A Phase IIb, Multi-National, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Efficacy of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS) (BENEFIT-ALS)

Study Purpose:

The purpose of this research study is to evaluate the safety and effectiveness of CK-2017357 when taken with or without riluzole (also called Rilutek®) in patients with Amyotrophic Lateral Sclerosis (ALS).

Disease:

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

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Not enrolling

Phase:

Phase II

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

Jeremy Shefner, MD, PhD (SUNY Upstate Medical University)

Clinicaltrials.gov ID (11 digit #):

NCT01709149

Neals Affiliated?

Yes

Coordinating Center Contact Information


Jinsy Andrews, MD / .(JavaScript must be enabled to view this email address) / 650-624-2929
.(JavaScript must be enabled to view this email address) United States

Full Study Summary:

The length of the study, including screening, dosing, and follow-up, is approximately 20 weeks. After a one-week open-label phase during which all patients will receive CK-2017357 125 milligrams (mg) twice daily, patients will be randomized one to one (fifty-fifty) to receive double-blind CK-2017357 or matching placebo. The CK-2017357/placebo dose will be increased no faster than weekly to each patient's highest tolerated daily dose, with a maximum of 250 mg twice daily. The dose may be decreased based on tolerability. Patients will continue treatment at the highest tolerated dose to complete a total of 12 weeks of double-blind treatment. Patients may be on riluzole or not on riluzole at study entry. Patients not on riluzole must stay off riluzole. Patients on riluzole who are getting double-blind CK-2017357 will be given riluzole at half the labeled dosage (50 mg once a day instead of 50 mg twice a day). Blood tests for safety will be performed. Information about any side effects that may occur will also be collected.

Study Sponsor:

Cytokinetics

Participant Duration:

approximately 20 weeks

Estimated Enrollment:

400

Estimated Study Start Date:

10/31/2012

Estimated Study Completion Date:

11/02/2016

Posting Last Modified Date:

02/28/2013

Date Study Added to alsconsortium.org:

10/21/2012
  • More Information

    ***Patients must be either on a stable dose of riluzole 50 mg twice daily for at least 30 days prior to screening or have not taken riluzole for at least 30 days prior to screening and are willing not to begin riluzole use during the conduct of this study.

    As sites become activated, their contact information will be added to this page. Please check back.

  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    Time since Diagnosis:

    Can participants use Riluzole?

    Yes


    Inclusion Criteria:
    Able to comprehend and willing to sign an Informed Consent Form (ICF)
    Male or female 18 years of age or older
    A diagnosis of familial or sporadic ALS (defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria)
    Upright Slow Vital Capacity (SVC) >60 % of predicted for age, height and sex
    At least 4 of the 12 ALSFRS-R questions must be scored 2 or 3
    Diminished but measurable maximum voluntary grip strength in at least one hand; i.e., between 10 and 40 pounds (females) and 10 and 60 pounds (males)
    Able to swallow tablets without crushing
    A caregiver (if one is needed) who can and will observe and report the patient's status
    Pre-study clinical laboratory findings within normal range or, if outside of the normal range, deemed not clinically significant by the Investigator
    Male patients must agree for the duration of the study and 10 weeks after the end of the study to use a condom during sexual intercourse with female partners who are of reproductive potential and to have female partners use an additional effective means of contraception (e.g., diaphragm plus spermicide, or oral contraceptives) or the male patient must agree to abstain from sexual intercourse during and for 10 weeks after the end of the study
    Female patients must be post-menopausal (≥ 1 year) or sterilized, or, if of childbearing potential, not be breastfeeding, have a negative pregnancy test, have no intention to become pregnant during the course of the study, and use contraceptive drugs or devices as detailed in item 10 for the duration of the study and for 10 weeks after the end of the study
    Patients must be either on a stable dose of riluzole 50 mg twice daily for at least 30 days prior to screening or have not taken riluzole for at least 30 days prior to screening and are willing not to begin riluzole use during the conduct of this study.

