Phase 3 Study of Dexpramipexole in ALS (EMPOWER)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Biogen Idec
ClinicalTrials.gov Identifier:
NCT01281189
First received: January 20, 2011
Last updated: September 12, 2013
Last verified: August 2013
  Purpose

The purpose of this study is to determine whether dexpramipexole (150 mg twice daily) is safe and effective in the treatment of Amyotrophic Lateral Sclerosis (ALS).


Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Drug: Dexpramipexole
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study of the Safety and Efficacy of Dexpramipexole in Subjects With Amyotrophic Lateral Sclerosis

Resource links provided by NLM:


Further study details as provided by Biogen Idec:

Primary Outcome Measures:
  • A joint rank of functional outcomes adjusted for mortality. [ Time Frame: 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Time to death or respiratory insufficiency [ Time Frame: 18 months ] [ Designated as safety issue: No ]
  • Time to death [ Time Frame: 18 months ] [ Designated as safety issue: No ]
  • Respiratory decline: time to reach less than or equal to 50% of predicted upright slow vital capacity (SVC) or death [ Time Frame: 18 months ] [ Designated as safety issue: No ]
  • Change in muscle strength measurements (MSM), as determined by the overall megascore for hand-held dynamometry (HHD) [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Change in ALS-related health quality, as measured by change in the total score on the Amyotrophic Lateral Sclerosis Assessment Questionnaire-5-Item Form (ALSAQ-5) [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Population pharmacokinetics. [ Time Frame: 18 months ] [ Designated as safety issue: No ]
  • Incidence of adverse events, serious adverse events. [ Time Frame: 18 Months ] [ Designated as safety issue: Yes ]

Enrollment: 943
Study Start Date: March 2011
Study Completion Date: November 2012
Primary Completion Date: November 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Dexpramipexole Drug: Dexpramipexole
Oral tablet 150mg twice daily for up to 18 months.
Other Name: BIIB050
Placebo Comparator: Placebo Drug: Placebo
Oral tablet twice daily for up to 18 months.

Detailed Description:

