Phase 3 Study of Dexpramipexole in ALS

NCT ID: NCT01281189

Last Updated: 2021-06-07

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

942 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-03-31

Study Completion Date

2012-11-30

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Amyotrophic Lateral Sclerosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Dexpramipexole

Group Type EXPERIMENTAL

Dexpramipexole

Intervention Type DRUG

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

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Oral tablet twice daily for up to 18 months.

Interventions

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Dexpramipexole

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

Intervention Type DRUG

Placebo

Oral tablet twice daily for up to 18 months.

Intervention Type DRUG

Other Intervention Names

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KNS-760704 BIIB050

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Knopp Biosciences

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Merit Cudkowicz, MD, MSc

Role: PRINCIPAL_INVESTIGATOR

Professor of Neurology of the Harvard Medical School

Locations

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Barrow Neurological Institute - St. Joseph's Hospital

Phoenix, Arizona, United States

Site Status

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States

Site Status

University of California at San Francisco - Fresno

Fresno, California, United States

Site Status

University of California, Irvine

Orange, California, United States

Site Status

University of California, Davis

Sacramento, California, United States

Site Status

California Pacific Medical Center

San Francisco, California, United States

Site Status

Hospital for Special Care

New Britain, Connecticut, United States

Site Status

Mayo Clinic - Jacksonville

Jacksonville, Florida, United States

Site Status

University of Miami Miller School of Medicine

Miami, Florida, United States

Site Status

University of South Florida Medical Center

Tampa, Florida, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

Indiana University

Indianapolis, Indiana, United States

Site Status

University of Iowa

Iowa City, Iowa, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Site Status

Massachusetts General Hospital

Charlestown, Massachusetts, United States

Site Status

St. Mary's Health Care

Grand Rapids, Michigan, United States

Site Status

Hennepin County Medical Center

Minneapolis, Minnesota, United States

Site Status

Mayo Clinic - Rochester

Rochester, Minnesota, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

Neurology Associates, P.C.

Lincoln, Nebraska, United States

Site Status

University of Nevada School of Medicine

Las Vegas, Nevada, United States

Site Status

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, United States

Site Status

Columbia University

New York, New York, United States

Site Status

Research Foundation of the State University of New York

Syracuse, New York, United States

Site Status

Carolinas Medical Center

Charlotte, North Carolina, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Wake Forest University

Winston-Salem, North Carolina, United States

Site Status

The Cleveland Clinic Foundation

Cleveland, Ohio, United States

Site Status

Ohio State University

Columbus, Ohio, United States

Site Status

Providence ALS Center

Portland, Oregon, United States

Site Status

Penn State Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

ALS Center at Penn

Philadelphia, Pennsylvania, United States

Site Status

Drexel University College of Medicine

Philadelphia, Pennsylvania, United States

Site Status

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Texas Neurology

Dallas, Texas, United States

Site Status

Methodist Neurological Institute

Houston, Texas, United States

Site Status

University of Texas Health Sciences Center

San Antonio, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

University of Virginia Health System

Charlottesville, Virginia, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

Prince of Wales Hospital

Randwick, New South Wales, Australia

Site Status

Westmead Hospital

Westmead, New South Wales, Australia

Site Status

Royal Brisbane and Women's Hospital

Herston, Queensland, Australia

Site Status

Calvary Health Care Bethlehem

Melbourne, Victoria, Australia

Site Status

AZ St-Lucas

Ghent, , Belgium

Site Status

UZ Leuven

Leuven, , Belgium

Site Status

Univ of Calgary / Foothills MC

Calgary, Alberta, Canada

Site Status

CHUM - Hopital Notre Dame

Montreal, Quebec, Canada

Site Status

Mcgill University

Montreal, Quebec, Canada

Site Status

London Health Sciences Centre

London, , Canada

Site Status

Sunnybrook and Women's College and Health Sciences Centre

Toronto, , Canada

Site Status

University of British Columbia

Vancouver, , Canada

Site Status

CHRU de Lille - Hôpital Roger Salengro

Lille, , France

Site Status

CHU de Limoges - Hôpital Dupuytren

Limoges, , France

Site Status

Centre Hospitalier La Timone

Marseille, , France

Site Status

CHU Gui de Chauliac

Montpellier, , France

Site Status

CHU de Nice - Hôpital de l'Archet 1

Nice, , France

Site Status

Hôpital La Pitié Salpétrière

Paris, , France

Site Status

Charité - Universitätsmedizin Berlin

Berlin, , Germany

Site Status

Bergmannsheil Gmbh

Bochum, , Germany

Site Status

Medizinische Hochschule Hannover (MHH)

Hanover, , Germany

Site Status

Universitätsklinikum Jena

Jena, , Germany

Site Status

University of Ulm, RKU

Ulm, , Germany

Site Status

Beaumont Hospital

Dublin, , Ireland

Site Status

Academisch Medisch Centrum

Amsterdam, , Netherlands

Site Status

UMC St. Radboud

Nijmegen, , Netherlands

Site Status

Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

Site Status

Hospital Universitario de Bellvitge

Barcelona, , Spain

Site Status

Hospital Vall d'Hebron

Barcelona, , Spain

Site Status

Hospital La Paz

Madrid, , Spain

Site Status

Hospital Carlos III

Madrid, , Spain

Site Status

Sahlgrenska Universitetssjukhuset

Gothenburg, , Sweden

Site Status

Karolinska Universitetssjukhuset, Solna

Stockholm, , Sweden

Site Status

Queen Elizabeth Hospital

Birmingham, , United Kingdom

Site Status

Walton Centre for Neurology & Neurosurgery

Liverpool, , United Kingdom

Site Status

Kings College Hospital NHS Foundation Trust

London, , United Kingdom

Site Status

Newcastle University Hospital - Clinical Ageing Research Unit

Newcastle, , United Kingdom

Site Status

John Radcliffe Hospital

Oxford, , United Kingdom

Site Status

Sheffield Institute for Transnational Neuroscience

Sheffield, , United Kingdom

Site Status

Countries

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United States Australia Belgium Canada France Germany Ireland Netherlands Spain Sweden United Kingdom

References

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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.

Reference Type DERIVED
PMID: 24067398 (View on PubMed)

Other Identifiers

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EUDRA CT NO: 2010-022818-19

Identifier Type: -

Identifier Source: secondary_id

223AS302

Identifier Type: -

Identifier Source: org_study_id

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