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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03160898
Recruitment Status : Recruiting
First Posted : May 19, 2017
Last Update Posted : October 2, 2018
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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 445 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
Estimated Primary Completion Date : March 2019
Estimated Study Completion Date : March 2019

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

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

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

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

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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

Contact: MD Cytokinetics 650-624-2929

  Hide Study Locations
United States, Arizona
St. Joseph's Hospital and Medical Center - Barrow Neurological Clinics Recruiting
Phoenix, Arizona, United States, 85013
Contact: Jacquelyn Nicolari    602-406-6606   
United States, California
Cedars-Sinai Medical Center Recruiting
Los Angeles, California, United States, 90048
Contact: Koral Wheeler   
University of California Irvine Recruiting
Orange, California, United States, 92868
Contact: Ivonne Turner   
Forbes Norris MDA/ALS Research Center Recruiting
San Francisco, California, United States, 94115
Contact: Marguerite Engel   
Stanford Hospital and Clinics Recruiting
Stanford, California, United States, 94305
Contact: Michael Nguyen    650-724-6147   
United States, Colorado
University of Colorado Hospital Anschutz Outpatient Pavilion Recruiting
Aurora, Colorado, United States, 80045
Contact: Nicola Haakonsen   
United States, Connecticut
Hospital for Special Care Recruiting
New Britain, Connecticut, United States, 06053
United States, District of Columbia
George Washington University Medical Faculty Associates Recruiting
Washington, District of Columbia, United States, 20037
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Mayo Clinic Recruiting
Jacksonville, Florida, United States, 32224
Contact: Pamela Desaro   
Carol & Frank Morsani Center for Advanced Healthcare - University of South Florida Recruiting
Tampa, Florida, United States, 33612
United States, Georgia
Emory Clinic Recruiting
Atlanta, Georgia, United States, 30322
Contact: Meraida Polak   
Contact: Christina Fournier, MD   
United States, Illinois
Duchossois Center for Advanced Medicine Recruiting
Chicago, Illinois, United States, 60637
Contact: Jacquelyn Hill    773-702-4610   
United States, Indiana
IU Health Neuroscience Center of Excellence Recruiting
Indianapolis, Indiana, United States, 46202
United States, Iowa
University of Iowa Hospitals and Clinics Recruiting
Iowa City, Iowa, United States, 52242
Contact: Jeri Sieren   
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Ayla McCalley   
United States, Maryland
Johns Hopkins University - Outpatient Center Recruiting
Baltimore, Maryland, United States, 21287
Contact: Kristin M. Riley, PhD    410-955-8511   
United States, Massachusetts
University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School Recruiting
Worcester, Massachusetts, United States, 01655
United States, Michigan
Michigan Medicine Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Jayna Duell    734-763-9037   
Henry Ford Health System Recruiting
Detroit, Michigan, United States, 48202
Contact: Ashkhen Movsisyan    313-916-4123   
United States, Minnesota
Hennepin County Medical Center Recruiting
Minneapolis, Minnesota, United States, 55415
Contact: Cindy Rohde, RN    612-873-2607   
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
United States, Missouri
Saint Louis University, Department of Neurology Recruiting
Saint Louis, Missouri, United States, 63104
Washington University School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
United States, Nebraska
Neurology Associates, P.C. Recruiting
Lincoln, Nebraska, United States, 68506
Contact: Kelli Ferguson   
Contact: Desirae Eschiti, LPN   
United States, New York
Hospital For Special Surgery Recruiting
New York, New York, United States, 10021
Contact: Shara Holzberg   
Neurological Institute, Columbia University Medical Center Recruiting
New York, New York, United States, 10032
SUNY Upstate Medical University Recruiting
Syracuse, New York, United States, 13210
United States, North Carolina
Neurosciences Institute, Neurology - Charlotte Recruiting
Charlotte, North Carolina, United States, 28207
Duke Neurological Disorders Clinic Recruiting
Durham, North Carolina, United States, 27705