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

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01281189

  Hide Study Locations
Locations
United States, Arizona
Barrow Neurological Institute - St. Joseph's Hospital
Phoenix, Arizona, United States, 85013
United States, Arkansas
University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States
United States, California
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, United States, 95817
California Pacific Medical Center
San Francisco, California, United States, 94115
United States, Connecticut
Hospital for Special Care
New Britain, Connecticut, United States, 06053
United States, Florida
Mayo Clinic - Jacksonville
Jacksonville, Florida, United States, 32224
University of Miami Miller School of Medicine
Miami, Florida, United States, 33136
University of South Florida Medical Center
Tampa, Florida, United States, 33612
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
United States, Indiana
Indiana University
Indianapolis, Indiana, United States, 46202
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States
United States, Massachusetts
Massachusetts General Hospital
Charlestown, Massachusetts, United States, 02129
United States, Michigan
St. Mary's Health Care
Grand Rapids, Michigan, United States, 49503
United States, Minnesota
Hennepin County Medical Center
Minneapolis, Minnesota, United States, 55404
Mayo Clinic - Rochester
Rochester, Minnesota, United States
United States, Missouri
Washington University School of Medicine
St Louis, Missouri, United States, 63110
United States, Nebraska
Neurology Associates, P.C.
Lincoln, Nebraska, United States, 68506
United States, Nevada
University of Nevada School of Medicine
Las Vegas, Nevada, United States, 89102
United States, New Hampshire
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, United States, 03756
United States, New York
Columbia University
New York, New York, United States, 10032
Research Foundation of the State University of New York
Syracuse, New York, United States
United States, North Carolina
Carolinas Medical Center
Charlotte, North Carolina, United States, 28207
Duke University Medical Center
Durham, North Carolina, United States
Wake Forest University
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
The Cleveland Clinic Foundation
Cleveland, Ohio, United States
Ohio State University
Columbus, Ohio, United States, 43210
United States, Oregon
Providence ALS Center
Portland, Oregon, United States, 97213
United States, Pennsylvania
Penn State Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Drexel University College of Medicine
Philadelphia, Pennsylvania, United States
ALS Center at Penn
Philadelphia, Pennsylvania, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
United States, Texas
Texas Neurology
Dallas, Texas, United States, 75214
Methodist Neurological Institute
Houston, Texas, United States, 77030
University of Texas Health Sciences Center
San Antonio, Texas, United States, 78229
United States, Utah
University of Utah
Salt Lake City, Utah, United States
United States, Virginia
University of Virginia Health System
Charlottesville, Virginia, United States, 22908
United States, Washington
University of Washington
Seattle, Washington, United States, 98195
Australia, New South Wales
Prince of Wales Hospital
Randwick, New South Wales, Australia
Westmead Hospital
Westmead, New South Wales, Australia, 2145
Australia, Queensland
Royal Brisbane and Women's Hospital
Herston, Queensland, Australia, 4029
Australia, Victoria
Calvary Health Care Bethlehem
Melbourne, Victoria, Australia, 3121
Belgium
AZ St-Lucas
Gent, Belgium, 9000
UZ Leuven
Leuven, Belgium, 3000
Canada, Alberta
Univ of Calgary / Foothills MC
Calgary, Alberta, Canada, T2V 1P9
Canada, Quebec
CHUM - Hopital Notre Dame
Montreal, Quebec, Canada, H2L 4M1
Mcgill University
Montreal, Quebec, Canada, H3A 2B4
Canada
London Health Sciences Centre
London, Canada
Sunnybrook and Women's College and Health Sciences Centre
Toronto, Canada, M4N 3M5
University of British Columbia
Vancouver, Canada
France
CHRU de Lille - Hôpital Roger Salengro
Lille, France, 59037
CHU de Limoges - Hôpital Dupuytren
Limoges, France
Centre Hospitalier La Timone
Marseille, France
CHU Gui de Chauliac
Montpellier, France, 34295
CHU de Nice - Hôpital de l'Archet 1
Nice, France
Hôpital La Pitié Salpétrière
Paris, France, 75013
Germany
Charité - Universitätsmedizin Berlin
Berlin, Germany
Bergmannsheil Gmbh
Bochum, Germany
Medizinische Hochschule Hannover (MHH)
Hannover, Germany
Universitätsklinikum Jena
Jena, Germany
University of Ulm, RKU
Ulm, Germany
Ireland
Beaumont Hospital
Dublin, Ireland, Dublin 9
Netherlands
Academisch Medisch Centrum
Amsterdam, Netherlands, 1105 AZ
UMC St. Radboud
Nijmegen, Netherlands, 6525 GA
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands, 3584 CX
Spain
Hospital Vall d'Hebron
Barcelona, Spain
Hospital Universitario de Bellvitge
Barcelona, Spain, 8907
Hospital La Paz
Madrid, Spain, 28046
Hospital Carlos III
Madrid, Spain
Sweden
Sahlgrenska Universitetssjukhuset
Göteborg, Sweden, 41345
Karolinska Universitetssjukhuset, Solna
Stockholm, Sweden, 17176
United Kingdom
Queen Elizabeth Hospital
Birmingham, United Kingdom, B15 2TH
Walton Centre for Neurology & Neurosurgery
Liverpool, United Kingdom, L9 7LJ
Kings College Hospital NHS Foundation Trust
London, United Kingdom, SE5 8AF
Newcastle University Hospital - Clinical Ageing Research Unit
Newcastle, United Kingdom, NE4 5PL
John Radcliffe Hospital
Oxford, United Kingdom
Sheffield Institute for Transnational Neuroscience
Sheffield, United Kingdom, S10 2HQ
Sponsors and Collaborators
Biogen Idec
  More Information

No publications provided

Responsible Party: Biogen Idec
ClinicalTrials.gov Identifier: NCT01281189     History of Changes
Other Study ID Numbers: 223AS302, EUDRA CT NO: 2010-022818-19
Study First Received: January 20, 2011
Last Updated: September 12, 2013
Health Authority: United States: Food and Drug Administration
Belgium: Federal Agency for Medicinal Products and Health Products
France: Agency for the Safety of drug and health products
Australia: Therapeutic Goods Administration (TGA)
Spain: Spanish Agency for Medicines and Health Products
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Ireland: Irish Medicines Board
Sweden: Medical Products Agency
Germany: Federal Institute for Drugs and Medical Devices
Canada: Health Canada
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Biogen Idec:
Amyotrophic Lateral Sclerosis
ALS
Motor Neurone Disease

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Sclerosis
Motor Neuron Disease
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Pathologic Processes
Pramipexole
Antioxidants
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protective Agents
Physiological Effects of Drugs
Antiparkinson Agents
Anti-Dyskinesia Agents
Central Nervous System Agents
Therapeutic Uses
Dopamine Agonists
Dopamine Agents
Neurotransmitter Agents

ClinicalTrials.gov processed this record on July 31, 2014