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)

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.

• 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

  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

  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

  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

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

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

  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

  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

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

  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

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

  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

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

  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

  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

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

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

  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

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

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

  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

  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

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

  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

  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

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

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

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

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

  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

  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

  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