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

Study Purpose:

The purpose of this study is to assess the long-term safety and tolerability of tirasemtiv in patients with ALS.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling by invitation

Phase:

Phase III

Study Chair(s)/Principal Investigator(s):

Clinicaltrials.gov ID (11 digit #):

NCT02936635

Neals Affiliated?

No

Coordinating Center Contact Information


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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

Participant Duration:

Estimated Enrollment:

350

Estimated Study Start Date:

10/01/2016

Estimated Study Completion Date:

10/01/2019

Posting Last Modified Date:

06/13/2017

Date Study Added to alsconsortium.org:

12/16/2016
  • More Information

    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 ]

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    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

  • Site Contact Information

    Barrow
    Phoenix , Arizona 85013
    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

    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, D.C., District of Columbia 20037
    United States

    University of South Florida, Carol & Frank Morsani Center for Advanced Health Care
    Tampa, Florida 33512
    United States

    Mayo Clinic
    Jacksonville, Florida 32224
    United States

    University of Miami, Miller School of Medicine
    Miami, Florida 33136
    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
    Worcester, Massachusetts 01655
    United States

    Henry Ford Health System
    Detroit, Michigan 48202
    United States

    Hennepin County Medical Center
    Minneapolis, Minnesota 55415
    United States

    St. Louis University
    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

    West Virginia University Hospitals
    Morgantown, West Virginia 26506
    United States

    Froedtert Memorial Lutheran Hospital
    Milwaukee, Wisconsin 53226
    United States

    CHU de Quebec - Univerite' Laval
    Quebec
    Canada

    Clinical Research Centre Beaumont Hospital
    Dublin, Ireland
    Ireland

    Hopital Notre-Dame/CHUM
    Montreal, Quebec
    Canada

    London Health Sciences Centre
    London, Ontario
    Canada

    McMaster University Medical Centre
    Hamilton, Ontario
    Canada

    Montreal Neurological Institute and Hospital
    Montreal, Quebec
    Canada

    Sunnybrook Health Sciences Center
    Toronto, Ontario
    Canada

    University of Alberta
    Edmonton, Alberta
    Canada

    University of Calgary
    Calgary, Alberta
    Canada