A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study of the Safety and Efficacy of Dexpramipexole in Subjects With Amyotrophic Lateral Sclerosis

Full Study Summary:

Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive, degenerative disease of motor neurons in the brain and spinal cord that leads to muscle atrophy and spasticity in limb and bulbar muscles resulting in weakness and loss of ambulation, oropharyngeal dysfunction, weight loss, and ultimately respiratory failure. The purpose of this study is to determine whether dexpramipexole (150 mg twice daily) is safe and effective in the treatment of ALS.

• Eligibility Criteria

  Gender:

  Female, Male

  Minimum Age:

  18

  Maximum Age:

  80

  Min Vital Capacity (% predicted normal):

  65

  Time since Symptom Onset:

  <24 months

  Time since Diagnosis:

  N/A

  Can participants use Riluzole?

  Yes

  
  

  Inclusion Criteria:
  
  Aged 18 to 80 years old, inclusive, on Day 1.
  Diagnosis of sporadic or familial ALS.
  Onset of first ALS symptoms within 24 months prior to Day 1.
  World Federation of Neurology El Escorial criteria are met for a possible, laboratory-supported probable, probable, or definite ALS diagnosis.
  Upright slow vital capacity (SVC) of 65% or more at screening.
  Patients taking or not taking Riluzole are eligible for this study: if a patient has never taken Riluzole, he or she is eligible; if a patient is currently taking Riluzole, he or she must have been on a stable dose for at least 60 days; if a patient has discontinued Riluzole, he or she must have stopped taking it for at least 30 days.
  Must be able to swallow tablets at the time of study entry.
  
  Exclusion Criteria:
  
  Other medically significant illness.
  Clinically significant abnormal laboratory values.
  Pregnant women or women breastfeeding.
  Prior exposure to dexpramipexole.
  Currently taking pramipexole or other dopamine agonists.
  Other protocol-defined inclusion/exclusion criteria may apply.

• Site Contact Information

  Barrow Neurological Institute - St. Joseph's Hospital
  Phoenix, Arizona 85013
  United States

  University of Arkansas for Medical Sciences
  Little Rock, Arkansas
  United States

  University of California at San Francisco - Fresno
  Fresno, California
  United States

  University of California, Irvine
  Orange, California
  United States

  University of California, Davis
  Sacramento, California 95817
  United States

  California Pacific Medical Center
  San Francisco, California 94115
  United States

  Hospital for Special Care
  New Britain, Connecticut 06053
  United States

  Mayo Clinic - Jacksonville
  Jacksonville, Florida 32224
  United States

  niversity of Miami Miller School of Medicine
  Miami, Florida 33136
  United States

  University of South Florida Medical Center
  Tampa, Florida 33612
  United States

  Emory University
  Atlanta, Georgia 30322
  United States

  Northwestern University
  Chicago, Illinois 60611
  United States

  Indiana University
  Indianapolis, Indiana 46202
  United States

  University of Iowa
  Iowa City, Iowa 52242
  United States

  University of Kansas Medical Center
  Kansas City, Kansas 66160
  United States

  Johns Hopkins University School of Medicine
  Baltimore, Maryland
  United States

  Massachusetts General Hospital
  Charlestown, Massachusetts 02129
  United States

  St. Mary's Health Care
  Grand Rapids, Michigan 49503
  United States

  Hennepin County Medical Center
  Minneapolis, Minnesota 55404
  United States

  Mayo Clinic - Rochester
  Rochester, Minnesota
  United States

  Washington University School of Medicine
  St Louis, Missouri 63110
  United States

  Neurology Associates, P.C.
  Lincoln, Nebraska 68506
  United States

  University of Nevada School of Medicine
  Las Vegas, Nevada 89102
  United States

  Dartmouth-Hitchcock Medical Center
  Lebanon, New Hampshire 03756
  United States

  Columbia University
  New York, New York 10032
  United States

  Research Foundation of the State University of New York
  Syracuse, New York
  United States

  Carolinas Medical Center
  Charlotte, North Carolina 28207
  United States

  Duke University Medical Center
  Durham, North Carolina
  United States

  Wake Forest University
  Winston-Salem, North Carolina 27157
  United States

  The Cleveland Clinic Foundation
  Cleveland, Ohio
  United States

  Ohio State University
  Columbus, Ohio 43210
  United States

  Providence ALS Center
  Portland, Oregon 97213
  United States

  Penn State Hershey Medical Center
  Hershey, Pennsylvania 17033
  United States

  ALS Center at Penn
  Philadelphia, Pennsylvania
  United States

  Drexel University College of Medicine
  Philadelphia, Pennsylvania
  United States

  University of Pittsburgh Medical Center
  Pittsburgh, Pennsylvania
  United States

  Vanderbilt University Medical Center
  Nashville, Tennessee 37232
  United States

  Texas Neurology
  Dallas, Texas 75214
  United States

  Methodist Neurological Institute
  Houston, Texas 77030
  United States

  University of Texas Health Sciences Center
  San Antonio, Texas 78229
  United States

  University of Utah
  Salt Lake City, Utah
  United States

  University of Virginia Health System
  Charlottesville, Virginia 22908
  United States

  University of Washington
  Seattle, Washington 98195
  United States

  Royal Brisbane and Women's Hospital
  Herston, Queensland, 4029
  Australia

  Prince of Wales Hospital
  Randwick, New South Wales,
  Australia

  Westmead Hospital
  Westmead, New South Wales, 2145
  Australia

• Study Results

  http://www.thelan...970221-7/abstract
  
  http://www.alscon...elease_biogen.php