Clinical trial NCT02936635: A Study for Patients Who Completed VITALITY-ALS (CY 4031)
Official title: A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients With Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY-ALS (CY 4031)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Amyotrophic Lateral Sclerosis (ALS)
Keywords: Amyotrophic Lateral Sclerosis
ALS
tirasemtiv
Lead sponsor: Cytokinetics
Sponsor class: INDUSTRY
First posted: 2016-10-18
Enrollment: 280
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With Adverse Events
timeFrame: From the first dose of tirasemtiv through 28 days after the last dose
Secondary outcomes:
measure: Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033
timeFrame: baseline and 24 weeks
measure: Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033
timeFrame: baseline and 48 weeks
measure: Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24
timeFrame: baseline and 24 weeks
measure: Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48
timeFrame: baseline and 48 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
state: Arizona
city: Los Angeles
country: United States
facility: Cedars-Sinai Medical Center
state: California
city: Orange
country: United States
facility: University of California, Irvine
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: San Francisco
country: United States
facility: Forbes Norris MDA/ALS Research Center
state: California
city: Stanford
country: United States
facility: Stanford Hospital and Clinics
state: California
city: Aurora
country: United States
facility: University of Colorado Hospital Anschutz Outpatient Pavilion
state: Colorado
city: New Britain
country: United States
facility: Hospital for Special Care
state: Connecticut
city: Washington
country: United States
facility: George Washington University Medical Center
state: District of Columbia
city: Jacksonville
country: United States
facility: Mayo Clinic
state: Florida
city: Miami
country: United States
facility: University of Miami, Miller School of Medicine
state: Florida
city: Tampa
country: United States
facility: Carol & Frank Morsani Center for Advanced Health Care - University of South Florida
state: Florida
city: Atlanta
country: United States
facility: The Emory Clinic
state: Georgia
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
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
state: Maryland
city: Worcester
country: United States
facility: University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Health System
state: Michigan
city: Detroit
country: United States
facility: Henry Ford Health System
state: Michigan
city: Minneapolis
country: United States
facility: Hennepin County Medical Center
state: Minnesota
city: Saint Louis
country: United States
facility: St. Louis University, Department of Neurology & Psychiatry
state: Missouri
city: Saint Louis
country: United States
facility: Barnes-Jewish Hospital
state: Missouri
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: New York
country: United States
facility: Neurological Institute
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical University
state: New York
city: Charlotte
country: United States
facility: Neurosciences Institute, Neurology - Charlotte
state: North Carolina
city: Durham
country: United States
facility: Duke Neurological Disorders Clinic
state: North Carolina
city: Winston-Salem
country: United States
facility: Department of Neurology, Wake Forest School of Medicine
state: North Carolina
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Portland
country: United States
facility: Providence Brain and Spine Inst. ALS Center
state: Oregon
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Temple University School of Medicine
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center - Clinical Research Center
state: Tennessee
city: Dallas
country: United States
facility: Texas Neurology, PA
state: Texas
city: San Antonio
country: United States
facility: UTHSCSA - First Outpatient Research Unit
state: Texas
city: Charlottesville
country: United States
facility: University of Virginia Health System
state: Virginia
city: Seattle
country: United States
facility: University of Washington Medical Center
state: Washington
city: Morgantown
country: United States
facility: West Virginia University Hospitals
state: West Virginia
city: Milwaukee
country: United States
facility: Froedtert Memorial Lutheran Hospital
state: Wisconsin
city: Leuven
country: Belgium
facility: UZ Leuven
state: Vlaams Brabant
city: Calgary
country: Canada
facility: University of Calgary
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Fredericton
country: Canada
facility: Stan Cassidy Centre for Rehabilitation
state: New Brunswick
city: Halifax
country: Canada
facility: QE II Health Sciences, Nova Scotia Health Authority
state: Nova Scotia
city: Hamilton
country: Canada
facility: McMaster University Medical Centre
state: Ontario
city: London
country: Canada
facility: London Health Sciences Centre
state: Ontario
city: Toronto
country: Canada
facility: Sunnybrook Health Sciences Center
state: Ontario
city: Montreal
country: Canada
facility: Hopital Notre-Dame/CHUM
state: Quebec
city: Montreal
country: Canada
facility: Montreal Neurological Institute and Hospital
state: Quebec
city: Quebec City
country: Canada
facility: CHU de Quebec - Univerite' Laval
state: Quebec
city: Limoges Cedex
country: France
facility: Hopital Dupuytren, service de neurologie
city: Montpellier Cedex 5
country: France
facility: Hopital Gui de chauliac
city: Nice Cedex 1
country: France
facility: CHU de Nice - Hopital Pasteur 2
city: Tours cedex 9
country: France
facility: Hopital Bretonneau
city: Ulm
country: Germany
facility: University of Ulm, Department of Neurology
state: Baden-Wuerttemberg
city: Hannover
country: Germany
facility: Hannover Medical School, Department of Neurology
state: Lower Saxony
city: Berlin
country: Germany
facility: Charite Campus Virchow-Klinikum, Department of Neurology
city: Dublin
country: Ireland
facility: Clinical Research Centre Beaumont Hospital
city: Milan
country: Italy
facility: IRCCS Istituto Auxologico Italiano - U.O. Neurologia
state: Lombardy
city: Milan
country: Italy
facility: Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda, Ospedale Niguarda
state: Lombardy
city: Torino
country: Italy
facility: Dipartimento di Neuroscienze "Rita Levi Moltalcini" A.O.U. Citta della Salute e della Scienza di Torino P.O. "Molinette"
state: Piemonte
city: Utrecht
country: Netherlands
facility: University Medical Center Utrecht
city: Lisboa
country: Portugal
facility: Hospital Santa Maria - Centro Hospitalar Lisboa Norte
city: Madrid
country: Spain
facility: Hospital San Rafael
city: London
country: United Kingdom
facility: King's College Hospital NHS Foundation Trust
Study officials:
affiliation: Cytokinetics, Inc.
name: MD, Cytokinetics
role: STUDY_DIRECTOR
References: