Phase 3 Study of Dexpramipexole in ALS
EMPOWER
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study of the Safety and Efficacy of Dexpramipexole in Subjects With Amyotrophic Lateral Sclerosis
2 other identifiers
interventional
942
11 countries
82
Brief Summary
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_3
Started Mar 2011
82 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 20, 2011
CompletedFirst Posted
Study publicly available on registry
January 21, 2011
CompletedStudy Start
First participant enrolled
March 1, 2011
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
November 1, 2012
CompletedResults Posted
Study results publicly available
June 7, 2021
CompletedJune 7, 2021
May 1, 2021
1.7 years
January 20, 2011
December 4, 2020
May 10, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
Composite Assessment of Function and Survival (CAFS) at 12 Months
The Composite Assessment of Function and Survival (CAFS) is a between-group comparison of a single ranked clinical outcome based on (1) the change from baseline in ALS Functional Rating Scale-Revised (ALSFRS-R) score and (2) time to death. Each subject is ranked according to time-to-death (earlier deaths ranked lower than later deaths). Subjects who survive are ranked more favorably than subjects who died. Among the survivors, subjects are ranked according to change in ALSFRS-R (greater worsening of ALSFRS-R is ranked lower than less worsening or an improvement in ALSFRS-R). The ranked scores range from 001 to 941 (the number of subjects in the Efficacy Population) with larger rank score numbers associated with a better outcome. The ranks were analyzed using an ANCOVA model, which includes treatment as a fixed effect and adjusts for baseline ALSFRS-R score, duration of symptoms, site of onset, and use of riluzole. The least square mean rank score is presented for each treatment group.
12 months
Death up to 12 Months (CAFs Individual Component)
The longest duration of follow-up for this time to the death analysis was 12 months. In the study, subjects were followed for 12-18 months.
12 months
Change From Baseline in ALSFRS-R at 12 Months (CAFs Individual Component)
The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score of 48, with higher scores representing better function.
12 months
Secondary Outcomes (3)
Death or Respiratory Insufficiency (DRI) up to Month 18
18 months
Death up to 18 Months
18 months
≤50% Predicted Upright Slow Vital Capacity (SVC) or Died up to 18 Months
18 months
Study Arms (2)
Dexpramipexole
EXPERIMENTALPlacebo
PLACEBO COMPARATORInterventions
Oral tablet 150mg twice daily for up to 18 months.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (82)
Barrow Neurological Institute - St. Joseph's Hospital
Phoenix, Arizona, 85013, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205, United States
University of California at San Francisco - Fresno
Fresno, California, 93701, United States
University of California, Irvine
Orange, California, 92868, 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
University 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, 21287, 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, 55905, 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, 12201, United States
Carolinas Medical Center
Charlotte, North Carolina, 28207, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
Wake Forest University
Winston-Salem, North Carolina, 27157, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195, 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, 19107, United States
Drexel University College of Medicine
Philadelphia, Pennsylvania, 19129, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15213, 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, 84132, United States
University of Virginia Health System
Charlottesville, Virginia, 22908, United States
University of Washington
Seattle, Washington, 98195, United States
Prince of Wales Hospital
Randwick, New South Wales, Australia
Westmead Hospital
Westmead, New South Wales, 2145, Australia
Royal Brisbane and Women's Hospital
Herston, Queensland, 4029, Australia
Calvary Health Care Bethlehem
Melbourne, Victoria, 3121, Australia
AZ St-Lucas
Ghent, 9000, Belgium
UZ Leuven
Leuven, 3000, Belgium
Univ of Calgary / Foothills MC
Calgary, Alberta, T2V 1P9, Canada
CHUM - Hopital Notre Dame
Montreal, Quebec, H2L 4M1, Canada
Mcgill University
Montreal, Quebec, H3A 2B4, Canada
London Health Sciences Centre
London, Canada
Sunnybrook and Women's College and Health Sciences Centre
Toronto, M4N 3M5, Canada
University of British Columbia
Vancouver, Canada
CHRU de Lille - Hôpital Roger Salengro
Lille, 59037, France
CHU de Limoges - Hôpital Dupuytren
Limoges, France
Centre Hospitalier La Timone
Marseille, France
CHU Gui de Chauliac
Montpellier, 34295, France
CHU de Nice - Hôpital de l'Archet 1
Nice, France
Hôpital La Pitié Salpétrière
Paris, 75013, France
Charité - Universitätsmedizin Berlin
Berlin, Germany
Bergmannsheil Gmbh
Bochum, Germany
Medizinische Hochschule Hannover (MHH)
Hanover, Germany
Universitätsklinikum Jena
Jena, Germany
University of Ulm, RKU
Ulm, Germany
Beaumont Hospital
Dublin, Dublin 9, Ireland
Academisch Medisch Centrum
Amsterdam, 1105 AZ, Netherlands
UMC St. Radboud
Nijmegen, 6525 GA, Netherlands
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
Hospital Universitario de Bellvitge
Barcelona, 8907, Spain
Hospital Vall d'Hebron
Barcelona, Spain
Hospital La Paz
Madrid, 28046, Spain
Hospital Carlos III
Madrid, Spain
Sahlgrenska Universitetssjukhuset
Gothenburg, 41345, Sweden
Karolinska Universitetssjukhuset, Solna
Stockholm, 17176, Sweden
Queen Elizabeth Hospital
Birmingham, B15 2TH, United Kingdom
Walton Centre for Neurology & Neurosurgery
Liverpool, L9 7LJ, United Kingdom
Kings College Hospital NHS Foundation Trust
London, SE5 8AF, United Kingdom
Newcastle University Hospital - Clinical Ageing Research Unit
Newcastle, NE4 5PL, United Kingdom
John Radcliffe Hospital
Oxford, United Kingdom
Sheffield Institute for Transnational Neuroscience
Sheffield, S10 2HQ, United Kingdom
Related Publications (1)
Cudkowicz ME, van den Berg LH, Shefner JM, Mitsumoto H, Mora JS, Ludolph A, Hardiman O, Bozik ME, Ingersoll EW, Archibald D, Meyers AL, Dong Y, Farwell WR, Kerr DA; EMPOWER investigators. Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial. Lancet Neurol. 2013 Nov;12(11):1059-67. doi: 10.1016/S1474-4422(13)70221-7. Epub 2013 Sep 23.
PMID: 24067398DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Head of Regulatory
- Organization
- Knopp Biosciences
Study Officials
- PRINCIPAL INVESTIGATOR
Merit Cudkowicz, MD, MSc
Professor of Neurology of the Harvard Medical School
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 20, 2011
First Posted
January 21, 2011
Study Start
March 1, 2011
Primary Completion
November 1, 2012
Study Completion
November 1, 2012
Last Updated
June 7, 2021
Results First Posted
June 7, 2021
Record last verified: 2021-05