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).

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:

Drug, N/A

Intervention Name:

CK-2017357, Placebo tablets, Riluzole



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?


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:


Participant Duration:

approximately 20 weeks

Estimated Enrollment:


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.

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Minimum Age:

18 Years

Maximum Age:


Can participants use Riluzole?


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

University of Alberta Hospital

Edmonton, Alberta B3H 3A7

University of British Columbia

Vancouver, British Columbia V5Z 2G9

Stan Cassidy Centre for Rehabilitation

Fredericton, New Brunswick E3B 0C7

QE II Health Sciences Centre

Halifax, Nova Scotia B3H 3A7

McMaster University Medical Centre

Hamilton, Ontario L8S 4K1

Queen's University : Kingston General

Kingston, Ontario K7L 2V7

London Health Sciences

London, Ontario N6A 5A5

Univ. of Toronto - Sunnybrook Health Sciences Centre

Toronto, Ontario M4N 3M5

Hôpital Notre Dame (CHUM) Centre Hospitalier de l'Universite de Montreal

Montreal, Quebec H2L4M1

Montreal Neurological Institute

Montreal, Quebec H3A 2B4

CHU de Quebec: Hopital l'Enfant-Jesus

Quebec G1J 1Z4

Hôpital Bretonneau

Tours 37000

Hôpital de la Salpêtrière

Paris Cedex 13

Hôpital Archet 1

Nice 06602

CHU Montepellier

Montpellier 34295 Montpellier Cedex 5

Hôpital La Timone Adulte

Marseille 13005

CHU de Limoges - Hôpital Dupuytren

Limoges 87042 LIMOGES CEDEX

CHRU de Lille - Hôpital Roger Salengro

Lille F-59037 LILLE cedex

Charite Universitätsmedizin

Berlin 13353

Hannover Medical School

Hannover 30625

University of Ulm

Ulm 89081

Trinity College, Beaumont Hospital

Dublin 9

Universitair Medisch Centrum Utrecht

Utrecht 3584 CX

Hospital Carlos III

Madrid 28029

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