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).Study Status:
Not recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug, N/A
Intervention Name:
CK-2017357, Placebo tablets, Riluzole
Placebo:
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
Jinsy Andrews, MD, Cytokinetics, Inc., Jeremy Shefner, MD, PhD, State University of New York - Upstate Medical University, Jeremy Shefner, MD, PhD, State University of New York - Upstate Medical University
Clinicaltrials.gov ID:
Neals Affiliated?
Yes
Coordinating Center Contact Information
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 who tolerate the open-label 125 mg of CK-2017357 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:
711
Estimated Study Start Date:
10 / 31 / 2012
Estimated Study Completion Date:
03 / 01 / 2014
Posting Last Modified Date:
03 / 31 / 2020
Date Study Added to neals.org:
10 / 18 / 2012
***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.
Minimum Age:
18 Years
Maximum Age:
N/A
Can participants use Riluzole?
Yes
Inclusion Criteria:1. Able to comprehend and willing to sign an Informed Consent Form (ICF)
2. Male or female 18 years of age or older
3. 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)
4. Upright Slow Vital Capacity (SVC) >50 % of predicted for age, height and sex
5. At least 4 of the 12 ALSFRS-R questions must be scored 2 or 3
6. Diminished but measurable maximum voluntary grip strength in at least one hand; i.e., between 10 and 50 pounds (females) and 10 and 70 pounds (males)
7. Able to swallow tablets without crushing
8. A caregiver (if one is needed) who can and will observe and report the patient's status
9. Pre-study clinical laboratory findings within normal range or, if outside of the normal range, deemed not clinically significant by the Investigator
10. 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
11. 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
12. 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:
1. 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
2. Patients with a diaphragm pacing system (DPS) at study entry or who anticipate DPS placement during the course of the study
3. Body Mass Index (BMI) of 19.0 kg/m2 or lower
4. Unwilling to discontinue tizanidine and theophylline-containing medications during study participation
5. Serum chloride < 100 mmol/L
6. Neurological impairment due to a condition other than ALS, including history of transient ischemic attack (TIA) within the past year
7. 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
8. Has taken any investigational study drug within 30 days or 5 half-lives of the prior agent, whichever is greater, prior to dosing
9. Previously received CK-2017357 in any previous clinical trial
Barrow Neurology
Phoenix, Arizona
85013
United States
University of California, San Diego
La Jolla, California
92093
United States
UC Irvine ALS & Neuromuscular Center
Orange, California
92868
United States
Coordinated Clinical Research
San Diego, California
92103
United States
California Pacific Medical Center Forbes Norris MDA/ALS Research Center
San Francisco, California
94115
United States
Hospital for Special Care
New Britain, Connecticut
06053
United States
The George Washington University
Washington, District of Columbia
20037
United States
Mayo Clinic Florida Department of Neurology
Jacksonville, Florida
32224
United States
Emory University, School of Medicine
Atlanta, Georgia
30322
United States
Georgia Health Sciences University
Augusta, Georgia
30912
United States
Indiana University Department of Neurology
Indianapolis, Indiana
46202
United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa
52242
United States
University of Kansas
Kansas City, Kansas
66160
United States
Johns Hopkins University
Baltimore, Maryland
21205
United States
Massachusetts General Hospital
Boston, Massachusetts
02114
United States
University of Massachusetts Medical School
Worcester, Massachusetts
01655
