Phase 3 Extension Study of Dexpramipexole in ALS

NCT01622088 | Terminated Phase 3 Results DMC

• 2 other identifiers: 223AS304EUDRA CT #: 2011-006119-70
• Study Type: interventional
• Target: 616
• Locations: 11 countries
• Sites: 80

Brief Summary

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

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS, Motor Neuron Disease, Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (7)

• Number of Subjects Who Reported an Adverse Event

  The number of subjects who reported an adverse event during the study

  Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

• Number of Subjects Who Experienced a Serious Adverse Event

  The number of subjects enrolled who reported a serious adverse event during the study

  Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

• Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event

  The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study

  Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

• Number of Participants With Potentially Clinically Significant Vital Sign Results

  Number of Participants with Potentially Clinically Significant Vital Sign Abnormalities. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.

  Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

• Number of Participants With Potentially Clinically Significant Hematology Results

  Number of Participants with Potentially Clinically Significant Hematology Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.

  Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

• Number of Participants With Potentially Clinically Significant Blood Chemistry Results

  Number of Participants with Potentially Clinically Significant Blood Chemistry Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.

  Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

• Number of Participants With Potentially Clinically Significant ECG Results

  Number of Participants with Potentially Clinically Significant ECG Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.

  Baseline through end of study (maximum 226 days: approximately 32.2 weeks)

Secondary Outcomes (4)

• Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study

  Up to maximum 226 days: approximately 32.2 weeks

• Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study

  Up to maximum 226 days: approximately 32.2 weeks

• Death up to 6 Months

  6 Months

• Percentage of Participants With Death or Death Equivalent up to 6 Months

  6 months

Study Arms (1)

Dexpramipexole

EXPERIMENTAL

Dexpramipexole open-label

Drug: Dexpramipexole

Interventions

Dexpramipexole DRUG

Oral tablet 150 mg given twice daily (BID)

Also known as: BIIB050

Dexpramipexole

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Knopp Biosciences: lead
• Biogen: collaborator

Study Sites (80)

Barrow Neurological Institute - St. Joseph's Hospital

Phoenix, Arizona, 85013, United States

Location

University of California at San Francisco - Fresno

Fresno, California, 03721, United States

Location

University of California, Irvine

Orange, California, 92868, United States

Location

University of California, Davis

Sacramento, California, 95817, United States

Location

California Pacific Medical Center

San Francisco, California, 94115, United States

Location

Hospital for Special Care

New Britain, Connecticut, 06053, United States

Location

Mayo Clinic - Jacksonville

Jacksonville, Florida, 32224, United States

Location

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

Location

University of South Florida Medical Center

Tampa, Florida, 33612, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Indiana University

Indianapolis, Indiana, 46202, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21287, United States

Location

Massachusetts General Hospital

Charlestown, Massachusetts, 02129, United States

Location

St. Mary's Health Care

Grand Rapids, Michigan, 49503, United States

Location

Hennepin County Medical Center

Minneapolis, Minnesota, 55404, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Neurology Associates, P.C.

Lincoln, Nebraska, 68506, United States

Location

University of Nevada School of Medicine

Las Vegas, Nevada, 89102, United States

Location

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

Columbia University

New York, New York, 10032, United States

Location

Research Foundation of the State University of New York

Syracuse, New York, 12207, United States

Location

Carolinas Medical Center

Charlotte, North Carolina, 28207, United States

Location

Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Wake Forest University

Winston-Salem, North Carolina, 27157, United States

Location

The Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Ohio State University

Columbus, Ohio, 43210, United States

Location

Providence ALS Center

Portland, Oregon, 97213, United States

Location

Penn State Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Drexel University College of Medicine

Philadelphia, Pennsylvania, 19102, United States

Location

ALS Center at Penn

Philadelphia, Pennsylvania, 19107, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Texas Neurology

Dallas, Texas, 75214, United States

Location

Methodist Neurological Institute

Houston, Texas, 77030, United States

Location

University of Texas Health Sciences Center

San Antonio, Texas, 78229, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

University of Virginia Health System

Charlottesville, Virginia, 22908, United States

Location

University of Washington

Seattle, Washington, 98195, United States

Location

Prince of Wales Hospital

Randwick, New South Wales, Australia

Location

Westmead Hospital

Westmead, New South Wales, 2145, Australia

Location

Royal Brisbane and Women's Hospital

Herston, Queensland, 4029, Australia

Location

Calvary Health Care Bethlehem

Melbourne, Victoria, 3121, Australia

Location

AZ St-Lucas

Ghent, 9000, Belgium

Location

UZ Leuven

Leuven, 3000, Belgium

Location

Univ of Calgary / Foothills MC

Calgary, Alberta, T2V 1P9, Canada

Location

CHUM - Hopital Notre Dame

Montreal, Quebec, H2L 4M1, Canada

Location

Mcgill University

Montreal, Quebec, H3A 2B4, Canada

Location

London Health Sciences Centre

London, Canada

Location

Sunnybrook and Women's College and Health Sciences Centre

Toronto, M4N 3M5, Canada

Location

University of British Columbia

Vancouver, Canada

Location

CHRU de Lille - Hôpital Roger Salengro

Lille, 59037, France

Location

Centre Hospitalier La Timone

Marseille, France

Location

CHU Gui de Chauliac

Montpellier, 34295, France

Location

CHU de Nice - Hôpital de l'Archet 1

Nice, France

Location

Hôpital La Pitié Salpétrière

Paris, 75013, France

Location

Charité - Universitätsmedizin Berlin

Berlin, Germany

Location

Bergmannsheil Gmbh

Bochum, Germany

Location

Medizinische Hochschule Hannover (MHH)

Hanover, Germany

Location

Universitätsklinikum Jena

Jena, Germany

Location

University of Ulm, RKU

Ulm, Germany

Location

Beaumont Hospital

Dublin, Dublin 9, Ireland

Location

Academisch Medisch Centrum

Amsterdam, 1105 AZ, Netherlands

Location

UMC St. Radboud

Nijmegen, 6525 GA, Netherlands

Location

Universitair Medisch Centrum Utrecht

Utrecht, 3584 CX, Netherlands

Location

Hospital Universitario de Bellvitge

Barcelona, 8907, Spain

Location

Hospital Vall d'Hebron

Barcelona, Spain

Location

Hospital La Paz

Madrid, 28046, Spain

Location

Hospital Carlos III

Madrid, Spain

Location

Sahlgrenska Universitetssjukhuset

Gothenburg, 41345, Sweden

Location

Karolinska Universitetssjukhuset, Solna

Stockholm, 17176, Sweden

Location

Queen Elizabeth Hospital

Birmingham, B15 2TH, United Kingdom

Location

Walton Centre for Neurology & Neurosurgery

Liverpool, L9 7LJ, United Kingdom

Location

Kings College Hospital NHS Foundation Trust

London, SE5 8AF, United Kingdom

Location

Newcastle University Hospital - Clinical Ageing Research Unit

Newcastle, NE4 5PL, United Kingdom

Location

John Radcliffe Hospital

Oxford, United Kingdom

Location

Sheffield Institute for Transnational Neuroscience

Sheffield, S10 2HQ, United Kingdom

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease

Interventions

Dexpramipexole

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Benzothiazoles; Thiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Head of Regulatory
• Organization: Knopp Biosciences

greg@knoppbio.com

4124881776

Study Officials

• Michael Bozik, MD

  Knopp Biosciences

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 3, 2012
• First Posted: June 18, 2012
• Study Start: June 1, 2012
• Primary Completion: February 1, 2013
• Study Completion: February 1, 2013
• Last Updated: May 3, 2022
• Results First Posted: April 7, 2022

Record last verified: 2022-04

Locations

BE (2); NL (3); US (42); CA (6); SE (2); ES (4); GB (6); DE (5); AU (4); FR (5); IE (1)