A Study for Patients Who Completed VITALITY-ALS (CY 4031)
- Registration Number
- NCT02936635
- Lead Sponsor
- Cytokinetics
- Brief Summary
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).
- Detailed Description
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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 280
-
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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Early Start Treatment tirasemtiv The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033. Delayed Start Treatment tirasemtiv The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033.
- Primary Outcome Measures
Name Time Method Number of Participants With Adverse Events From the first dose of tirasemtiv through 28 days after the last dose The number of participants with adverse events was used as the measure for the long-term safety and tolerability of tirasemtiv.
- Secondary Outcome Measures
Name Time Method Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033 baseline and 24 weeks Slow vital capacity (SVC) was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to percent predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics \[eg, height, age, sex\]).
Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033 baseline and 48 weeks Slow vital capacity was measured using a spirometer (in units of liters). Following 3 to 5 breaths at rest, patients were instructed to take as deep an inspiration as possible followed by a maximum exhalation (blowing out all the air in their lungs). Values obtained were converted to percent predicted values (ie, the test result as a percent of predicted values for patients of similar demographic and baseline characteristics \[eg, height, age, sex\]).
Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24 baseline and 24 weeks The ALSFRS-R is used to measure the progression and severity of disability in patients with ALS. The ALSFRS-R consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in the following 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. Comparing postbaseline to baseline assessments, a negative value indicates a worsening in function.
Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48 baseline and 48 weeks The ALSFRS-R is used to measure the progression and severity of disability in patients with ALS. The ALSFRS-R consists of 12 questions, assessing a patient's capability and independence in functional activities relevant to ALS, categorized in the following 4 domains: gross motor tasks, fine motor tasks, bulbar functions, and respiratory function. Each question is scored from 0 (indicating incapable or dependent) to 4 (normal). The total score ranges from 0 to 48, with higher scores reflecting more normal function and lower scores reflecting more impaired function. Comparing postbaseline to baseline assessments, a negative value indicates a worsening in function.
Trial Locations
- Locations (67)
Cedars-Sinai Medical Center
🇺🇸Los Angeles, California, United States
Stanford Hospital and Clinics
🇺🇸Stanford, California, United States
The Emory Clinic
🇺🇸Atlanta, Georgia, United States
University of Iowa Hospitals and Clinics
🇺🇸Iowa City, Iowa, United States
Johns Hopkins University
🇺🇸Baltimore, Maryland, United States
University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School
🇺🇸Worcester, Massachusetts, United States
Barnes-Jewish Hospital
🇺🇸Saint Louis, Missouri, United States
St. Louis University, Department of Neurology & Psychiatry
🇺🇸Saint Louis, Missouri, United States
SUNY Upstate Medical University
🇺🇸Syracuse, New York, United States
Neurological Institute
🇺🇸New York, New York, United States
Hospital for Special Surgery
🇺🇸New York, New York, United States
Neurosciences Institute, Neurology - Charlotte
🇺🇸Charlotte, North Carolina, United States
The Ohio State University Wexner Medical Center
🇺🇸Columbus, Ohio, United States
