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Quick Info
Status
This study has been completed
Phase
2/ 3
Trial Type
Randomized, Parallel Assignment
Treatment Type
Drug: AMX0035
Randomization
Unknown
Enrollment
137
Start Date
6/22/2017
Contact Information
Locations
United States, Arizona
Barrow Neurological Institute, Phoenix, AZ, 85013, United States
United States, California
UC Irvine Medical Center, Orange, CA, 92868, United States
Forbes Norris MDA/ALS Research Center - California Pacific Medical Center, San Francisco, CA, 94114, United States
United States, Florida
University of Florida Medical Center, Gainesville, FL, 32610, United States
Carol and Frank Morsini Center for Advanced Health Care - University of South Florida, Tampa, FL, 33612, United States
United States, Georgia
Emory University Hospital, Atlanta, GA, 30322, United States
United States, Iowa
University of Iowa Hospitals and Clinics, Iowa City, IA, 52242, United States
United States, Kentucky
University of Kentucky Medical Center, Lexington, KY, 40536, United States
United States, Louisiana
Ochsner Neuroscience Institute, New Orleans, LA, 70121, United States
United States, Maryland
Johns Hopkins Hospital, Baltimore, MD, 21287, United States
United States, Massachusetts
Massachusetts General Hospital, Boston, MA, 02114, United States
University of Massachusetts Memorial Medical Center, Worcester, MA, 01655, United States
United States, Michigan
University of Michigan Medical Center, Ann Arbor, MI, 48109, United States
United States, Minnesota
Hennepin County Medical Center, Minneapolis, MN, 55415, United States
United States, Missouri
Washington University Medical Center, Saint Louis, MO, 63110, United States
United States, Nebraska
Neurology Associates P.C., Lincoln, NE, 68506, United States
United States, New York
Mount Sinai Beth Israel, New York, NY, 10003, United States
United States, North Carolina
Wake Forest Baptist Medical Center, Winston-Salem, NC, 27157, United States
United States, Ohio
The Ohio State University Wexner Medical Center, Columbus, OH, 43221, United States
United States, Oregon
Oregon Health & Science University, Portland, OR, 97239, United States
United States, Pennsylvania
The Penn Comprehensive ALS Center, Philadelphia, PA, 19107, United States
Temple University Hospital, Philadelphia, PA, 19140, United States
United States, Texas
Texas Neurology, P.A., Dallas, TX, 75214, United States
University of Texas Health Science Center at San Antonio, San Antonio, TX, 78229, United States
United States, Washington
ALS Center at the Swedish Neuroscience Institute, Seattle, WA, 98122, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
(SVC) >60%
Months Since Onset
Number of months since first symptoms of ALS.
less/equal to 18 months
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
not specified
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
Unknown
Update Notes
Changes to drug names
11/3/2020
No significant updates.
5/29/2020
Trial has been completed
12/18/2019
No significant updates
10/10/2019
No significant updates
9/23/2019
Recruitment status updated
3/11/2019
Contact info updated
2/19/2019
No significant updates
11/10/2018
No significant updates
9/25/2018
Changed to Emily as Main Contact
6/5/2018
Recruitment at location updated
3/23/2018
Recruitment at locations updated
1/31/2018
Recruitment status updated
1/3/2018
12/20/2017
Recruitment status updated
12/7/2017
Recruitment status updated
11/30/2017
Recruitment status updated
11/20/2017
Recruitment status updated
11/9/2017
Recruitment status updated
10/24/2017
Recruitment status updated
10/11/2017
Recruitment status updated
9/27/2017
Recruitment status updated
9/8/2017
Recruitment status at location updated
8/17/2017
Recruitment status at location updated
8/7/2017
Recruitment status updated
7/12/2017
No significant updates
6/19/2017
Recruitment status updated
6/13/2017
Contact info updated
5/31/2017
Trial added
4/26/2017

Other Information

Purpose
The CENTAUR trial will be 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.
Eligibility
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 AST and/or 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 (SBP>160mmHg or 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)
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. The trial will also assess the effects of AMX0035 on muscle strength, vital capacity, and biomarkers of ALS including markers of neuronal death and neuroinflammation.
Trial Protocol as Published on Clinicaltrials.gov
NCT03127514 (First Published: 4/12/2017)