Study Purpose:
The CENTAUR trial was a 2:1 (active:placebo) randomized, double-blind, placebo-controlled Phase II trial to evaluate the safety and efficacy of AMX0035 for the treatment of ALS.Study Status:
Not recruiting
Disease:
Amyotrophic Lateral Sclerosis , Motor Neuron Disease , Neuromuscular Diseases , Neurodegenerative Diseases , Spinal Cord Diseases , TDP-43 Proteinopathies , Nervous System Diseases , Central Nervous System Diseases
Study Type:
Interventional
Type of Intervention:
Drug, N/A
Intervention Name:
AMX0035, Placebo
Placebo:
Yes
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
Patrick Yeramian, MD, Amylyx Pharmaceuticals Inc., Sabrina Paganoni, MD, Massachusetts General Hospital
Clinicaltrials.gov ID:
Neals Affiliated?
Yes
Coordinating Center Contact Information
Massachusetts General Hospital, NCRI
Boston, Massachusetts, 02114 United States
Full Study Summary:
Study Sponsor:
Amylyx Pharmaceuticals Inc.
Participant Duration:
6 months
Estimated Enrollment:
137
Estimated Study Start Date:
06 / 22 / 2017
Estimated Study Completion Date:
11 / 24 / 2019
Posting Last Modified Date:
08 / 11 / 2021
Date Study Added to neals.org:
04 / 25 / 2017
Minimum Age:
18 Years
Maximum Age:
80 Years
Can participants use Riluzole?
Yes
Key Inclusion Criteria:1. Male or female, aged 18-80 years of age
2. Sporadic or familial ALS diagnosed as definite as defined by the World Federation of Neurology revised El Escorial criteria
3. Less than or equal to 18 months since ALS symptom onset
4. Capable of providing informed consent and following trial procedures
5. Slow Vital Capacity (SVC) >60% of predicted value for gender, height, and age at the Screening Visit
6. Subjects must either not take riluzole or be on a stable dose of riluzole for at least 30 days prior to the Screening Visit. Riluzole-naïve subjects are permitted in the study.
7. 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
8. 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
Key Exclusion Criteria:
1. Presence of tracheostomy
2. Exposure to PB, Taurursodiol or UDCA within 3 months prior to the Screening Visit or planning to use these medications during the course of the study
3. History of known allergy to PB or bile salts
4. Abnormal liver function defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) > 3 times the upper limit of the normal
5. Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of normal
6. Poorly controlled arterial hypertension (systolic blood pressure (SBP)>160mmHg or diastolic blood pressure (DBP)>100mmHg) at the Screening Visit
7. Pregnant women or women currently breastfeeding
8. History of cholecystectomy
9. Biliary disease which impedes biliary flow including active cholecystitis, primary biliary cirrhosis, sclerosing cholangitis, gallbladder cancer, gallbladder polyps, gangrene of the gallbladder, abscess of the gallbladder.
10. History of Class III/IV heart failure (per New York Heart Association - NYHA)
11. Severe pancreatic or intestinal disorders that may alter the enterohepatic circulation and absorption of TUDCA including biliary infections, pancreatitis and ileal resection
12. The presence of unstable psychiatric disease, cognitive impairment, dementia or substance abuse that would impair ability of the subject to provide informed consent, according to Site Investigator judgment
13. Clinically significant unstable medical condition (other than ALS) that would pose a risk to the subject if they were to participate in the study
14. Active participation in an ALS clinical trial evaluating a small molecule within 30 days of the Screening Visit
15. Exposure at any time to any biologic under investigation for the treatment of subjects with ALS (off-label use or investigational) including cell therapies, gene therapies, and monoclonal antibodies.
16. Implantation of Diaphragm Pacing System (DPS)
Barrow Neurological Institute
Phoenix, Arizona
85013
United States
UC Irvine Medical Center
Orange, California
92868
United States
Forbes Norris MDA/ALS Research Center - California Pacific Medical Center
San Francisco, California
94114
United States
University of Florida Medical Center
Gainesville, Florida
32610
United States
Carol and Frank Morsini Center for Advanced Health Care - University of South Florida
Tampa, Florida
33612
United States
Emory University Hospital
Atlanta, Georgia
30322
United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa
52242
United States
University of Kentucky Medical Center
Lexington, Kentucky
40536
United States
Ochsner Neuroscience Institute
New Orleans, Louisiana
70121
United States
Johns Hopkins Hospital
Baltimore, Maryland
21287
United States
Massachusetts General Hospital
Boston, Massachusetts
02114
United States
University of Massachusetts Memorial Medical Center
Worcester, Massachusetts
01655
United States
University of Michigan Medical Center
Ann Arbor, Michigan
48109
United States
Hennepin County Medical Center
Minneapolis, Minnesota
55415
United States
Washington University Medical Center
Saint Louis, Missouri
63110
United States
Neurology Associates P.C.
Lincoln, Nebraska
68506
United States
Mount Sinai Beth Israel
New York, New York
10003
United States
Wake Forest Baptist Medical Center
Winston-Salem, North Carolina
27157
United States
The Ohio State University Wexner Medical Center
Columbus, Ohio
43221
United States
Oregon Health & Science University
Portland, Oregon
97239
United States
The Penn Comprehensive ALS Center
Philadelphia, Pennsylvania
19107
United States
Temple University Hospital
Philadelphia, Pennsylvania
19140
United States
Texas Neurology, P.A.
Dallas, Texas
75214
United States
University of Texas Health Science Center at San Antonio
San Antonio, Texas
78229
United States
ALS Center at the Swedish Neuroscience Institute
Seattle, Washington
98122
United States