    Exclusion Criteria:
    Any use of non-invasive positive pressure ventilation (NIPPV, e.g. continuous positive airway pressure [CPAP] or bilevel positive airway pressure [BiPAP]) for any portion of the day, or mechanical ventilation via tracheostomy, or on any form of oxygen supplementation
    Patients with a diaphragm pacing system (DPS) at study entry or who anticipate DPS placement during the course of the study
    Body Mass Index (BMI) of 19.0 kg/m2 or lower
    Unwilling to discontinue theophylline-containing medications during study participation
    Serum chloride < 100 mg/deciliter (dL)
    Neurological impairment due to a condition other than ALS, including history of transient ischemic attack (TIA) within the past year
    Presence at screening of any medically significant cardiac, pulmonary, gastrointestinal (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 5 half-lives of the prior agent, whichever is greater, prior to dosing
    Previously received CK-2017357 in any previous clinical trial

  • Site Contact Information

    Barrow Neurology
    Gale Kittle / .(JavaScript must be enabled to view this email address) / 602-406-4792
    Phoenix, Arizona 85013
    United States

    University of California, San Diego
    Aaryn Belfer / .(JavaScript must be enabled to view this email address) / 858-246-0247
    La Jolla, California 92093
    United States

    California Pacific Medical Center Forbes Norris MDA/ALS Research Center
    Marguerite Engel / .(JavaScript must be enabled to view this email address) / 415-600-3758
    San Francisco, California 94115
    United States

    UC Irvine ALS & Neuromuscular Center
    Brian Minton / .(JavaScript must be enabled to view this email address) / 714-456-8520
    Orange, California 92868
    United States

    Coordinated Clinical Research
    Luci Barbie / .(JavaScript must be enabled to view this email address) / 619-297-3023
    San Diego, California 92037
    United States

    Hospital for Special Care
    Agnes Koczon-Jaremko / .(JavaScript must be enabled to view this email address) / 860-612-6356
    New Britain, Connecticut 06053
    United States

    Mayo Clinic Florida
    Catherine Ruiz / .(JavaScript must be enabled to view this email address) / 904-953-6523
    Jacksonville, Florida 32224
    United States

    Emory University
    Meraida Polak / .(JavaScript must be enabled to view this email address) / 404-778-3807
    Atlanta, Georgia 30322
    United States

    Georgia Health Sciences University
    Brandy Quarles / .(JavaScript must be enabled to view this email address) / 706-721-2681
    Augusta, Georgia 30912
    United States

    Indiana University Department of Neurology
    Sandra Guingrich / .(JavaScript must be enabled to view this email address) / 317-963-7382
    Indianapolis, Indiana 46202
    United States

    University of Iowa Hospitals and Clinics
    Jeri Sieren / .(JavaScript must be enabled to view this email address) / 319-356-8744
    Iowa City, Iowa 52242
    United States

    University of Kansas
    Maureen Walsh / .(JavaScript must be enabled to view this email address) / 913-588-0645
    Kansas City, Kansas 66160
    United States

    Johns Hopkins University
    Kristen Riley / .(JavaScript must be enabled to view this email address) / 410-955-8511
    Baltimore, Maryland 21205
    United States

    University of Massachusetts Medical School
    Diane McKenna-Yasek, RN, BSN / .(JavaScript must be enabled to view this email address) / 508-856-4697
    Worcester, Massachusetts 01655
    United States

    Massachusetts General Hospital
    Lorena Loci / .(JavaScript must be enabled to view this email address) / 617-724-0783
    Boston, Massachusetts 02114
    United States

    Henry Ford Hospital
    Helen Foley / .(JavaScript must be enabled to view this email address) / 313-916-3955
    Detroit, Michigan 48202
    United States

    St Mary's Healthcare
    Lynn Cherney / .(JavaScript must be enabled to view this email address) / 616-685-5091
    Grand Rapids, Michigan 49503
    United States

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

    Hennepin County Medical Center - Berman Center for Research
    Cindy Rohde / .(JavaScript must be enabled to view this email address) / 612-341-7923
    Minneapolis, Minnesota 55415
    United States

    Saint Louis University
    AnneMarie Fann / .(JavaScript must be enabled to view this email address) / 314-977-4868
    St. Louis, Missouri 63104
    United States

    Washington University
    Charlie Wulf / .(JavaScript must be enabled to view this email address) / 314-362-6980
    St. Louis, Missouri 63110
    United States

    Neurology Associates
    Becky Weber / .(JavaScript must be enabled to view this email address) / 402-483-7226
    Lincoln, Nebraska 68506
    United States

