A Phase 2, Multi-Center, Double-Blind, Randomized, Dose-Ranging, Placebo-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of CK-2127107 in Patients With ALS

Study Purpose:

The purpose of this study is to assess the effect of CK-2127107 versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.

Disease:

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

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

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

Clinicaltrials.gov ID (11 digit #):

NCT03160898

Neals Affiliated?

No

Coordinating Center Contact Information


MD Cytokinetics / .(JavaScript must be enabled to view this email address) / 650-624-2929
.(JavaScript must be enabled to view this email address)

Full Study Summary:

Enrolled participants will be dosed with CK-2127107 150, 300, 450 mg or placebo twice daily (300, 600, 900 mg/day or placebo) for a period of 12 weeks.

Study Sponsor:

Cytokinetics

Participant Duration:

12 weeks

Estimated Enrollment:

445

Estimated Study Start Date:

07/24/2017

Estimated Study Completion Date:

07/01/2018

Posting Last Modified Date:

01/11/2018

Date Study Added to alsconsortium.org:

09/01/2017
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    80

    Min Vital Capacity (% predicted normal):

    65

    Time since Symptom Onset:

    Time since Diagnosis:

    <24 months

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    Diagnosis of familial or sporadic ALS ≤ 24 months prior to screening
    Upright Slow Vital Capacity (SVC) ≥ 65% of predicted for age, height and sex at screening
    Able to swallow tablets
    A caregiver (if one is needed)
    Able to perform reproducible pulmonary function tests
    Pre-study clinical laboratory findings within the normal range or, if outside the normal range, deemed not clinically significant by the Investigator
    Male patients who have not had a vasectomy and confirmed zero sperm count must agree to either use acceptable methods of contraception or abstain from sex
    Female patients must be post-menopausal or sterilized or must not be breastfeeding, have a negative pregnancy test, have no intention to become pregnant during the study and use acceptable methods of contraception
    Patients must be either on riluzole for at least 30 days prior to screening or have not taken riluzole for at least 30 days prior to screening and not planning to start riluzole during the course of the study.

    Exclusion Criteria:
    At the time of screening, any use of non-invasive ventilation (NIV), e.g. continuous positive airway pressure [CPAP], noninvasive bi-level positive airway pressure [NPPV] or noninvasive volume ventilation [NVV]for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation
    Neurological impairment due to a condition other than ALS
    Presence at screening of any medically significant cardiac, pulmonary, GI, musculoskeletal, or psychiatric illness that might interfere with the patient's ability to comply with study procedures or that might confound the interpretation of clinical safety or efficacy data
    Has taken any investigational study drug within 30 days or five half-lives of the prior agent, whichever is longer, prior to dosing
    Known to have received CK-2127107 or tirasemtiv in any previous clinical trial
    Has received or is considering receiving during the course of the study any form of stem cell therapy for the treatment of ALS
    Has received or is considering receiving during the course of the study any form of gene therapy for the treatment of ALS
    Has received or is considering obtaining during the course of the study a diaphragmatic pacing system
    History of substance abuse within the past 2 years
    Use of certain medications

  • Site Contact Information

    Barrow Neurological Institute
    St. Joseph's Hospital and Medical Center
    Phoenix, Arizona 85013
    United States

    University of California Irvine
    Orange, California 92868
    United States

    Forbes Norris MDA/ALS Research Center
    San Francisco, California 94115
    United States

    Stanford Hospital and Clinics
    Stanford, California 94305
    United States

    University of Colorado Hospital Anschutz Outpatient Pavilion
    Aurora, Colorado 80045
    United States

    Hospital for Special Care
    New Britain , Connecticut 06053
    United States

    Duchossois Center for Advanced Medicine
    Chicago, Illinois 60637
    United States

    IU Health Neuroscience Center of Excellence
    Indianapolis, Indiana 46202
    United States

    Johns Hopkins University - Outpatient Center
    Baltimore, Maryland 21287
    United States

    University of Massachusetts Medical School
    Worcester, Massachusetts 01655
    United States

    Michigan Medicine
    Ann Arbor, Michigan 48109
    United States

    Henry Ford Health System
    Detroit, Michigan 48202
    United States

    Hennepin County Medical Center
    Minneapolis, Minnesota 55415
    United States

    Saint Louis University, Department of Neurology
    Saint Louis, Missouri 63104
    United States

    Neurology Associates, P.C.
    Lincoln, Nebraska 68506
    United States

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

    Neurological Institute, Columbia University Medical Center
    New York, New York 10032
    United States

    SUNY Upstate Medical University
    Syracuse, New York 13210
    United States

    Neurosciences Institute, Neurology
    Charlotte, North Carolina 28207
    United States

    Duke Neurological Disorders Clinic
    Durham, North Carolina 27705
    United States

    Wake Forest School of Medicine
    Winston-Salem, North Carolina 27157
    United States

    Cleveland Clinic
    Cleveland, Ohio 44195
    United States

    The Ohio State University Wexner Medical Center
    Columbus, Ohio 43210
    United States

    Providence Brain and Spine Institute ALS Center
    Portland, Oregon 97213
    United States

    Penn State Milton S. Hershey Medical Center
    Hershey, Pennsylvania 17033
    United States

    Vanderbilt University Medical Center - Clinical Research Center
    Nashville, Tennessee 37232
    United States

    Texas Neurology
    Dallas, Texas 75214
    United States

    UTHSCSA Medical Arts and Research Center
    San Antonio, Texas 78229
    United States

    University of Virginia Health System
    Charlottesville, Virginia 22908
    United States

    University of Washington Medical Center
    Seattle , Washington 98195
    United States

    West Virginia University, Dept. of Neurology
    Morgantown, West Virginia 26506-9180
    United States

    Froedtert Memorial Lutheran Hospital
    Milwaukee , Wisconsin 53226
    United States

    Centre de recherche du Centre Hospitalier de l'Universite de Montreal
    Montréal, Quebec H2X 0A9
    Canada

    CHU de Quebec-Universite Laval, Hopital de l'Enfant Jesus
    Quebec G1J 1Z4
    Canada

    McMaster University Medical Centre
    Hamilton, Ontario L8N 3Z5
    Canada

    Montreal Neurological Institute and Hospital
    Montreal, Quebec H3A 2B4
    Canada

    Sunnybrook Health Science Centre
    Toronto, Ontario M4N 3M5
    Canada

    University of Calgary, Heritage Medical Research Center
    Calgary, Alberta T2N 4Z6
    Canada