Penn State Milton S. Hershey Medical Center
🇺🇸Hershey, Pennsylvania, United States
Texas Neurology, PA
🇺🇸Dallas, Texas, United States
Temple University School of Medicine
🇺🇸Philadelphia, Pennsylvania, United States
West Virginia University Hospitals
🇺🇸Morgantown, West Virginia, United States
University of Virginia Health System
🇺🇸Charlottesville, Virginia, United States
UZ Leuven
🇧🇪Leuven, Vlaams Brabant, Belgium
University of Calgary
🇨🇦Calgary, Alberta, Canada
QE II Health Sciences, Nova Scotia Health Authority
🇨🇦Halifax, Nova Scotia, Canada
London Health Sciences Centre
🇨🇦London, Ontario, Canada
Stan Cassidy Centre for Rehabilitation
🇨🇦Fredericton, New Brunswick, Canada
McMaster University Medical Centre
🇨🇦Hamilton, Ontario, Canada
Sunnybrook Health Sciences Center
🇨🇦Toronto, Ontario, Canada
Hopital Dupuytren, service de neurologie
🇫🇷Limoges Cedex, France
University of Ulm, Department of Neurology
🇩🇪Ulm, Baden-Wuerttemberg, Germany
Hannover Medical School, Department of Neurology
🇩🇪Hannover, Lower Saxony, Germany
Hopital Gui de chauliac
🇫🇷Montpellier Cedex 5, France
Hopital Bretonneau
🇫🇷Tours cedex 9, France
CHU de Nice - Hopital Pasteur 2
🇫🇷Nice Cedex 1, France
Charite Campus Virchow-Klinikum, Department of Neurology
🇩🇪Berlin, Germany
Hospital San Rafael
🇪🇸Madrid, Spain
King's College Hospital NHS Foundation Trust
🇬🇧London, United Kingdom
Dipartimento di Neuroscienze "Rita Levi Moltalcini" A.O.U. Citta della Salute e della Scienza di Torino P.O. "Molinette"
🇮🇹Torino, Piemonte, Italy
University of California, Irvine
🇺🇸Orange, California, United States
CHU de Quebec - Univerite' Laval
🇨🇦Quebec City, Quebec, Canada
Dartmouth Hitchcock Medical Center
🇺🇸Lebanon, New Hampshire, United States
Hospital for Special Care
🇺🇸New Britain, Connecticut, United States
George Washington University Medical Center
🇺🇸Washington, District of Columbia, United States
Mayo Clinic
🇺🇸Jacksonville, Florida, United States
Clinical Research Centre Beaumont Hospital
🇮🇪Dublin, Ireland
University of Alberta
🇨🇦Edmonton, Alberta, Canada
Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda, Ospedale Niguarda
🇮🇹Milan, Lombardy, Italy
Hopital Notre-Dame/CHUM
🇨🇦Montreal, Quebec, Canada
Montreal Neurological Institute and Hospital
🇨🇦Montreal, Quebec, Canada
IRCCS Istituto Auxologico Italiano - U.O. Neurologia
🇮🇹Milan, Lombardy, Italy
Hospital Santa Maria - Centro Hospitalar Lisboa Norte
🇵🇹Lisboa, Portugal
Indiana University
🇺🇸Indianapolis, Indiana, United States
Froedtert Memorial Lutheran Hospital
🇺🇸Milwaukee, Wisconsin, United States
St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
🇺🇸Phoenix, Arizona, United States
University of Miami, Miller School of Medicine
🇺🇸Miami, Florida, United States
Duke Neurological Disorders Clinic
🇺🇸Durham, North Carolina, United States
Forbes Norris MDA/ALS Research Center
🇺🇸San Francisco, California, United States
University of Colorado Hospital Anschutz Outpatient Pavilion
🇺🇸Aurora, Colorado, United States
Carol & Frank Morsani Center for Advanced Health Care - University of South Florida
🇺🇸Tampa, Florida, United States
Hennepin County Medical Center
🇺🇸Minneapolis, Minnesota, United States
Henry Ford Health System
🇺🇸Detroit, Michigan, United States
Vanderbilt University Medical Center - Clinical Research Center
🇺🇸Nashville, Tennessee, United States
Providence Brain and Spine Inst. ALS Center
🇺🇸Portland, Oregon, United States
University of Washington Medical Center
🇺🇸Seattle, Washington, United States
UTHSCSA - First Outpatient Research Unit
🇺🇸San Antonio, Texas, United States
UC Davis Medical Center
🇺🇸Sacramento, California, United States
University of Michigan Health System
🇺🇸Ann Arbor, Michigan, United States
Department of Neurology, Wake Forest School of Medicine
🇺🇸Winston-Salem, North Carolina, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
University Medical Center Utrecht
🇳🇱Utrecht, Netherlands