sweden
United Kingdom, Other
The Walton Centre NHS Trust, Liverpool, United Kingdom
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 Clinical Research Team, Salford, United Kingdom
Sheffield Institute for Translational Neuroscience (SITraN), Sheffield, United Kingdom
United States, Arizona
Barrow Neurological Institute, Phoenix, AZ, 85013, United States
United States, California
University of Southern California, Los Angeles, CA, 90033, United States
University of California Irvine, Orange, CA, 92868, United States
California Pacific Medical Center Research Institute, San Francisco, CA, 94109, United States
United States, Colorado
University of Colorado, Aurora, CO, 80045, United States
United States, Florida
University of Florida, Gainesville, FL, 32068, United States
University of South Florida, Tampa, FL, 33612, United States
United States, Georgia
Emory University, Atlanta, GA, 30322, United States
Augusta University Neuroscience Center, Augusta, GA, 30912, United States
United States, Illinois
Northwestern University, Chicago, IL, 60611, United States
United States, Maryland
Johns Hopkins University School of Medicine Outpatient Center, Baltimore, MD, 21287, United States
United States, Massachusetts
Healey & AMG Center for ALS Research at Massachusetts General Hospital, Boston, MA, 02114, United States
University of Massachusetts, Worcester, MA, 01655, United States
United States, Minnesota
Hennepin Healthcare Research Institute, Minneapolis, MN, 55415, United States
United States, Missouri
Washington University School of Medicine, Saint Louis, MO, 63110, United States
United States, Nebraska
Somnos Clinical Research, Lincoln, NE, 68510, United States
United States, New Jersey
Rutgers University, New Brunswick, NJ, 08901, United States
United States, New York
Columbia University, New York, NY, 10032, United States
United States, North Carolina
University of North Carolina at Chapel Hill, Chapel Hill, NC, 27514, United States
Wake Forest University Health Sciences, Winston-Salem, NC, 27109, United States
United States, Ohio
The Ohio State University, Columbus, OH, 43210, United States
United States, Pennsylvania
University of Pennsylvania, Philadelphia, PA, 19107, United States
Lewis Katz School of Medicine at Temple University, Philadelphia, PA, 19140, United States
United States, Texas
Austin Neuromuscular Center, Austin, TX, 78756, United States
Texas Neurology, Dallas, TX, 72506, United States
United States, Virginia
Virginia Commonwealth University, Henrico, VA, 23233, United States
United States, Washington
Swedish Neuroscience Institute, Seattle, WA, 98122, United States
University of Washington, Seattle, WA, 98195, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
>55%
Months Since Onset
Number of months since first symptoms of ALS.
<24 months
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
No
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
No
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Yes
Open Label
No
Update Notes
Sites added/new sites recruiting
10/7/2022
New sites recruiting
8/4/2022
New sites recruiting
6/6/2022
Sites added
5/6/2022
Sites added
4/4/2022
Study recruiting
12/1/2021
Start date updated
11/5/2021

Other Information

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
Eligibility
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
Details
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.
Collaborator(s)
  • Amylyx Pharmaceuticals Inc.
Trial Protocol as Published on Clinicaltrials.gov
NCT05021536 (First Published: 8/20/2021)