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Amyotrophic Lateral SclerosisPhase 3EMPOWERNCT01281189

Phase 3 Study of Dexpramipexole in ALS

A Phase 3 study testing Dexpramipexole in people with Amyotrophic Lateral Sclerosis, run by Knopp Biosciences at 82 sites in 11 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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). Read more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
Dexpramipexole
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
942 participants
Where
82 sitesin 11 countries
Study dates
Mar 2011to Nov 2012
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

82 sites in 11 countries.

82 sites in 11 countries

United States43 sites

  • Emory University

    Atlanta, Georgia, 30322

  • Johns Hopkins University School of Medicine

    Baltimore, Maryland, 21287

  • Massachusetts General Hospital

    Charlestown, Massachusetts, 02129

  • Carolinas Medical Center

    Charlotte, North Carolina, 28207

  • University of Virginia Health System

    Charlottesville, Virginia, 22908

  • Northwestern University

    Chicago, Illinois, 60611

  • The Cleveland Clinic Foundation

    Cleveland, Ohio, 44195

  • Ohio State University

    Columbus, Ohio, 43210

  • Texas Neurology

    Dallas, Texas, 75214

  • Duke University Medical Center

    Durham, North Carolina, 27705

  • University of California at San Francisco - Fresno

    Fresno, California, 93701

  • St. Mary's Health Care

    Grand Rapids, Michigan, 49503

  • Penn State Hershey Medical Center

    Hershey, Pennsylvania, 17033

  • Methodist Neurological Institute

    Houston, Texas, 77030

  • Indiana University

    Indianapolis, Indiana, 46202

  • University of Iowa

    Iowa City, Iowa, 52242

  • Mayo Clinic - Jacksonville

    Jacksonville, Florida, 32224

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

  • University of Nevada School of Medicine

    Las Vegas, Nevada, 89102

  • Dartmouth-Hitchcock Medical Center

    Lebanon, New Hampshire, 03756

  • Neurology Associates, P.C.

    Lincoln, Nebraska, 68506

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205

  • University of Miami Miller School of Medicine

    Miami, Florida, 33136

  • Hennepin County Medical Center

    Minneapolis, Minnesota, 55404

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Hospital for Special Care

    New Britain, Connecticut, 06053

  • Columbia University

    New York, New York, 10032

  • University of California, Irvine

    Orange, California, 92868

  • ALS Center at Penn

    Philadelphia, Pennsylvania, 19107

  • Drexel University College of Medicine

    Philadelphia, Pennsylvania, 19129

  • Barrow Neurological Institute - St. Joseph's Hospital

    Phoenix, Arizona, 85013

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213

  • Providence ALS Center

    Portland, Oregon, 97213

  • Mayo Clinic - Rochester

    Rochester, Minnesota, 55905

  • University of California, Davis

    Sacramento, California, 95817

  • University of Utah

    Salt Lake City, Utah, 84132

  • University of Texas Health Sciences Center

    San Antonio, Texas, 78229

  • California Pacific Medical Center

    San Francisco, California, 94115

  • University of Washington

    Seattle, Washington, 98195

  • Washington University School of Medicine

    St Louis, Missouri, 63110

  • Research Foundation of the State University of New York

    Syracuse, New York, 12201

  • University of South Florida Medical Center

    Tampa, Florida, 33612

  • Wake Forest University

    Winston-Salem, North Carolina, 27157

Other trials in United States

Canada6 sites

  • Univ of Calgary / Foothills MC

    Calgary, Alberta, T2V 1P9

  • London Health Sciences Centre

    London

  • CHUM - Hopital Notre Dame

    Montreal, Quebec, H2L 4M1

  • Mcgill University

    Montreal, Quebec, H3A 2B4

  • Sunnybrook and Women's College and Health Sciences Centre

    Toronto, M4N 3M5

  • University of British Columbia

    Vancouver

Other trials in Canada

France6 sites

  • CHRU de Lille - Hôpital Roger Salengro

    Lille, 59037

  • CHU de Limoges - Hôpital Dupuytren

    Limoges

  • Centre Hospitalier La Timone

    Marseille

  • CHU Gui de Chauliac

    Montpellier, 34295

  • CHU de Nice - Hôpital de l'Archet 1

    Nice

  • Hôpital La Pitié Salpétrière

    Paris, 75013

Other trials in France

United Kingdom6 sites

  • Queen Elizabeth Hospital

    Birmingham, B15 2TH

  • Walton Centre for Neurology & Neurosurgery

    Liverpool, L9 7LJ

  • Kings College Hospital NHS Foundation Trust

    London, SE5 8AF

  • Newcastle University Hospital - Clinical Ageing Research Unit

    Newcastle, NE4 5PL

  • John Radcliffe Hospital

    Oxford

  • Sheffield Institute for Transnational Neuroscience

    Sheffield, S10 2HQ

Other trials in United Kingdom

Germany5 sites

  • Charité - Universitätsmedizin Berlin

    Berlin

  • Bergmannsheil Gmbh

    Bochum

  • Medizinische Hochschule Hannover (MHH)

