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 Amyotrophic Lateral Sclerosis (ALS)

Study Purpose:

The purpose of this study was to assess the effect of CK-2127107 (hereafter referred to as reldesemtiv) versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

Reldesemtiv, Placebo




Phase 2

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

MD Cytokinetics, Cytokinetics

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

Full Study Summary:

This was a double-blind, randomized, placebo-controlled, dose ranging study of reldesemtiv in patients with ALS. Eligible patients were randomized (1:1:1:1) to receive placebo or one of three doses of reldesemtiv (150, 300, or 450 mg twice daily) for 12 weeks. Randomization was stratified by riluzole concomitant use/non-use and edaravone concomitant use/non-use. Concomitant riluzole and edaravone were allowed as long as the riluzole dose had been stable for at least 30 days prior to screening and edaravone had been taken for 2 cycles prior to screening; these drugs could not be initiated during the study.

A total of 7 study visits were planned: screening, Day 1 (first dosing day), Weeks 2, 4, 8, and 12, and follow-up (4 weeks after the last dose of study drug). Study drug (placebo or reldesemtiv) was to be taken twice daily, approximately 12 hours (± 2 hours) apart and within 2 hours following a meal.

Study Sponsor:


Participant Duration:

12 weeks

Estimated Enrollment:


Estimated Study Start Date:

07 / 24 / 2017

Estimated Study Completion Date:

03 / 07 / 2019

Posting Last Modified Date:

09 / 11 / 2020

Date Study Added to neals.org:

05 / 19 / 2017

Minimum Age:

18 Years

Maximum Age:

80 Years

Can participants use Riluzole?


Inclusion Criteria:

- Diagnosis of familial or sporadic ALS ≤ 24 months prior to screening

- Upright Slow Vital Capacity (SVC) ≥ 60% 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 after receiving the first dose of study drug until 10 weeks after the last dose 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 or abstain from heterosexual intercourse from Screening until 10 weeks after last dose of study drug

- 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.

- Patients on edaravone must have completed at least 2 cycles of dosing with edaravone at the time of screening or have not taken edaravone for at least 30 days prior to screening and not planning to start edaravone 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

St. Joseph's Hospital and Medical Center - Barrow Neurological Clinics

Phoenix, Arizona 85013
United States

Cedars-Sinai Medical Center

Los Angeles, California 90048
United States

University of California Irvine

Orange, California 92868
United States

Forbes Norris MDA/ALS Research Center

San Francisco, California 94115
United States

Stanford Hospital and Clinics

Stanford, California 94305
United States

University of Colorado Hospital Anschutz Outpatient Pavilion

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

University of Florida

Gainesville, Florida 32610
United States

Mayo Clinic

Jacksonville, Florida 32224
United States

Carol & Frank Morsani Center for Advanced Healthcare - University of South Florida

Tampa, Florida 33612
United States

Emory Clinic

Atlanta, Georgia 30322
United States

Duchossois Center for Advanced Medicine

Chicago, Illinois 60637
United States

IU Health Neuroscience Center of Excellence

Indianapolis, Indiana 46202
United States

University of Iowa Hospitals and Clinics

Iowa City, Iowa 52242
United States

University of Kansas Medical Center

Kansas City, Kansas 66160
United States

Johns Hopkins University - Outpatient Center

Baltimore, Maryland 21287
United States

University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School

Worcester, Massachusetts 01655
United States

Michigan Medicine

Ann Arbor, Michigan 48109
United States

Henry Ford Health System

Detroit, Michigan 48202
United States

Hennepin County Medical Center

Minneapolis, Minnesota 55415
United States

Mayo Clinic

Rochester, Minnesota 55905
United States

Washington University School of Medicine

Saint Louis, Missouri 63110
United States

Saint Louis University, Department of Neurology

Saint Louis, Missouri 63104
United States

Neurology Associates, P.C.

Lincoln, Nebraska 68506
United States

Hospital For Special Surgery

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

Charlotte, North Carolina 28207
United States

Duke Neurological Disorders Clinic

Durham, North Carolina 27705
United States

Wake Forest School of Medicine

Winston-Salem, North Carolina 27157
United States

Cleveland Clinic

Cleveland, Ohio 44195
United States

The Ohio State University Wexner Medical Center

Columbus, Ohio 43210
United States

Providence Brain and Spine Institute ALS Center

Portland, Oregon 97213
United States

Oregon Health & Science University

Portland, Oregon 97239
United States

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania 17033
United States

Temple University School of Medicine

Philadelphia, Pennsylvania 19140
United States

Vanderbilt University Medical Center - Clinical Research Center

Nashville, Tennessee 37232
United States

Texas Neurology

Dallas, Texas 75214
United States

Houston Methodist Hospital

Houston, Texas 77030
United States

UTHSCSA Medical Arts and Research Center

San Antonio, Texas 78229
United States

University of Vermont Medical Center

Burlington, Vermont 05405
United States

University of Virginia Health System

Charlottesville, Virginia 22908
United States

VCU Health - Ambulatory Care Center (ACC)

Richmond, Virginia 23298
United States

University of Washington Medical Center

Seattle, Washington 98195
United States

West Virginia University, Dept. of Neurology

Morgantown, West Virginia 26506-9180
United States

Froedtert Memorial Lutheran Hospital

Milwaukee, Wisconsin 53226
United States

Brain and Mind Centre, The University of Sydney

Camperdown, New South Wales 2050

Royal Brisbane and Women's Hospital

Herston, Queensland 4029

Flinders Medical Centre

Bedford Park, South Australia 5042

The Perron Institute for Neurological and Translation Science

Nedlands, Western Australia 6009

Department of Neurology, Westmead Hospital

Westmead, New South Wales 2145

Montreal Neurological Institute and Hospital

Montreal, Quebec H3A 2B4

CHU de Quebec-Universite Laval, Hopital de l'Enfant Jesus

Quebec G1J 1Z4

Saskatoon City Hospital

Saskatoon, Saskatchewan S7H 0G9

Centre de recherche du Centre Hospitalier de l'Universite de Montreal

Montréal, Quebec H2X 0A9

London Health Sciences Centre University Hospital

London, Ontario N6A 5A5

Sunnybrook Health Science Centre

Toronto, Ontario M4N 3M5

McMaster University Medical Centre

Hamilton, Ontario L8N 3Z5

Edmonton Kaye Clinic

Edmonton, Alberta T6GT 1Z1

University of Calgary, Heritage Medical Research Center

Calgary, Alberta T2N 4Z6

Beaumont Hospital

Dublin Dublin 9

University Medical Center Utrecht

Utrecht 3584 CX

Hospital San Rafael Servicio de Neurologia

Madrid 28016