Study Purpose:
The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS who had completed the double-blind placebo-controlled study of tirasemtiv in ALS (CY 4031).Study Status:
Not recruiting
Disease:
Amyotrophic Lateral Sclerosis (ALS)
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
tirasemtiv
Placebo:
No
Phase:
Phase 3
Study Chair(s)/Principal Investigator(s):
MD, Cytokinetics, Cytokinetics, Inc.
Clinicaltrials.gov ID:
Neals Affiliated?
No
Coordinating Center Contact Information
Full Study Summary:
Enrolled participants will begin dosing of tirasemtiv 125 mg twice daily (250 mg/day) for a period of 4 weeks and will titrate to their tolerated dose, the maximum dose being 250 mg twice daily (500 mg/day). This study will also compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 with those who completed treatment with placebo in CY 4031 during continued treatment of both groups with tirasemtiv during CY 4033, compare the clinical course of patients who completed treatment with tirasemtiv in CY 4031 during that study with their clinical course during continued treatment with tirasemtiv during CY 4033, and compare the clinical course of patients who completed treatment with placebo in CY 4031 during that study with their clinical course during treatment with tirasemtiv during CY 4033.
Study Sponsor:
Cytokinetics
Estimated Enrollment:
280
Estimated Study Start Date:
10 / 17 / 2016
Estimated Study Completion Date:
10 / 26 / 2018
Posting Last Modified Date:
06 / 15 / 2021
Date Study Added to neals.org:
10 / 18 / 2016
Primary Outcome Measures:
Incidence of adverse events (AEs) in patient population [ Time Frame: Until end of study, up to 36 months ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
Time to first use of assisted ventilation or death [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ] [ Designated as safety issue: No ]
Time to the first occurrence of respiratory insufficiency (defined as tracheostomy or the use of non-invasive ventilation (NIV) for ≥22 hours per day for ≥10 consecutive days) or death [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ] [ Designated as safety issue: No ]
Time to death [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ] [ Designated as safety issue: No ]
Decline in percent predicted Slow Vital Capacity (SVC) from baseline [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ] [ Designated as safety issue: No ]
Decline in ALS Functional Rating Scale - Revised (ALSFRS-R) score from baseline [ Time Frame: From date of entry into the study until the date of first documented progression or date of death from any cause, which came first, assessed up to 36 months ] [ Designated as safety issue: No ]
Slope of the change from baseline in percent predicted SVC [ Time Frame: First 24 weeks and first 48 weeks of either CY 4031 or CY 4033 ] [ Designated as safety issue: No ]
Slope of the change from baseline in ALSFRS-R [ Time Frame: First 24 weeks and first 48 weeks of either CY 4031 or CY 4033 ] [ Designated as safety issue: No ]
Minimum Age:
18 Years
Maximum Age:
N/A
Can participants use Riluzole?
Yes
Inclusion Criteria:- Able to comprehend and willing to sign an Informed Consent Form (ICF). If verbal consent is given, a Legal Designee of the patient must sign the ICF form
- Completed participation on study drug and the Follow-Up Visit in the CY 4031 study
- Male patients, who have not had a vasectomy AND confirmed zero sperm count, must agree for the duration of their participation in the study to either:
- Use a condom during sexual intercourse with female partners who are of childbearing potential AND to have female partners use a highly effective means of contraception OR
- Abstain from sexual intercourse during participation in the study
- Female patients who are not post-menopausal (≥ 1 year) or sterilized, must:
- Not be breastfeeding
- Have a negative pregnancy test
- Have no intention to become pregnant during participation in the study AND
- Practice sexual abstinence, defined as refraining from intercourse during the duration of the study OR if male partners are not vasectomized with a confirmed zero sperm count, require use of a condom AND use of a highly effective contraceptive measure
Exclusion Criteria:
- Has a diaphragm pacing system (DPS) at study entry or anticipate DPS placement during the course of the study
- Has taken an investigational study drug (other than tirasemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater
- Use of tizanidine and theophylline-containing medications during study participation
- Participation or planning to participate in any form of stem cell therapy for the treatment of ALS or another investigational drug
St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
Phoenix, Arizona
85013
United States
Cedars-Sinai Medical Center
Los Angeles, California
90048
United States
University of California, Irvine
