Phase 3 Study of Dexpramipexole in ALS (EMPOWER)

May 10, 2021 updated by: Knopp Biosciences

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
942

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • New South Wales
      • Randwick, New South Wales, Australia
        • Prince of Wales Hospital
      • Westmead, New South Wales, Australia, 2145
        • Westmead Hospital
    • Queensland
      • Herston, Queensland, Australia, 4029
        • Royal Brisbane and Women's Hospital
    • Victoria
      • Melbourne, Victoria, Australia, 3121
        • Calvary Health Care Bethlehem
  • Belgium
      • Gent, Belgium, 9000
        • AZ St-Lucas
      • Leuven, Belgium, 3000
        • UZ Leuven
  • Canada
      • London, Canada
        • London Health Sciences Centre
      • Toronto, Canada, M4N 3M5
        • Sunnybrook and Women's College and Health Sciences Centre
      • Vancouver, Canada
        • University of British Columbia
    • Alberta
      • Calgary, Alberta, Canada, T2V 1P9
        • Univ of Calgary / Foothills MC
    • Quebec
      • Montreal, Quebec, Canada, H3A 2B4
        • McGill University
      • Montreal, Quebec, Canada, H2L 4M1
        • CHUM - Hopital Notre Dame
  • France
      • Lille, France, 59037
        • CHRU de Lille - Hôpital Roger Salengro
      • Limoges, France
        • CHU de Limoges - Hôpital Dupuytren
      • Marseille, France
        • Centre Hospitalier La Timone
      • Montpellier, France, 34295
        • CHU Gui de Chauliac
      • Nice, France
        • CHU de Nice - Hôpital de l'Archet 1
      • Paris, France, 75013
        • Hôpital la pitié salpètrière
  • Germany
      • Berlin, Germany
        • Charité - Universitätsmedizin Berlin
      • Bochum, Germany
        • Bergmannsheil Gmbh
      • Hannover, Germany
        • Medizinische Hochschule Hannover (MHH)
      • Jena, Germany
        • Universitatsklinikum Jena
      • Ulm, Germany
        • University of Ulm, RKU
  • Ireland
      • Dublin, Ireland, Dublin 9
        • Beaumont Hospital
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Academisch Medisch Centrum
      • Nijmegen, Netherlands, 6525 GA
        • UMC St. Radboud
      • Utrecht, Netherlands, 3584 CX
        • Universitair Medisch Centrum Utrecht
  • Spain
      • Barcelona, Spain
        • Hospital Vall d'Hebron
      • Barcelona, Spain, 8907
        • Hospital Universitario de Bellvitge
      • Madrid, Spain, 28046
        • Hospital La Paz
      • Madrid, Spain
        • Hospital Carlos III
  • Sweden
      • Göteborg, Sweden, 41345
        • Sahlgrenska Universitetssjukhuset
      • Stockholm, Sweden, 17176
        • Karolinska Universitetssjukhuset, Solna
  • United Kingdom
      • Birmingham, United Kingdom, B15 2TH
        • Queen Elizabeth Hospital
      • Liverpool, United Kingdom, L9 7LJ
        • Walton Centre for Neurology & Neurosurgery
      • London, United Kingdom, SE5 8AF
        • Kings College Hospital NHS Foundation Trust
      • Newcastle, United Kingdom, NE4 5PL
        • Newcastle University Hospital - Clinical Ageing Research Unit
      • Oxford, United Kingdom
        • John Radcliffe Hospital
      • Sheffield, United Kingdom, S10 2HQ
        • Sheffield Institute for Transnational Neuroscience
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • Barrow Neurological Institute - St. Joseph's Hospital
    • Arkansas
      • Little Rock, Arkansas, United States, 72205
        • University of Arkansas for Medical Sciences
    • California
      • Fresno, California, United States, 93701
        • University of California at San Francisco - Fresno
      • Orange, California, United States, 92868
        • University of California, Irvine
      • Sacramento, California, United States, 95817
        • University of California, Davis
      • San Francisco, California, United States, 94115
        • California Pacific Medical Center
    • Connecticut
      • New Britain, Connecticut, United States, 06053
        • Hospital for Special Care
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic - Jacksonville
      • Miami, Florida, United States, 33136
        • University of Miami Miller School of Medicine
      • Tampa, Florida, United States, 33612
        • University Of South Florida Medical Center
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins University School of Medicine
    • Massachusetts
      • Charlestown, Massachusetts, United States, 02129
        • Massachusetts General Hospital
    • Michigan
      • Grand Rapids, Michigan, United States, 49503
        • St. Mary's Health Care
    • Minnesota
      • Minneapolis, Minnesota, United States, 55404
        • Hennepin County Medical Center
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic - Rochester
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • Nebraska
      • Lincoln, Nebraska, United States, 68506
        • Neurology Associates, P.C.
    • Nevada
      • Las Vegas, Nevada, United States, 89102
        • University of Nevada School of Medicine
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Dartmouth-Hitchcock Medical Center
    • New York
      • New York, New York, United States, 10032
        • Columbia University
      • Syracuse, New York, United States, 12201
        • Research Foundation of the State University of New York
    • North Carolina
      • Charlotte, North Carolina, United States, 28207
        • Carolinas Medical Center
      • Durham, North Carolina, United States, 27705
        • Duke University Medical Center
      • Winston-Salem, North Carolina, United States, 27157
        • Wake Forest University
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • The Cleveland Clinic Foundation
      • Columbus, Ohio, United States, 43210
        • Ohio State University
    • Oregon
      • Portland, Oregon, United States, 97213
        • Providence ALS Center
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Hershey Medical Center
      • Philadelphia, Pennsylvania, United States, 19129
        • Drexel University College of Medicine
      • Philadelphia, Pennsylvania, United States, 19107
        • ALS Center at Penn
      • Pittsburgh, Pennsylvania, United States, 15213
        • University of Pittsburgh Medical Center
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center
    • Texas
      • Dallas, Texas, United States, 75214
        • Texas Neurology
      • Houston, Texas, United States, 77030
        • Methodist Neurological Institute
      • San Antonio, Texas, United States, 78229
        • University of Texas Health Sciences Center
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • University of Virginia Health System
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: Placebo
Drug: Placebo
Oral tablet twice daily for up to 18 months.
Experimental: Dexpramipexole
Drug: Dexpramipexole
Oral tablet 150mg twice daily for up to 18 months.
Other Names:
  • KNS-760704
  • BIIB050

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Composite Assessment of Function and Survival (CAFS) at 12 Months
Time Frame: 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)
Time Frame: 12 months
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)
Time Frame: 12 months
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 Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Death or Respiratory Insufficiency (DRI) up to Month 18
Time Frame: 18 months
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.
18 months
Death up to 18 Months
Time Frame: 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.
18 months
≤50% Predicted Upright Slow Vital Capacity (SVC) or Died up to 18 Months
Time Frame: 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.
18 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Knopp Biosciences

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Merit Cudkowicz, MD, MSc, Professor of Neurology of the Harvard Medical School

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 1, 2011

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 1, 2012

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 1, 2012

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 20, 2011

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 20, 2011

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 21, 2011

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 7, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 10, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 223AS302
  • EUDRA CT NO: 2010-022818-19

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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