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


.(JavaScript must be enabled to view this email address)
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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:

12/16/2016

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

    Hospital for Special Surgery
    New York, New York 10021
    United States

    Texas Neurology, PA
    Dallas, Texas 75214
    United States

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

    Montreal Neurological Institute and Hospital
    Montreal, Quebec
    Canada

    McMaster University Medical Centre
    Hamilton, Ontario
    Canada