    Dartmouth Hitchcock Medical Center
    Jessica Harrington, CCRC / .(JavaScript must be enabled to view this email address) / 603-650-4649
    Lebanon, New Hampshire 03756
    United States

    University of Rochester
    Farheen Hussain / .(JavaScript must be enabled to view this email address) / 585-275-4715
    Rochester, New York 14642
    United States

    Hospital for Special Surgery
    Nicole Kassebaum / .(JavaScript must be enabled to view this email address) / 646-797-8592
    New York, New York 10021
    United States

    SUNY Upstate Medical University
    Tanya Perry / .(JavaScript must be enabled to view this email address) / 315-464-4998
    Syracuse, New York 13120
    United States

    Wake Forest University, School of Medicine
    Mozhdeh Marandi / .(JavaScript must be enabled to view this email address) / 336-713-8577
    Winston-Salem, North Carolina 27157
    United States

    Duke University
    Karen Grace, RN, BSN / .(JavaScript must be enabled to view this email address) / 909-668-2844
    Durham, North Carolina 27705
    United States

    Carolinas Medical Center Department of Neurology
    Carissa Ingram / .(JavaScript must be enabled to view this email address) / 704-446-0836
    Charlotte, North Carolina 27406
    United States

    Ohio State University Department of Neurology
    Sharon Chelnick / .(JavaScript must be enabled to view this email address) / 614-293-4973
    Columbus, Ohio 43221
    United States

    Oregon Health & Science University
    Diana Dimitrova / .(JavaScript must be enabled to view this email address) / 503-494-7269
    Portland, Oregon 97239
    United States

    Providence ALS Center
    Kimberly Siebers, RN, BSN / .(JavaScript must be enabled to view this email address) / 503-962-1171
    Portand, Oregon 97213
    United States

    University of Pennsylvania
    Kelly L Almasy / .(JavaScript must be enabled to view this email address) / 215-829-5041
    Philadelphia, Pennsylvania 19107
    United States

    University of Pittsburgh
    Danielle Rowlands / .(JavaScript must be enabled to view this email address) / 412-648-9053
    Pittsburgh, Pennsylvania 15213
    United States

    Penn State Hershey Neuroscience Clinics
    Beth Stephens / .(JavaScript must be enabled to view this email address) / 717-531-0003 ext 283395
    Hershey, Pennsylvania 17033
    United States

    Drexel Neurology
    Christine Barr, RN / .(JavaScript must be enabled to view this email address) / 267-507-2633
    Philadelphia, Pennsylvania 19107
    United States

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

    Baylor College of Medicine
    Claire MacAdam / .(JavaScript must be enabled to view this email address) / 713-798-5694
    Houston, Texas 77030
    United States

    Texas Neurology
    Janine McCloskey / .(JavaScript must be enabled to view this email address) / jmccloskey@texasneurology.com
    Dallas, Texas 75214
    United States

    University of Texas Health Science Center at San Antonio
    Pamela Kittrell, RN, MSN, CCRC / .(JavaScript must be enabled to view this email address) / 210-450-0524
    San Antonio, Texas 78229
    United States

    University of Virginia
    Amruta Joshi / .(JavaScript must be enabled to view this email address) / 434-982-0293
    Charlottesville, Virginia 22908
    United States

    The George Washington University
    Angela Kelly / .(JavaScript must be enabled to view this email address) / 202-741-2717
    Washington D.C. 20037
    United States

    West Virginia University Department of Neurology
    Patricia A Altemus, MS / .(JavaScript must be enabled to view this email address) / 304-598-4000 ext 75656
    Morgantown, West Virginia 26506
    United States

    Medical College of Wisconsin
    Clara Schindler Propsom / .(JavaScript must be enabled to view this email address) / 414-805-3260
    Milwaukee, Wisconsin 53226
    United States

    Hapital Notre Dame (CHUM) Centre Hospitalier de l'Universite de Montreal
    Ann Rusnov / .(JavaScript must be enabled to view this email address) / 514 890 8000
    Montreal, Quebec H2L4M1
    Canada

    QE II Health Sciences Centre
    Margo MacFarlane / .(JavaScript must be enabled to view this email address) / (902) 473-2728
    Halifax, Nova Scotia B3H 3A7
    Canada

    University of British Columbia
    Marife Fabros / .(JavaScript must be enabled to view this email address) / 604-737-6319
    Vancouver, British Columbia V5Z 2G9
    Canada