United States
University of Michigan
Ann Arbor, Michigan
48109
United States
Henry Ford Hospital
Detroit, Michigan
48202
United States
St Mary's Healthcare
Grand Rapids, Michigan
49503
United States
Hennepin County Medical Center - Berman Center for Research
Minneapolis, Minnesota
55415
United States
Saint Louis University
Saint Louis, Missouri
63104
United States
Washington University
Saint Louis, Missouri
63110
United States
Neurology Associates
Lincoln, Nebraska
68506
United States
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire
03756
United States
University of Rochester
Rochester, New York
14642
United States
SUNY Upstate Medical University
Syracuse, New York
13120
United States
Hospital for Special Surgery
New York, New York
10021
United States
Carolinas Medical Center Department of Neurology
Charlotte, North Carolina
27406
United States
Duke University
Durham, North Carolina
27705
United States
Wake Forest University, School of Medicine
Winston-Salem, North Carolina
27157
United States
Ohio State University Department of Neurology
Columbus, Ohio
43221
United States
Providence ALS Center
Portland, Oregon
97213
United States
Oregon Health & Science University
Portland, Oregon
97239
United States
University of Pittsburgh
Pittsburgh, Pennsylvania
15213
United States
Penn State Hershey Neuroscience Clinics
Hershey, Pennsylvania
17033
United States
University of Pennsylvania
Philadelphia, Pennsylvania
19107
United States
Drexel Neurology
Philadelphia, Pennsylvania
19107
United States
Vanderbilt University Medical Center
Nashville, Tennessee
37232
United States
Texas Neurology
Dallas, Texas
75214
United States
Baylor College of Medicine
Houston, Texas
77030
United States
UTHSCSA Department of Neurology
San Antonio, Texas
78229
United States
University of Virginia
Charlottesville, Virginia
22908
United States
West Virginia University Department of Neurology
Morgantown, West Virginia
26506
United States
Medical College of Wisconsin
Milwaukee, Wisconsin
53226
United States
Heritage Medical Research
Calgary, Alberta
T2N 4Z6
Canada
University of Alberta Hospital
Edmonton, Alberta
B3H 3A7
Canada
University of British Columbia
Vancouver, British Columbia
V5Z 2G9
Canada
Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick
E3B 0C7
Canada
QE II Health Sciences Centre
Halifax, Nova Scotia
B3H 3A7
Canada
McMaster University Medical Centre
Hamilton, Ontario
L8S 4K1
Canada
Queen's University : Kingston General
Kingston, Ontario
K7L 2V7
Canada
London Health Sciences
London, Ontario
N6A 5A5
Canada
Univ. of Toronto - Sunnybrook Health Sciences Centre
Toronto, Ontario
M4N 3M5
Canada
Hôpital Notre Dame (CHUM) Centre Hospitalier de l'Universite de Montreal
Montreal, Quebec
H2L4M1
Canada
Montreal Neurological Institute
Montreal, Quebec
H3A 2B4
Canada
CHU de Quebec: Hopital l'Enfant-Jesus
Quebec G1J 1Z4
Canada
Hôpital Bretonneau
Tours 37000
France
Hôpital de la Salpêtrière
Paris Cedex 13
France
Hôpital Archet 1
Nice 06602
France
CHU Montepellier
Montpellier 34295 Montpellier Cedex 5
France
Hôpital La Timone Adulte
Marseille 13005
France
CHU de Limoges - Hôpital Dupuytren
Limoges 87042 LIMOGES CEDEX
France
CHRU de Lille - Hôpital Roger Salengro
Lille F-59037 LILLE cedex
France
Charite Universitätsmedizin
Berlin 13353
Germany
Hannover Medical School
Hannover 30625
Germany
University of Ulm
Ulm 89081
Germany
Trinity College, Beaumont Hospital
Dublin 9
Ireland
Universitair Medisch Centrum Utrecht
Utrecht 3584 CX
Netherlands
Hospital Carlos III
Madrid 28029
Spain
Barts and the London MND & the Centre Royal London Hospital
Whitechapel, London
United Kingdom
Walton Centre for Neurology and Neurosurgery
Liverpool L9 7LJ
United Kingdom
Kings College Hospital NHS Foundation Trust
London SE5 8AF
United Kingdom
John Radcliffe Hospital
Oxford OX3 9DU
United Kingdom
Plymouth Hospitals NHS Trust
Plymouth PL6 8DH
United Kingdom
Sheffield Institute for Translational Neuroscience
Sheffield S10 2HQ
United Kingdom