A Study for Patients Who Completed VITALITY-ALS (CY 4031)

NCT ID: NCT02936635

Last Updated: 2021-06-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 280 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-10-17
• Study Completion Date: 2018-10-26

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.

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Delayed Start Treatment

The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033.

Group Type: EXPERIMENTAL

tirasemtiv

Intervention Type: DRUG

Oral

Early Start Treatment

The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033.

Group Type: EXPERIMENTAL

tirasemtiv

Intervention Type: DRUG

Oral

Interventions

tirasemtiv

Oral

Intervention Type: DRUG

Other Intervention Names

CK-2017357

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cytokinetics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

MD, Cytokinetics

Role: STUDY_DIRECTOR

Cytokinetics, Inc.

Locations

St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics

Phoenix, Arizona, United States

Cedars-Sinai Medical Center

Los Angeles, California, United States

University of California, Irvine

Orange, California, United States

UC Davis Medical Center

Sacramento, California, United States

Forbes Norris MDA/ALS Research Center

San Francisco, California, United States

Stanford Hospital and Clinics

Stanford, California, United States

University of Colorado Hospital Anschutz Outpatient Pavilion

Aurora, Colorado, United States

Hospital for Special Care

New Britain, Connecticut, United States

George Washington University Medical Center

Washington D.C., District of Columbia, United States

Mayo Clinic

Jacksonville, Florida, United States

University of Miami, Miller School of Medicine

Miami, Florida, United States

Carol & Frank Morsani Center for Advanced Health Care - University of South Florida

Tampa, Florida, United States

The Emory Clinic

Atlanta, Georgia, United States

Indiana University

Indianapolis, Indiana, United States

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Johns Hopkins University

Baltimore, Maryland, United States

University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School

Worcester, Massachusetts, United States

University of Michigan Health System

Ann Arbor, Michigan, United States

Henry Ford Health System

Detroit, Michigan, United States

Hennepin County Medical Center

Minneapolis, Minnesota, United States

St. Louis University, Department of Neurology & Psychiatry

St Louis, Missouri, United States

Barnes-Jewish Hospital

St Louis, Missouri, United States

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, United States

Hospital for Special Surgery

New York, New York, United States

Neurological Institute

New York, New York, United States

SUNY Upstate Medical University

Syracuse, New York, United States

Neurosciences Institute, Neurology - Charlotte

Charlotte, North Carolina, United States

Duke Neurological Disorders Clinic

Durham, North Carolina, United States

Department of Neurology, Wake Forest School of Medicine

Winston-Salem, North Carolina, United States

The Ohio State University Wexner Medical Center

Columbus, Ohio, United States

Providence Brain and Spine Inst. ALS Center

Portland, Oregon, United States

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Temple University School of Medicine

Philadelphia, Pennsylvania, United States

Vanderbilt University Medical Center - Clinical Research Center

Nashville, Tennessee, United States

Texas Neurology, PA

Dallas, Texas, United States

UTHSCSA - First Outpatient Research Unit

San Antonio, Texas, United States

University of Virginia Health System

Charlottesville, Virginia, United States

University of Washington Medical Center

Seattle, Washington, United States

West Virginia University Hospitals

Morgantown, West Virginia, United States

Froedtert Memorial Lutheran Hospital

Milwaukee, Wisconsin, United States

UZ Leuven

Leuven, Vlaams Brabant, Belgium

University of Calgary

Calgary, Alberta, Canada

University of Alberta

Edmonton, Alberta, Canada

Stan Cassidy Centre for Rehabilitation

Fredericton, New Brunswick, Canada

QE II Health Sciences, Nova Scotia Health Authority

Halifax, Nova Scotia, Canada

McMaster University Medical Centre

Hamilton, Ontario, Canada

London Health Sciences Centre

London, Ontario, Canada

Sunnybrook Health Sciences Center

Toronto, Ontario, Canada

Hopital Notre-Dame/CHUM

Montreal, Quebec, Canada

Montreal Neurological Institute and Hospital

Montreal, Quebec, Canada

CHU de Quebec - Univerite' Laval

Québec, Quebec, Canada

Hopital Dupuytren, service de neurologie

Limoges, , France

Hopital Gui de chauliac

Montpellier, , France

CHU de Nice - Hopital Pasteur 2

Nice, , France

Hopital Bretonneau

Tours, , France

University of Ulm, Department of Neurology

Ulm, Baden-Wurttemberg, Germany

Hannover Medical School, Department of Neurology

Hanover, Lower Saxony, Germany

Charite Campus Virchow-Klinikum, Department of Neurology

Berlin, , Germany

Clinical Research Centre Beaumont Hospital

Dublin, , Ireland

IRCCS Istituto Auxologico Italiano - U.O. Neurologia

Milan, Lombardy, Italy

Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda, Ospedale Niguarda

Milan, Lombardy, Italy

Dipartimento di Neuroscienze "Rita Levi Moltalcini" A.O.U. Citta della Salute e della Scienza di Torino P.O. "Molinette"

Turin, Piedmont, Italy

University Medical Center Utrecht

Utrecht, , Netherlands

Hospital Santa Maria - Centro Hospitalar Lisboa Norte

Lisbon, , Portugal

Hospital San Rafael

Madrid, , Spain

King's College Hospital NHS Foundation Trust

London, , United Kingdom

Countries

United States; Belgium; Canada; France; Germany; Ireland; Italy; Netherlands; Portugal; Spain; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2016-002629-13

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CY 4033

Identifier Type: -

Identifier Source: org_study_id