    CHU de Quebec: Hopital l'Enfant-Jesus
    Lucie Morel / .(JavaScript must be enabled to view this email address) / 418-649-0252
    Quebec, Quebec G1J 1Z4
    Canada

    Queen's University: Kingston General
    Adriana Breen / .(JavaScript must be enabled to view this email address) / 613-549-6666
    Kingston, Ontario K7L 2V7
    Canada

    Univ. of Toronto - Sunnybrook Health Sciences Centre
    Kristiana Salmon / .(JavaScript must be enabled to view this email address) / 514-398-1779
    Toronto, Ontario M4N 3M5
    Canada

    University of Alberta Hospital
    Michael Kreuzer / .(JavaScript must be enabled to view this email address) / 780-407-2944
    Edmonton, Alberta B3H 3A7
    Canada

    London Health Sciences
    Joan Martin / .(JavaScript must be enabled to view this email address) / 905-521-2100 ext 75232
    Hamilton, Ontario L8S 4K1
    Canada

    Heritage Medical Research
    Janet Petrillo / .(JavaScript must be enabled to view this email address) / 403-210-7006
    Calgary, Alberta T2N 4Z6
    Canada

    Stan Cassidy Centre for Rehabilitation
    Susan Brophy / .(JavaScript must be enabled to view this email address) / 506-447-4294
    Fredericton, New Brunswick E3B 0C7
    Canada

    McMaster University Medical Centre
    Joan Martin / .(JavaScript must be enabled to view this email address) / 905-521-2100 ext 75232
    Hamilton, Ontario L8S 4K1
    Canada

    Montreal Neurological Institute
    Marc Lemieux / .(JavaScript must be enabled to view this email address) / 514-398-2667
    Montreal, Quebec H3A 2B4
    Canada

    Charite Universitatsmedizin
    Dorit Strassenburg / .(JavaScript must be enabled to view this email address) / 49-30-450-6603-96
    Berlin, 13353
    Germany

    University of Ulm
    Sabine Raubold / .(JavaScript must be enabled to view this email address) / 49-731- 500 63006
    Ulm, 89081
    Germany

    Hannover Medical School
    Chantal Fischer / .(JavaScript must be enabled to view this email address) / 0049 (0) 511 532-8333
    Hannover, 30625
    Germany

    CHRU de Lille - Hôpital Roger Salengro
    Francine Niset-Ratel / .(JavaScript must be enabled to view this email address) / 0033 (0)6 07 50 34 20
    Lille, F-59037
    France

    CHU de Limoges - Hôpital Dupuytren
    Olivier Villeneuve / .(JavaScript must be enabled to view this email address) / 0033 (0)5 55 05 65 59
    Limoges, 87042
    France

    CHU Montepellier
    Sébastien Alphandéry / .(JavaScript must be enabled to view this email address) / 0033 (0)4 67 52 93 28
    Montpellier, 34295
    France

    Hapital de la Salpatrière
    Lucette Lacomblez / .(JavaScript must be enabled to view this email address) / 0033 (0)1 42 16 24 72
    Paris, Cedex 13
    France

    Kings College Hospital NHS Foundation Trust
    Andrew Dougherty / .(JavaScript must be enabled to view this email address) / +44 (0)20 7848 5355
    London, SE5 8AF
    United Kingdom

    Walton Centre for Neurology and Neurosurgery
    Louise Pate / .(JavaScript must be enabled to view this email address) / +44 (0)1515295666
    Liverpool, L9 7LJ
    United Kingdom

    John Radcliffe Hospital
    Edna Gonzalvo / .(JavaScript must be enabled to view this email address) / +44 (0)1865231556
    Oxford, OX3 9DU
    United Kingdom

    Sheffield Institute for Translational Neuroscience
    Theresa Walsh / .(JavaScript must be enabled to view this email address) / 44 -0-114-222-2226
    Sheffield,
    United Kingdom

    Barts and the London MND & the Centre Royal London Hospital
    Anna Belin / .(JavaScript must be enabled to view this email address) / 44-020- 788-27150
    Whitechapel, London,
    United Kingdom

    Trinity College, Beaumont Hospital
    Liz Fogarty / .(JavaScript must be enabled to view this email address) / 00353-1-809-3874
    Dublin, 9
    Ireland