Orange, California
92868
United States
UC Davis Medical Center
Sacramento, California
95817
United States
Forbes Norris MDA/ALS Research Center
San Francisco, California
94115
United States
Stanford Hospital and Clinics
Stanford, California
94305
United States
University of Colorado Hospital Anschutz Outpatient Pavilion
Aurora, Colorado
80045
United States
Hospital for Special Care
New Britain, Connecticut
06053
United States
George Washington University Medical Center
Washington, District of Columbia
20037
United States
Carol & Frank Morsani Center for Advanced Health Care - University of South Florida
Tampa, Florida
33512
United States
University of Miami, Miller School of Medicine
Miami, Florida
33136
United States
Mayo Clinic
Jacksonville, Florida
32224
United States
The Emory Clinic
Atlanta, Georgia
30322
United States
Indiana University
Indianapolis, Indiana
46202
United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa
52242
United States
University of Kansas Medical Center
Kansas City, Kansas
66160
United States
Johns Hopkins University
Baltimore, Maryland
21287
United States
University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School
Worcester, Massachusetts
01655
United States
University of Michigan Health System
Ann Arbor, Michigan
48109
United States
Henry Ford Health System
Detroit, Michigan
48202
United States
Hennepin County Medical Center
Minneapolis, Minnesota
55415
United States
St. Louis University, Department of Neurology & Psychiatry
Saint Louis, Missouri
63104
United States
Barnes-Jewish Hospital
Saint Louis, Missouri
63110
United States
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire
03756
United States
Hospital for Special Surgery
New York, New York
10021
United States
Neurological Institute
New York, New York
10032
United States
SUNY Upstate Medical University
Syracuse, New York
13210
United States
Neurosciences Institute, Neurology - Charlotte
Charlotte, North Carolina
28207
United States
Duke Neurological Disorders Clinic
Durham, North Carolina
27705
United States
Department of Neurology, Wake Forest School of Medicine
Winston-Salem, North Carolina
27157
United States
The Ohio State University Wexner Medical Center
Columbus, Ohio
43210
United States
Providence Brain and Spine Inst. ALS Center
Portland, Oregon
97213
United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania
17033
United States
Temple University School of Medicine
Philadelphia, Pennsylvania
19140
United States
Vanderbilt University Medical Center - Clinical Research Center
Nashville, Tennessee
37232
United States
Texas Neurology, PA
Dallas, Texas
75214
United States
UTHSCSA - First Outpatient Research Unit
San Antonio, Texas
78229
United States
University of Virginia Health System
Charlottesville, Virginia
22908
United States
University of Washington Medical Center
Seattle, Washington
98195
United States
West Virginia University Hospitals
Morgantown, West Virginia
26506
United States
Froedtert Memorial Lutheran Hospital
Milwaukee, Wisconsin
53226
United States
UZ Leuven
Leuven, Vlaams Brabant
3000
Belgium
University of Calgary
Calgary, Alberta
T2N 1N4
Canada
University of Alberta
Edmonton, Alberta
T6G 1Z1
Canada
Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick
E3B 0C7
Canada
QE II Health Sciences, Nova Scotia Health Authority
Halifax, Nova Scotia
B3H 3A7
Canada
McMaster University Medical Centre
Hamilton, Ontario
L8N 3Z5
Canada
London Health Sciences Centre
London, Ontario
N6A 5A5
Canada
Sunnybrook Health Sciences Center
Toronto, Ontario
M4N 3M5
Canada
Hopital Notre-Dame/CHUM
Montreal, Quebec
H2L 4M1
Canada
Montreal Neurological Institute and Hospital
Montreal, Quebec
H3A 2B4
Canada
CHU de Quebec - Univerite' Laval
Quebec City, Quebec
G1J 1Z4
Canada
Hopital Bretonneau
Tours cedex 9 37044
France
CHU de Nice - Hopital Pasteur 2
Nice Cedex 1 06001
France
Hopital Gui de chauliac
Montpellier Cedex 5 34295
France
Hopital Dupuytren, service de neurologie
Limoges Cedex 87042
France
University of Ulm, Department of Neurology
Ulm, Baden-Wuerttemberg
89081
Germany
Hannover Medical School, Department of Neurology
Hannover, Lower Saxony
30625
Germany
Charite Campus Virchow-Klinikum, Department of Neurology
Berlin 13353
Germany
Clinical Research Centre Beaumont Hospital
Dublin Dublin 9
Ireland
IRCCS Istituto Auxologico Italiano - U.O. Neurologia
Milan, Lombardy
20149
Italy
Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda, Ospedale Niguarda
Milan, Lombardy
20162
Italy
Dipartimento di Neuroscienze "Rita Levi Moltalcini" A.O.U. Citta della Salute e della Scienza di Torino P.O. "Molinette"
Torino, Piemonte
10126
Italy
University Medical Center Utrecht
Utrecht 3584 CX
Netherlands
Hospital Santa Maria - Centro Hospitalar Lisboa Norte
Lisboa 1649-035
Portugal
Hospital San Rafael
Madrid 28016
Spain
King's College Hospital NHS Foundation Trust
London SE5 9RS
United Kingdom