Clinical trial NCT01281189: Phase 3 Study of Dexpramipexole in ALS
Official title: A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study of the Safety and Efficacy of Dexpramipexole in Subjects With Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic Lateral Sclerosis
ALS
Motor Neurone Disease
Motor Neuron Disease
Lou Gehrig's disease
Lead sponsor: Knopp Biosciences
Sponsor class: INDUSTRY
First posted: 2011-01-21
Enrollment: 942
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

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 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.

Primary outcomes:
measure: Composite Assessment of Function and Survival (CAFS) at 12 Months
timeFrame: 12 months
measure: Death up to 12 Months (CAFs Individual Component)
timeFrame: 12 months
measure: Change From Baseline in ALSFRS-R at 12 Months (CAFs Individual Component)
timeFrame: 12 months
Secondary outcomes:
measure: Death or Respiratory Insufficiency (DRI) up to Month 18
timeFrame: 18 months
measure: Death up to 18 Months
timeFrame: 18 months
measure: ≤50% Predicted Upright Slow Vital Capacity (SVC) or Died up to 18 Months
timeFrame: 18 months
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Barrow Neurological Institute - St. Joseph's Hospital
state: Arizona
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: Fresno
country: United States
facility: University of California at San Francisco - Fresno
state: California
city: Orange
country: United States
facility: University of California, Irvine
state: California
city: Sacramento
country: United States
facility: University of California, Davis
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Center
state: California
city: New Britain
country: United States
facility: Hospital for Special Care
state: Connecticut
city: Jacksonville
country: United States
facility: Mayo Clinic - Jacksonville
state: Florida
city: Miami
country: United States
facility: University of Miami Miller School of Medicine
state: Florida
city: Tampa
country: United States
facility: University of South Florida Medical Center
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins University School of Medicine
state: Maryland
city: Charlestown
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Grand Rapids
country: United States
facility: St. Mary's Health Care
state: Michigan
city: Minneapolis
country: United States
facility: Hennepin County Medical Center
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic - Rochester
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Lincoln
country: United States
facility: Neurology Associates, P.C.
state: Nebraska
city: Las Vegas
country: United States
facility: University of Nevada School of Medicine
state: Nevada
city: Lebanon
country: United States
facility: Dartmouth-Hitchcock Medical Center
state: New Hampshire
city: New York
country: United States
facility: Columbia University
state: New York
city: Syracuse
country: United States
facility: Research Foundation of the State University of New York
state: New York
city: Charlotte
country: United States
facility: Carolinas Medical Center
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest University
state: North Carolina
city: Cleveland
country: United States
facility: The Cleveland Clinic Foundation
state: Ohio
city: Columbus
country: United States
facility: Ohio State University
state: Ohio
city: Portland
country: United States
facility: Providence ALS Center
state: Oregon
city: Hershey
country: United States
facility: Penn State Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: ALS Center at Penn
state: Pennsylvania
city: Philadelphia
country: United States
facility: Drexel University College of Medicine
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: Texas Neurology
state: Texas
city: Houston
country: United States
facility: Methodist Neurological Institute
state: Texas
city: San Antonio
country: United States
facility: University of Texas Health Sciences Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Charlottesville
country: United States
facility: University of Virginia Health System
state: Virginia
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: Randwick
country: Australia
facility: Prince of Wales Hospital
state: New South Wales
city: Westmead
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Herston
country: Australia
facility: Royal Brisbane and Women's Hospital
state: Queensland
city: Melbourne
country: Australia
facility: Calvary Health Care Bethlehem
state: Victoria
city: Gent
country: Belgium
facility: AZ St-Lucas
city: Leuven
country: Belgium
facility: UZ Leuven
city: Calgary
country: Canada
facility: Univ of Calgary / Foothills MC
state: Alberta
city: Montreal
country: Canada
facility: CHUM - Hopital Notre Dame
state: Quebec
city: Montreal
country: Canada
facility: Mcgill University
state: Quebec
city: London
country: Canada
facility: London Health Sciences Centre
city: Toronto
country: Canada
facility: Sunnybrook and Women's College and Health Sciences Centre
city: Vancouver
country: Canada
facility: University of British Columbia
city: Lille
country: France
facility: CHRU de Lille - Hôpital Roger Salengro
city: Limoges
country: France
facility: CHU de Limoges - Hôpital Dupuytren
city: Marseille
country: France
facility: Centre Hospitalier La Timone
city: Montpellier
country: France
facility: CHU Gui de Chauliac
city: Nice
country: France
facility: CHU de Nice - Hôpital de l'Archet 1
city: Paris
country: France
facility: Hôpital La Pitié Salpétrière
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Bochum
country: Germany
facility: Bergmannsheil Gmbh
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover (MHH)
city: Jena
country: Germany
facility: Universitätsklinikum Jena
city: Ulm
country: Germany
facility: University of Ulm, RKU
city: Dublin
country: Ireland
facility: Beaumont Hospital
city: Amsterdam
country: Netherlands
facility: Academisch Medisch Centrum
city: Nijmegen
country: Netherlands
facility: UMC St. Radboud
city: Utrecht
country: Netherlands
facility: Universitair Medisch Centrum Utrecht
city: Barcelona
country: Spain
facility: Hospital Universitario de Bellvitge
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
city: Madrid
country: Spain
facility: Hospital La Paz
city: Madrid
country: Spain
facility: Hospital Carlos III
city: Göteborg
country: Sweden
facility: Sahlgrenska Universitetssjukhuset
city: Stockholm
country: Sweden
facility: Karolinska Universitetssjukhuset, Solna
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital
city: Liverpool
country: United Kingdom
facility: Walton Centre for Neurology & Neurosurgery
city: London
country: United Kingdom
facility: Kings College Hospital NHS Foundation Trust
city: Newcastle
country: United Kingdom
facility: Newcastle University Hospital - Clinical Ageing Research Unit
city: Oxford
country: United Kingdom
facility: John Radcliffe Hospital
city: Sheffield
country: United Kingdom
facility: Sheffield Institute for Transnational Neuroscience
Study officials:
affiliation: Professor of Neurology of the Harvard Medical School
name: Merit Cudkowicz, MD, MSc
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.