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A Study to Evaluate Efficacy, Safety and Tolerability of CK-2127107 in Patients With Amyotrophic Lateral Sclerosis (ALS) (FORTITUDE-ALS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03160898
Recruitment Status : Completed
First Posted : May 19, 2017
Last Update Posted : February 17, 2020
Astellas Pharma Inc
Information provided by (Responsible Party):

Brief Summary:
The purpose of this study is to assess the effect of CK-2127107 versus placebo on respiratory function and other measures of skeletal muscle function in patients with ALS.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: CK-2127107 Drug: Placebo Phase 2

Detailed Description:
Enrolled participants will be dosed with CK-2127107 150, 300, 450 mg or placebo twice daily (300, 600, 900 mg/day or placebo) for a period of 12 weeks.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 458 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : July 24, 2017
Actual Primary Completion Date : March 7, 2019
Actual Study Completion Date : March 7, 2019

Arm Intervention/treatment
Experimental: CK-2127107 300 mg
Participants will receive CK-2127107 300 mg for 12 weeks
Drug: CK-2127107
Other Name: reldesemtiv

Experimental: CK-2127107 600 mg
Participants will receive CK-2127107 600 mg for 12 weeks
Drug: CK-2127107
Other Name: reldesemtiv

Experimental: CK-2127107 900 mg
Participants will receive CK-2127107 900 mg for 12 weeks
Drug: CK-2127107
Other Name: reldesemtiv

Placebo Comparator: Placebo
Participants will receive placebo for 12 weeks
Drug: Placebo

Primary Outcome Measures :
  1. Change from baseline to Week 12 in the percent predicted slow vital capacity (SVC) [ Time Frame: Baseline to Week 12 ]

Secondary Outcome Measures :
  1. Slope of change from baseline in the mega-score of muscle strength measured by hand held dynamometry and handgrip dynamometry from baseline to Week 12 [ Time Frame: Baseline to Week 12 ]
  2. Change from baseline to Week 12 in the ALS Functional Rating Scale - Revised (ALSFRS-R) [ Time Frame: Baseline to Week 12 ]
  3. Mean plasma concentrations over time of CK-2127107 at Week 12 [ Time Frame: Day 1 to Week 12 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03160898