Contact: Karen Grace   
Wake Forest School of Medicine Recruiting
Winston-Salem, North Carolina, United States, 27157
Contact: Mozhdeh Marandi   
United States, Ohio
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44195
The Ohio State University Wexner Medical Center Recruiting
Columbus, Ohio, United States, 43210
Contact: Ifeanyi Okoh   
United States, Oregon
Providence Brain and Spine Institute ALS Center Recruiting
Portland, Oregon, United States, 97213
Contact: Arlena Cummings    503-962-1171   
Oregon Health & Science University Recruiting
Portland, Oregon, United States, 97239
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center Recruiting
Hershey, Pennsylvania, United States, 17033
Temple University School of Medicine Recruiting
Philadelphia, Pennsylvania, United States, 19140
Contact: Kathleen Hatala    215-707-4171   
United States, Tennessee
Vanderbilt University Medical Center - Clinical Research Center Recruiting
Nashville, Tennessee, United States, 37232
Contact: Diana Davis    615-322-8957   
United States, Texas
Texas Neurology Recruiting
Dallas, Texas, United States, 75214
Contact: Todd Morgan    214-827-3610 ext 228   
Houston Methodist Hospital Recruiting
Houston, Texas, United States, 77030
Contact: Luis F Lay, Jr.    713-441-3057   
UTHSCSA Medical Arts and Research Center Recruiting
San Antonio, Texas, United States, 78229
Contact: Pamela Kittrell, MSN RN    210-450-0524   
United States, Vermont
University of Vermont Medical Center Recruiting
Burlington, Vermont, United States, 05405
United States, Virginia
University of Virginia Health System Recruiting
Charlottesville, Virginia, United States, 22908
Contact: Mary Wagoner   
VCU Health - Ambulatory Care Center (ACC) Recruiting
Richmond, Virginia, United States, 23298
Contact: Tamika Walthour   
United States, Washington
University of Washington Medical Center Recruiting
Seattle, Washington, United States, 98195
Contact: Laura Sissons-Ross   
United States, West Virginia
West Virginia University, Dept. of Neurology Recruiting
Morgantown, West Virginia, United States, 26506-9180
United States, Wisconsin
Froedtert Memorial Lutheran Hospital Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Lynn Wheeler   
Australia, New South Wales
Brain and Mind Centre, The University of Sydney Recruiting
Camperdown, New South Wales, Australia, 2050
Contact: Eleanor Ramsey    61 2 9351 0976   
Department of Neurology, Westmead Hospital Recruiting
Westmead, New South Wales, Australia, 2145
Australia, Queensland
Royal Brisbane and Women's Hospital Recruiting
Herston, Queensland, Australia, 4029
Australia, South Australia
Flinders Medical Centre Recruiting
Bedford Park, South Australia, Australia, 5042
Australia, Western Australia
The Perron Institute for Neurological and Translation Science Recruiting
Nedlands, Western Australia, Australia, 6009
Canada, Alberta
University of Calgary, Heritage Medical Research Center Recruiting
Calgary, Alberta, Canada, T2N 4Z6
Contact: Jose Antonio Martinez   
Edmonton Kaye Clinic Recruiting
Edmonton, Alberta, Canada, T6GT 1Z1
Contact: Shelley Wrona   
Canada, Ontario
McMaster University Medical Centre Recruiting
Hamilton, Ontario, Canada, L8N 3Z5
Contact: Daniela Trapsa   
London Health Sciences Centre University Hospital Recruiting
London, Ontario, Canada, N6A 5A5
Sunnybrook Health Science Centre Recruiting
Toronto, Ontario, Canada, M4N 3M5
Canada, Quebec
Montreal Neurological Institute and Hospital Recruiting
Montreal, Quebec, Canada, H3A 2B4
Centre de recherche du Centre Hospitalier de l'Universite de Montreal Recruiting
Montréal, Quebec, Canada, H2X 0A9
Contact: Stefanie Houle   
Canada, Saskatchewan
Saskatoon City Hospital Recruiting
Saskatoon, Saskatchewan, Canada, S7H 0G9
Contact: Janet DeGirolamo, RN    306-978-8308   
CHU de Quebec-Universite Laval, Hopital de l'Enfant Jesus Recruiting
Quebec, Canada, G1J 1Z4
Beaumont Hospital Recruiting
Dublin, Ireland, Dublin 9
University Medical Center Utrecht Recruiting
Utrecht, Netherlands, 3584 CX
Contact: Tommy Bunte   
Hospital San Rafael Servicio de Neurologia Recruiting
Madrid, Spain, 28016
Sponsors and Collaborators
Astellas Pharma Inc
Study Director: Study Director MD Cytokinetics

Responsible Party: Cytokinetics Identifier: NCT03160898     History of Changes
Other Study ID Numbers: CY 5022
First Posted: May 19, 2017    Key Record Dates
Last Update Posted: October 2, 2018
Last Verified: October 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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:
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