Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Amyotrophic Lateral Sclerosis

In brief

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

Key facts

Study ID

NCT05021536

Run by

Amylyx Pharmaceuticals Inc.

People needed

664

Starts

2021-10-28

Expected to finish

2026-01-01

Last updated by the study team

2024-08-14

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• 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
• Barrow Neurological Institute — Phoenix, Arizona, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.