Hide Hide 64 study locations
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United States, Arizona
St. Joseph's Hospital and Medical Center - Barrow Neurological Clinics
Phoenix, Arizona, United States, 85013
United States, California
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
University of California Irvine
Orange, California, United States, 92868
Forbes Norris MDA/ALS Research Center
San Francisco, California, United States, 94115
Stanford Hospital and Clinics
Stanford, California, United States, 94305
United States, Colorado
University of Colorado Hospital Anschutz Outpatient Pavilion
Aurora, Colorado, United States, 80045
United States, Connecticut
Hospital for Special Care
New Britain, Connecticut, United States, 06053
United States, District of Columbia
George Washington University Medical Faculty Associates
Washington, District of Columbia, United States, 20037
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
Mayo Clinic
Jacksonville, Florida, United States, 32224
Carol & Frank Morsani Center for Advanced Healthcare - University of South Florida
Tampa, Florida, United States, 33612
United States, Georgia
Emory Clinic
Atlanta, Georgia, United States, 30322
United States, Illinois
Duchossois Center for Advanced Medicine
Chicago, Illinois, United States, 60637
United States, Indiana
IU Health Neuroscience Center of Excellence
Indianapolis, Indiana, United States, 46202
United States, Iowa
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
Johns Hopkins University - Outpatient Center
Baltimore, Maryland, United States, 21287
United States, Massachusetts
University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School
Worcester, Massachusetts, United States, 01655
United States, Michigan
Michigan Medicine
Ann Arbor, Michigan, United States, 48109
Henry Ford Health System
Detroit, Michigan, United States, 48202
United States, Minnesota
Hennepin County Medical Center
Minneapolis, Minnesota, United States, 55415
Mayo Clinic
Rochester, Minnesota, United States, 55905
United States, Missouri
Saint Louis University, Department of Neurology
Saint Louis, Missouri, United States, 63104
Washington University School of Medicine
Saint Louis, Missouri, United States, 63110
United States, Nebraska
Neurology Associates, P.C.
Lincoln, Nebraska, United States, 68506
United States, New York
Hospital For Special Surgery
New York, New York, United States, 10021
Neurological Institute, Columbia University Medical Center
New York, New York, United States, 10032
SUNY Upstate Medical University
Syracuse, New York, United States, 13210
United States, North Carolina
Neurosciences Institute, Neurology - Charlotte
Charlotte, North Carolina, United States, 28207
Duke Neurological Disorders Clinic
Durham, North Carolina, United States, 27705
Wake Forest School of Medicine
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
Cleveland Clinic
Cleveland, Ohio, United States, 44195
The Ohio State University Wexner Medical Center
Columbus, Ohio, United States, 43210
United States, Oregon
Providence Brain and Spine Institute ALS Center
Portland, Oregon, United States, 97213
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Temple University School of Medicine
Philadelphia, Pennsylvania, United States, 19140
United States, Tennessee
Vanderbilt University Medical Center - Clinical Research Center
Nashville, Tennessee, United States, 37232
United States, Texas
Texas Neurology
Dallas, Texas, United States, 75214
Houston Methodist Hospital
Houston, Texas, United States, 77030
UTHSCSA Medical Arts and Research Center
San Antonio, Texas, United States, 78229
United States, Vermont
University of Vermont Medical Center
Burlington, Vermont, United States, 05405
United States, Virginia
University of Virginia Health System
Charlottesville, Virginia, United States, 22908
VCU Health - Ambulatory Care Center (ACC)
Richmond, Virginia, United States, 23298
United States, Washington
University of Washington Medical Center
Seattle, Washington, United States, 98195
United States, West Virginia
West Virginia University, Dept. of Neurology
Morgantown, West Virginia, United States, 26506-9180
United States, Wisconsin
Froedtert Memorial Lutheran Hospital
Milwaukee, Wisconsin, United States, 53226
Australia, New South Wales
Brain and Mind Centre, The University of Sydney
Camperdown, New South Wales, Australia, 2050
Department of Neurology, Westmead Hospital
Westmead, New South Wales, Australia, 2145
Australia, Queensland
Royal Brisbane and Women's Hospital
Herston, Queensland, Australia, 4029
Australia, South Australia
Flinders Medical Centre
Bedford Park, South Australia, Australia, 5042
Australia, Western Australia
The Perron Institute for Neurological and Translation Science
Nedlands, Western Australia, Australia, 6009
Canada, Alberta
University of Calgary, Heritage Medical Research Center
Calgary, Alberta, Canada, T2N 4Z6
Edmonton Kaye Clinic
Edmonton, Alberta, Canada, T6GT 1Z1
Canada, Ontario
McMaster University Medical Centre
Hamilton, Ontario, Canada, L8N 3Z5
London Health Sciences Centre University Hospital
London, Ontario, Canada, N6A 5A5
Sunnybrook Health Science Centre
Toronto, Ontario, Canada, M4N 3M5
Canada, Quebec
Montreal Neurological Institute and Hospital
Montreal, Quebec, Canada, H3A 2B4
Centre de recherche du Centre Hospitalier de l'Universite de Montreal
Montréal, Quebec, Canada, H2X 0A9
Canada, Saskatchewan
Saskatoon City Hospital
Saskatoon, Saskatchewan, Canada, S7H 0G9
CHU de Quebec-Universite Laval, Hopital de l'Enfant Jesus
Quebec, Canada, G1J 1Z4
Beaumont Hospital
Dublin, Ireland, Dublin 9
University Medical Center Utrecht
Utrecht, Netherlands, 3584 CX
Hospital San Rafael Servicio de Neurologia
Madrid, Spain, 28016
Sponsors and Collaborators
Astellas Pharma Inc
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Study Director: Study Director MD Cytokinetics
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Cytokinetics Identifier: NCT03160898    
Other Study ID Numbers: CY 5022
First Posted: May 19, 2017    Key Record Dates
Last Update Posted: February 17, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cytokinetics:
Amyotrophic Lateral Sclerosis
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases