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 (FORTITUDE-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?

Yes

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:

04/16/2018

Date Study Added to alsconsortium.org:

09/01/2017
  • More Information

    Watch this webinar from January 30, 2018 to learn about the FORTITUDE-ALS trial design and hopes for the drug: recording link.

  • 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
    Jacquelyn Nicolari / .(JavaScript must be enabled to view this email address) / 602-406-6606
    St. Joseph's Hospital and Medical Center
    Phoenix, Arizona 85013
    United States

    University of California Irvine
    Ivonne Turner / .(JavaScript must be enabled to view this email address) / Orange, California 92868
    United States

    Forbes Norris MDA/ALS Research Center
    Marguerite Engel / .(JavaScript must be enabled to view this email address) / San Francisco, California 94115
    United States

    Stanford Hospital and Clinics
    Michael Nguyen / .(JavaScript must be enabled to view this email address) / 650-724-6147
    Stanford, California 94305
    United States

    Cedars-Sinai Medical Center
    Koral Wheeler / .(JavaScript must be enabled to view this email address) / Los Angeles , California 90048
    United States

    University of Colorado Hospital Anschutz Outpatient Pavilion
    Nicola Haakonsen / .(JavaScript must be enabled to view this email address) / Aurora, Colorado 80045
    United States

    Hospital for Special Care
    New Britain, Connecticut 06053
    United States

    George Washington University Medical Faculty Associates
    Washington, District of Columbia 20037
    United States

    Mayo Clinic
    Pamela Desaro / .(JavaScript must be enabled to view this email address) / Jacksonville, Florida 32224
    United States

    University of Florida
    Gainesville, Florida 32610
    United States

    University of South Florida, Carol & Frank Morsani Center for Advanced Healthcare
    Tampa, Florida 33612
    United States

    Emory ALS Clinic
    Meraida Polak / .(JavaScript must be enabled to view this email address) / Atlanta, Georgia 30322
    United States

    Duchossois Center for Advanced Medicine
    Jacquelyn Hill / .(JavaScript must be enabled to view this email address) / 773-702-4610
    Chicago, Illinois 60637
    United States

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

    University of Iowa Hospitals and Clinics
    Jeri Sieren / .(JavaScript must be enabled to view this email address) / Iowa City, Iowa 52242
    United States

    University of Kansas Medical Center
    Ayla McCalley / .(JavaScript must be enabled to view this email address) / Kansas City , Kansas 66160
    United States

    Johns Hopkins University - Outpatient Center
    Kristin M. Riley / .(JavaScript must be enabled to view this email address) / 410-955-8511
    Baltimore, Maryland 21287
    United States

    University of Massachusetts Medical School
    Worcester, Massachusetts 01655
    United States

    Michigan Medicine
    Jayna Duell / .(JavaScript must be enabled to view this email address) / 734-763-9037
    Ann Arbor, Michigan 48109
    United States

    Henry Ford Health System
    Ashkhen Movsisyan / .(JavaScript must be enabled to view this email address) / 313-916-4123
    Detroit, Michigan 48202
    United States

    Hennepin County Medical Center
    Cindy Rohde / .(JavaScript must be enabled to view this email address) / 612-873-2607
    Minneapolis, Minnesota 55415
    United States

    Mayo Clinic
    Rochester, Minnesota 55905
    United States

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

    Washington University School of Medicine
    Saint Louis, Missouri 63110
    United States

    Neurology Associates, P.C.
    Kelli Ferguson / .(JavaScript must be enabled to view this email address) / Desirae Eschiti / .(JavaScript must be enabled to view this email address) / Lincoln, Nebraska 68506
    United States

    Hospital For Special Surgery
    Shara Holzberg / .(JavaScript must be enabled to view this email address) / 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
    Karen Grace / .(JavaScript must be enabled to view this email address) / Durham, North Carolina 27705
    United States

    Wake Forest School of Medicine
    Mozhdeh Marandi / .(JavaScript must be enabled to view this email address) / Winston-Salem, North Carolina 27157
    United States

    Cleveland Clinic
    Cleveland, Ohio 44195
    United States

    The Ohio State University Wexner Medical Center
    Ifeanyi Okoh / .(JavaScript must be enabled to view this email address) / Columbus, Ohio 43210
    United States

    Providence Brain and Spine Institute ALS Center
    Arlena Cummings / .(JavaScript must be enabled to view this email address) / 503-962-1171
    Portland, Oregon 97213
    United States

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

    Temple University School of Medicine
    Kathleen Hatala / .(JavaScript must be enabled to view this email address) / 215-707-4171
    Philadelphia, Pennsylvania 19140
    United States

    Vanderbilt University Medical Center - Clinical Research Center
    Diana Davis / .(JavaScript must be enabled to view this email address) / 615-322-8957
    Nashville, Tennessee 37232
    United States

    Texas Neurology
    Todd Morgan / .(JavaScript must be enabled to view this email address) / 214-827-3610 ext 228
    Dallas, Texas 75214
    United States

    UTHSCSA Medical Arts and Research Center
    Pamela Kittrell / .(JavaScript must be enabled to view this email address) / 210-450-0524
    San Antonio, Texas 78229
    United States

    Houston Methodist Hospital
    Luis F Lay, Jr. / .(JavaScript must be enabled to view this email address) / 713-441-3057
    Houston, Texas 77030
    United States

    University of Vermont Medical Center
    Burlington , Vermont 05405
    United States

    University of Virginia Health System
    Mary Wagoner / .(JavaScript must be enabled to view this email address) / Charlottesville, Virginia 22908
    United States

    VCU Health - Ambulatory Care Center (ACC)
    Tamika Walthour / .(JavaScript must be enabled to view this email address) / Richmond, Virginia 23298
    United States

    University of Washington Medical Center
    Laura Sissons-Ross / .(JavaScript must be enabled to view this email address) / Seattle , Washington 98195
    United States

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

    Froedtert Memorial Lutheran Hospital
    Lynn Wheeler / .(JavaScript must be enabled to view this email address) / Milwaukee , Wisconsin 53226
    United States

    University of Calgary, Heritage Medical Research Center
    Jose Antonio Martinez / .(JavaScript must be enabled to view this email address) / Calgary, Alberta T2N 4Z6
    Canada

    McMaster University Medical Centre
    Daniela Trapsa / .(JavaScript must be enabled to view this email address) / Hamilton, Ontario L8N 3Z5
    Canada

    Sunnybrook Health Science Centre
    Toronto, Ontario M4N 3M5
    Canada

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

    Centre de recherche du Centre Hospitalier de l'Universite de Montreal
    Stefanie Houle / .(JavaScript must be enabled to view this email address) / Montréal, Quebec H2X 0A9
    Canada

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

    Edmonton Kaye Clinic
    Shelley Wrona / .(JavaScript must be enabled to view this email address) / Edmonton, Alberta T6GT 1Z1
    Canada