Evaluation of the Safety, Tolerability, Efficacy and Activity of AMX0035, a Fixed Combination of Phenylbutyrate (PB) and Tauroursodeoxycholic Acid (TUDCA), for the Treatment of ALS (CENTAUR)

Full Study Summary:

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.

• More Information

  The lead Principal Investigator for the AMX0035 trial, Sabrina Paganoni, MD, PhD, Massachusetts General Hospital, has led three webinars on the CENTAUR trial. Click the webinar recording links below for more information about the study and how to enroll.

  Click here to watch the January 10, 2018 webinar recording.

  Click here to watch the May 7, 2018 webinar recording.

  Click here to watch the September 5, 2018 webinar recording.

• Eligibility Criteria

  Gender:

  Female, Male

  Minimum Age:

  18

  Maximum Age:

  80

  Min Vital Capacity (% predicted normal):

  60

  Time since Symptom Onset:

  <18 months

  Time since Diagnosis:

  N/A

  Can participants use Riluzole?

  Yes

  
  

  Key Inclusion Criteria:
  Male or female, aged 18-80 years of age
  Sporadic or familial ALS diagnosed as definite as defined by the World Federation of Neurology revised El Escorial criteria
  Less than or equal to 18 months since ALS symptom onset
  Capable of providing informed consent and following trial procedures
  Slow Vital Capacity (SVC) >60% of predicted value for gender, height, and age at the Screening Visit
  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.
  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

  Key Exclusion Criteria:
  Presence of tracheostomy
  Exposure to PB, TUDCA or UDCA within 3 months prior to the Screening Visit or planning to use these medications during the course of the study
  History of known allergy to PB or bile salts
  Abnormal liver function defined as AST and/or ALT > 3 times the upper limit of the normal
  Renal insufficiency as defined by a serum creatinine > 1.5 times the upper limit of normal
  Poorly controlled arterial hypertension (SBP>160mmHg or DBP>100mmHg) at the Screening Visit
  Pregnant women or women currently breastfeeding
  History of cholecystectomy
  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.
  History of Class III/IV heart failure (per New York Heart Association - NYHA)
  Severe pancreatic or intestinal disorders that may alter the enterohepatic circulation and absorption of TUDCA including biliary infections, pancreatitis and ileal resection
  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
  Clinically significant unstable medical condition (other than ALS) that would pose a risk to the subject if they were to participate in the study
  Active participation in an ALS clinical trial evaluating a small molecule within 30 days of the Screening Visit
  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.
  Implantation of Diaphragm Pacing System (DPS)

• Site Contact Information

  Barrow Neurological Institute
  Nicole Turcotte / .(JavaScript must be enabled to view this email address) / 602-406-4775
  Phoenix, Arizona 85013
  United States

  UC Irvine Medical Center
  Veena Mathew / .(JavaScript must be enabled to view this email address) / 714-456-2864
  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
  Jennifer Steshyn / .(JavaScript must be enabled to view this email address) / 352-273-9022
  Gainesville, Florida 32610
  United States

  Carol and Frank Morsini Center for Advanced Health Care - University of South Florida
  Brittany Harvey / .(JavaScript must be enabled to view this email address) / 813-974-9413
  Tampa, Florida 33612
  United States

  Emory University
  Janet Brantley / .(JavaScript must be enabled to view this email address) / 404-727-1673
  Atlanta, Georgia 30322
  United States

  University of Iowa Hospitals and Clinics
  Jen Sieren / .(JavaScript must be enabled to view this email address) / 319-356-8744
  Iowa City, Iowa 52242
  United States

  University of Kentucky Medical Center
  Meha Joshi / .(JavaScript must be enabled to view this email address) / 859-218-5046
  Lexington, Kentucky 40536
  United States

  Ochsner Neuroscience Institute
  Ashley Laroche / .(JavaScript must be enabled to view this email address) / 504-703-0755
  New Orleans, Louisiana 70121
  United States

  Johns Hopkins Hospital
  Kristen Riley / .(JavaScript must be enabled to view this email address) / 410-955-9036
  Baltimore, Maryland 21287
  United States

  Massachusetts General Hospital
  Aileen Shaughnessy / .(JavaScript must be enabled to view this email address) / 617-643-0801
  Boston, Massachusetts 02114
  United States

  University of Massachusetts Memorial Medical Center
  Diane McKenna-Yasek, RN, BSN / .(JavaScript must be enabled to view this email address) / 508-856-4697
  Worcester, Massachusetts 01655
  United States

  University of Michigan Medical Center
  Jayna Duell / .(JavaScript must be enabled to view this email address) / 734-763-9037
  Ann Arbor, Michigan 48109
  United States

  Hennepin County Medical Center
  Cindy Rohde / .(JavaScript must be enabled to view this email address) / 612-873-2607
  Twin Cities
  Minneapolis, Minnesota 55415
  United States

  Washington University Medical Center
  Jennifer Jockel-Balsarotti / .(JavaScript must be enabled to view this email address) / 314-362-8624
  Saint Louis, Missouri 63110
  United States

  Neurology Associates P.C.
  Desi Eschiti / .(JavaScript must be enabled to view this email address) / 402-486-3430
  Lincoln, Nebraska 68506
  United States

  Mount Sinai Beth Israel
  Emily Ripps / .(JavaScript must be enabled to view this email address) / 212-844-6188
  New York, New York 10003
  United States

  Wake Forest Baptist Medical Center
  Mozhdeh Marandi / .(JavaScript must be enabled to view this email address) / 336-713-8577
  Winston-Salem, North Carolina 27157
  United States

  The Ohio State University Wexner Medical Center
  Ifeanyi Okoh / .(JavaScript must be enabled to view this email address) / 614-688-7837
  Columbus, Ohio 43221
  United States

  Oregon Health & Science University
  Diana Dimitrova / .(JavaScript must be enabled to view this email address) / 503-494-7269
  Portland, Oregon 97239
  United States

  The Penn Comprehensive ALS Center
  Kelly Almasy / .(JavaScript must be enabled to view this email address) / 215-829-5041
  Philadelphia, Pennsylvania 19107
  United States

  Temple University Hospital
  Kathleen Hatala / .(JavaScript must be enabled to view this email address) / 215-707-4171
  Philadelphia, Pennsylvania 19140
  United States

  Texas Neurology, P.A.
  Todd Morgan / .(JavaScript must be enabled to view this email address) / 214-827-3610, ext. 228
  Dallas, Texas 75214
  United States

  University of Texas Health Science Center at San Antonio
  Pamela Kittrell, RN, MSN, CCRC / .(JavaScript must be enabled to view this email address) / 210-450-0524
  San Antonio, Texas 78229
  United States

  ALS Center at the Swedish Neuroscience Institute
  Lindsey Maassel / .(JavaScript must be enabled to view this email address) / 206-320-7121
  Seattle, Washington 98122
  United States