Clinical trial NCT01622088: Phase 3 Extension Study of Dexpramipexole in ALS
Official title: An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS, Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Lead sponsor: Knopp Biosciences
Sponsor class: INDUSTRY
Collaborators: name: Biogen
First posted: 2012-06-18
Enrollment: 616
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.

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 collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.

Eligibility criteria: Inclusion Criteria:

* Subject has the ability to understand the purpose and risks of the study and provide signed and dated informed consent (or have the consent confirmed by a witness if unable to write) and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
* Subject was enrolled in either CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
* Subject has completed their last visit in Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
* Subjects of childbearing potential must practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after their last dose of study treatment.

Exclusion Criteria:

* Subject withdrew prematurely from Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
* Subject permanently discontinued study treatment in Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189) for any reason other than enrollment into this study.
* Subject from Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189) has a significant change in medical history (including laboratory tests or a clinically significant condition) that in the opinion of the Investigator would impair the subject's medical fitness for participation and preclude treatment.
* Female subject who is pregnant or breastfeeding.
* Subject is currently enrolled in any investigational drug study other than Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
* Subject is taking pramipexole, other dopamine agonists, any other agent with dopaminergic activity, or any other disallowed concomitant medication.
* Subject is unwilling or unable to comply with the requirements of the protocol including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to comply with the protocol. At a minimum, subjects who are not able to travel to the study site must be willing to agree to remote blood draws for clinical laboratory evaluations and telephone visits to report Adverse Events, concomitant medications, and Amyotrophic Lateral Sclerosis Functional Rating Scale (revised) (ALSFRS-R) scores.

Primary outcomes:
measure: Number of Subjects Who Reported an Adverse Event
timeFrame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
measure: Number of Subjects Who Experienced a Serious Adverse Event
timeFrame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
measure: Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event
timeFrame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
measure: Number of Participants With Potentially Clinically Significant Vital Sign Results
timeFrame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
measure: Number of Participants With Potentially Clinically Significant Hematology Results
timeFrame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
measure: Number of Participants With Potentially Clinically Significant Blood Chemistry Results
timeFrame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
measure: Number of Participants With Potentially Clinically Significant ECG Results
timeFrame: Baseline through end of study (maximum 226 days: approximately 32.2 weeks)
Secondary outcomes:
measure: Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study
timeFrame: Up to maximum 226 days: approximately 32.2 weeks
measure: Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study
timeFrame: Up to maximum 226 days: approximately 32.2 weeks
measure: Death up to 6 Months
timeFrame: 6 Months
measure: Percentage of Participants With Death or Death Equivalent up to 6 Months
timeFrame: 6 months
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Barrow Neurological Institute - St. Joseph's Hospital
state: Arizona
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: Drexel University College of Medicine
state: Pennsylvania
city: Philadelphia
country: United States
facility: ALS Center at Penn
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: 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: Knopp Biosciences
name: Michael Bozik, MD
role: STUDY_CHAIR
References: