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

May 27, 2021 updated by: Cytokinetics

A Phase 3, Open-Label Extension Study of Tirasemtiv for Patients With Amyotrophic Lateral Sclerosis (ALS) Who Completed VITALITY-ALS (CY 4031)

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 Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
280

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
    • Vlaams Brabant
      • Leuven, Vlaams Brabant, Belgium, 3000
        • UZ Leuven
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 1N4
        • University of Calgary
      • Edmonton, Alberta, Canada, T6G 1Z1
        • University of Alberta
    • New Brunswick
      • Fredericton, New Brunswick, Canada, E3B 0C7
        • Stan Cassidy Centre for Rehabilitation
    • Nova Scotia
      • Halifax, Nova Scotia, Canada, B3H 3A7
        • QE II Health Sciences, Nova Scotia Health Authority
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • McMaster University Medical Centre
      • London, Ontario, Canada, N6A 5A5
        • London Health Sciences Centre
      • Toronto, Ontario, Canada, M4N 3M5
        • Sunnybrook Health Sciences Center
    • Quebec
      • Montreal, Quebec, Canada, H3A 2B4
        • Montreal Neurological Institute and Hospital
      • Montreal, Quebec, Canada, H2L 4M1
        • Hopital Notre-Dame/CHUM
      • Quebec City, Quebec, Canada, G1J 1Z4
        • CHU de Quebec - Univerite' Laval
  • France
      • Limoges Cedex, France, 87042
        • Hopital Dupuytren, service de neurologie
      • Montpellier Cedex 5, France, 34295
        • Hopital Gui de Chauliac
      • Nice Cedex 1, France, 06001
        • CHU de Nice - Hôpital Pasteur 2
      • Tours cedex 9, France, 37044
        • Hopital Bretonneau
  • Germany
      • Berlin, Germany, 13353
        • Charite Campus Virchow-Klinikum, Department of Neurology
    • Baden-Wuerttemberg
      • Ulm, Baden-Wuerttemberg, Germany, 89081
        • University of Ulm, Department of Neurology
    • Lower Saxony
      • Hannover, Lower Saxony, Germany, 30625
        • Hannover Medical School, Department of Neurology
  • Ireland
      • Dublin, Ireland, Dublin 9
        • Clinical Research Centre Beaumont Hospital
  • Italy
    • Lombardy
      • Milan, Lombardy, Italy, 20149
        • IRCCS Istituto Auxologico Italiano - U.O. Neurologia
      • Milan, Lombardy, Italy, 20162
        • Centro Clinico NEMO - Fondazione Serena Onlus, ASST Grande Ospedale Metropolitano Niguarda, Ospedale Niguarda
    • Piemonte
      • Torino, Piemonte, Italy, 10126
        • Dipartimento di Neuroscienze "Rita Levi Moltalcini" A.O.U. Citta della Salute e della Scienza di Torino P.O. "Molinette"
  • Netherlands
      • Utrecht, Netherlands, 3584 CX
        • University Medical Center Utrecht
  • Portugal
      • Lisboa, Portugal, 1649-035
        • Hospital Santa Maria - Centro Hospitalar Lisboa Norte
  • Spain
      • Madrid, Spain, 28016
        • Hospital San Rafael
  • United Kingdom
      • London, United Kingdom, SE5 9RS
        • King's College Hospital NHS Foundation Trust
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85013
        • St. Joseph's Hospital and Medical Center - Barrow Neurology Clinics
    • California
      • Los Angeles, California, United States, 90048
        • Cedars-Sinai Medical Center
      • Orange, California, United States, 92868
        • University of California, Irvine
      • Sacramento, California, United States, 95817
        • UC Davis Medical Center
      • San Francisco, California, United States, 94115
        • Forbes Norris MDA/ALS Research Center
      • Stanford, California, United States, 94305
        • Stanford Hospital and Clinics
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado Hospital Anschutz Outpatient Pavilion
    • Connecticut
      • New Britain, Connecticut, United States, 06053
        • Hospital for Special Care
    • District of Columbia
      • Washington, District of Columbia, United States, 20037
        • George Washington University Medical Center
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Mayo Clinic
      • Miami, Florida, United States, 33136
        • University of Miami, Miller School of Medicine
      • Tampa, Florida, United States, 33512
        • Carol & Frank Morsani Center for Advanced Health Care - University of South Florida
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • The Emory Clinic
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University Of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Johns Hopkins University
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
        • University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • University of Michigan Health System
      • Detroit, Michigan, United States, 48202
        • Henry Ford Health System
    • Minnesota
      • Minneapolis, Minnesota, United States, 55415
        • Hennepin County Medical Center
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Barnes-Jewish Hospital
      • Saint Louis, Missouri, United States, 63104
        • St. Louis University, Department of Neurology & Psychiatry
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Dartmouth Hitchcock Medical Center
    • New York
      • New York, New York, United States, 10021
        • Hospital for Special Surgery
      • New York, New York, United States, 10032
        • Neurological Institute
      • Syracuse, New York, United States, 13210
        • SUNY Upstate Medical University
    • North Carolina
      • Charlotte, North Carolina, United States, 28207
        • Neurosciences Institute, Neurology - Charlotte
      • Durham, North Carolina, United States, 27705
        • Duke Neurological Disorders Clinic
      • Winston-Salem, North Carolina, United States, 27157
        • Department of Neurology, Wake Forest School of Medicine
    • Ohio
      • Columbus, Ohio, United States, 43210
        • The Ohio State University Wexner Medical Center
    • Oregon
      • Portland, Oregon, United States, 97213
        • Providence Brain and Spine Inst. ALS Center
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Milton S. Hershey Medical Center
      • Philadelphia, Pennsylvania, United States, 19140
        • Temple University School of Medicine
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University Medical Center - Clinical Research Center
    • Texas
      • Dallas, Texas, United States, 75214
        • Texas Neurology, PA
      • San Antonio, Texas, United States, 78229
        • UTHSCSA - First Outpatient Research Unit
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • University of Virginia Health System
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington Medical Center
    • West Virginia
      • Morgantown, West Virginia, United States, 26506
        • West Virginia University Hospitals
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Froedtert Memorial Lutheran Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Delayed Start Treatment
The Delayed Start Treatment group consisted of patients who received placebo in CY 4031 and tirasemtiv in CY 4033.
Drug: tirasemtiv
Oral
Other Names:
  • CK-2017357
Experimental: Early Start Treatment
The Early Start Treatment group consisted of patients who received tirasemtiv in both CY 4031 and CY 4033.
Drug: tirasemtiv
Oral
Other Names:
  • CK-2017357

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Participants With Adverse Events
Time Frame: 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.
From the first dose of tirasemtiv through 28 days after the last dose

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 24 in CY 4033
Time Frame: 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]).
baseline and 24 weeks
Change From CY 4031 Baseline in Percent Predicted Slow Vital Capacity to Week 48 in CY 4033
Time Frame: 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]).
baseline and 48 weeks
Change From CY 4031 Baseline in ALS Functional Rating Scale - Revised (ALSFRS-R) Total Score at Week 24
Time Frame: 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.
baseline and 24 weeks
Change From CY 4031 Baseline in ALSFRS-R Total Score at Week 48
Time Frame: 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.
baseline and 48 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Cytokinetics

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: MD, Cytokinetics, Cytokinetics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 17, 2016

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 26, 2018

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 26, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 12, 2016

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 14, 2016

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 18, 2016

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 15, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 27, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CY 4033
  • 2016-002629-13 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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