    Hanover

  • Universitätsklinikum Jena

    Jena

  • University of Ulm, RKU

    Ulm

Other trials in Germany

Australia4 sites

  • Royal Brisbane and Women's Hospital

    Herston, Queensland, 4029

  • Calvary Health Care Bethlehem

    Melbourne, Victoria, 3121

  • Prince of Wales Hospital

    Randwick, New South Wales

  • Westmead Hospital

    Westmead, New South Wales, 2145

Other trials in Australia

Spain4 sites

  • Hospital Universitario de Bellvitge

    Barcelona, 8907

  • Hospital Vall d'Hebron

    Barcelona

  • Hospital La Paz

    Madrid, 28046

  • Hospital Carlos III

    Madrid

Other trials in Spain

Netherlands3 sites

  • Academisch Medisch Centrum

    Amsterdam, 1105 AZ

  • UMC St. Radboud

    Nijmegen, 6525 GA

  • Universitair Medisch Centrum Utrecht

    Utrecht, 3584 CX

Other trials in Netherlands

Belgium2 sites

  • AZ St-Lucas

    Ghent, 9000

  • UZ Leuven

    Leuven, 3000

Other trials in Belgium

Sweden2 sites

  • Sahlgrenska Universitetssjukhuset

    Gothenburg, 41345

  • Karolinska Universitetssjukhuset, Solna

    Stockholm, 17176

Other trials in Sweden

Ireland1 site

  • Beaumont Hospital

    Dublin, Dublin 9

Other trials in Ireland

About this study

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

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).

Read the 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.

Being tested
Dexpramipexole, Placebo
Keywords
Amyotrophic Lateral Sclerosis, ALS, Motor Neurone Disease, Motor Neuron Disease, Lou Gehrig's disease
Medical subject headings
Amyotrophic Lateral Sclerosis, Motor Neuron Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DexpramipexoleDrug

    Also called KNS-760704, BIIB050

    Oral tablet 150mg twice daily for up to 18 months.

  • PlaceboDrug

    Oral tablet twice daily for up to 18 months.

Study groups in NCT01281189
Study groupRoleWhat they receive
DexpramipexoleExperimentalNot described
Receives: Drug: Dexpramipexole
PlaceboPlacebo comparatorNot described
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: 12 months

  2. 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.

    Measured: 12 months

  3. 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.

    Measured: 12 months

Secondary outcomes (3)

  1. Death or Respiratory Insufficiency (DRI) up to Month 18

    Time to Death or Respiratory Insufficiency (DRI) is defined as receipt of a tracheostomy or the use of non-invasive ventilation (NIV) for ≥22 hours per day for at least 10 consecutive days. If NIV is used to meet the criteria for respiratory insufficiency, no measured slow vital capacity (SVC) at any subsequent assessment may be >50%. Time to DRI is calculated from the date of the first dose to the first date of one of the following events: death, tracheostomy, or the 10th day of consecutive NIV with no measured SVC >50% at any subsequent assessment.

    Measured: 18 months

  2. Death up to 18 Months

    Estimated time to death up to 18 months. This includes deaths reported greater than 30 days following discontinuation from the study (the time period for reporting all-cause mortality), regardless of subject disposition, up to 18 months from first dose.

    Measured: 18 months

  3. ≤50% Predicted Upright Slow Vital Capacity (SVC) or Died up to 18 Months

    The date of reaching ≤50% of predicted upright slow vital capacity (SVC) is defined as the date of the first visit at which a predicted upright SVC is ≤50% and continues to remain ≤50% at the subsequent visit except for the last available observation. The time to reach ≤50% of predicted upright SVC is defined as the duration between the date of reaching ≤50% of predicted upright SVC and the date of the first dose of study medication. If the subject is alive and does not reach ≤50% of predicted upright SVC, the time to reach ≤50% of predicted upright SVC will be censored and equal to the number of days from the first dose of study medication until the visit date when the subject's last available SVC assessment is performed. The earliest time (Reaching ≤50% Predicted Upright SVC or death) is used in analysis.

    Measured: 18 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Jan 2011 (estimated)
  2. Study startMar 2011
  3. Primary completionNov 2012
  4. Study completionNov 2012

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
942 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01281189
Sponsor's protocol ID
223AS302
Other IDs
EUDRA CT NO: 2010-022818-19
Acronym
EMPOWER
Status last verified
May 2021
First submitted
20 Jan 2011
Last updated
7 Jun 2021
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 24067398

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Dexpramipexole in people with Amyotrophic Lateral Sclerosis, run by Knopp Biosciences at 82 sites in 11 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 7 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 82 sites in United States, Canada, France, United Kingdom, Germany and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 7 Jun 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Composite Assessment of Function and Survival (CAFS) at 12 Months (time frame: 12 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01281189, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Jun 2021.

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Phase 3 Study of Dexpramipexole in ALS (NCT01281189) | AzTrials