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 ALS (FORTITUDE-ALS)

Study Purpose:

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.

Disease:

Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Enrolling

Phase:

Phase II

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

Clinicaltrials.gov ID (11 digit #):

NCT03160898

Neals Affiliated?

Yes

Coordinating Center Contact Information

MD Cytokinetics / .(JavaScript must be enabled to view this email address) / 650-624-2929
.(JavaScript must be enabled to view this email address)

Full Study Summary:

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 Sponsor:

Cytokinetics

Participant Duration:

12 weeks

Estimated Enrollment:

445

Estimated Study Start Date:

07/24/2017

Estimated Study Completion Date:

07/01/2018

Posting Last Modified Date:

04/16/2018

Date Study Added to alsconsortium.org:

09/01/2017

More Information

Watch this webinar from January 30, 2018 to learn about the FORTITUDE-ALS trial design and hopes for the drug: recording link.
Eligibility Criteria

Gender:
Female, Male
Minimum Age:
18
Maximum Age:
80
Min Vital Capacity (% predicted normal):
65

Time since Symptom Onset:

Time since Diagnosis:
<24 months
Can participants use Riluzole?
Yes

Inclusion Criteria:
Diagnosis of familial or sporadic ALS ≤ 24 months prior to screening
Upright Slow Vital Capacity (SVC) ≥ 65% 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 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
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
Site Contact Information

Barrow Neurological Institute
Jacquelyn Nicolari / .(JavaScript must be enabled to view this email address) / 602-406-6606
St. Joseph's Hospital and Medical Center
Phoenix, Arizona 85013
United States
University of California Irvine
Ivonne Turner / .(JavaScript must be enabled to view this email address) / Orange, California 92868
United States
Forbes Norris MDA/ALS Research Center
Marguerite Engel / .(JavaScript must be enabled to view this email address) / San Francisco, California 94115
United States
Stanford Hospital and Clinics
Michael Nguyen / .(JavaScript must be enabled to view this email address) / 650-724-6147
Stanford, California 94305
United States
Cedars-Sinai Medical Center
Koral Wheeler / .(JavaScript must be enabled to view this email address) / Los Angeles , California 90048
United States
University of Colorado Hospital Anschutz Outpatient Pavilion
Nicola Haakonsen / .(JavaScript must be enabled to view this email address) / 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
Mayo Clinic
Pamela Desaro / .(JavaScript must be enabled to view this email address) / Jacksonville, Florida 32224
United States
University of Florida
Gainesville, Florida 32610
United States
University of South Florida, Carol & Frank Morsani Center for Advanced Healthcare
Tampa, Florida 33612
United States
Emory ALS Clinic
Meraida Polak / .(JavaScript must be enabled to view this email address) / Atlanta, Georgia 30322
United States
Duchossois Center for Advanced Medicine
Jacquelyn Hill / .(JavaScript must be enabled to view this email address) / 773-702-4610
Chicago, Illinois 60637
United States
IU Health Neuroscience Center of Excellence
Indianapolis, Indiana 46202
United States
University of Iowa Hospitals and Clinics
Jeri Sieren / .(JavaScript must be enabled to view this email address) / Iowa City, Iowa 52242
United States
University of Kansas Medical Center
Ayla McCalley / .(JavaScript must be enabled to view this email address) / Kansas City , Kansas 66160
United States
Johns Hopkins University - Outpatient Center
Kristin M. Riley / .(JavaScript must be enabled to view this email address) / 410-955-8511
Baltimore, Maryland 21287
United States
University of Massachusetts Medical School
Worcester, Massachusetts 01655
United States
Michigan Medicine
Jayna Duell / .(JavaScript must be enabled to view this email address) / 734-763-9037
Ann Arbor, Michigan 48109
United States
Henry Ford Health System
Ashkhen Movsisyan / .(JavaScript must be enabled to view this email address) / 313-916-4123
Detroit, Michigan 48202
United States
Hennepin County Medical Center
Cindy Rohde / .(JavaScript must be enabled to view this email address) / 612-873-2607
Minneapolis, Minnesota 55415
United States
Mayo Clinic
Rochester, Minnesota 55905
United States
Saint Louis University, Department of Neurology
Saint Louis, Missouri 63104
United States
Washington University School of Medicine
Saint Louis, Missouri 63110
United States
Neurology Associates, P.C.
Kelli Ferguson / .(JavaScript must be enabled to view this email address) / Desirae Eschiti / .(JavaScript must be enabled to view this email address) / Lincoln, Nebraska 68506
United States
Hospital For Special Surgery
Shara Holzberg / .(JavaScript must be enabled to view this email address) / 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, North Carolina 28207
United States
Duke Neurological Disorders Clinic
Karen Grace / .(JavaScript must be enabled to view this email address) / Durham, North Carolina 27705
United States
Wake Forest School of Medicine
Mozhdeh Marandi / .(JavaScript must be enabled to view this email address) / Winston-Salem, North Carolina 27157
United States
Cleveland Clinic
Cleveland, Ohio 44195
United States
The Ohio State University Wexner Medical Center
Ifeanyi Okoh / .(JavaScript must be enabled to view this email address) / Columbus, Ohio 43210
United States
Providence Brain and Spine Institute ALS Center
Arlena Cummings / .(JavaScript must be enabled to view this email address) / 503-962-1171
Portland, Oregon 97213
United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania 17033
United States
Temple University School of Medicine
Kathleen Hatala / .(JavaScript must be enabled to view this email address) / 215-707-4171
Philadelphia, Pennsylvania 19140
United States
Vanderbilt University Medical Center - Clinical Research Center
Diana Davis / .(JavaScript must be enabled to view this email address) / 615-322-8957
Nashville, Tennessee 37232
United States
Texas Neurology
Todd Morgan / .(JavaScript must be enabled to view this email address) / 214-827-3610 ext 228
Dallas, Texas 75214
United States
UTHSCSA Medical Arts and Research Center
Pamela Kittrell / .(JavaScript must be enabled to view this email address) / 210-450-0524
San Antonio, Texas 78229
United States
Houston Methodist Hospital
Luis F Lay, Jr. / .(JavaScript must be enabled to view this email address) / 713-441-3057
Houston, Texas 77030
United States
University of Vermont Medical Center
Burlington , Vermont 05405
United States
University of Virginia Health System
Mary Wagoner / .(JavaScript must be enabled to view this email address) / Charlottesville, Virginia 22908
United States
VCU Health - Ambulatory Care Center (ACC)
Tamika Walthour / .(JavaScript must be enabled to view this email address) / Richmond, Virginia 23298
United States
University of Washington Medical Center
Laura Sissons-Ross / .(JavaScript must be enabled to view this email address) / Seattle , Washington 98195
United States
West Virginia University, Dept. of Neurology
Morgantown, West Virginia 26506-9180
United States
Froedtert Memorial Lutheran Hospital
Lynn Wheeler / .(JavaScript must be enabled to view this email address) / Milwaukee , Wisconsin 53226
United States

Centre de recherche du Centre Hospitalier de l'Universite de Montreal
Stefanie Houle / .(JavaScript must be enabled to view this email address) / Montréal, Quebec H2X 0A9
Canada
CHU de Quebec-Universite Laval, Hopital de l'Enfant Jesus
Quebec G1J 1Z4
Canada
Edmonton Kaye Clinic
Shelley Wrona / .(JavaScript must be enabled to view this email address) / Edmonton, Alberta T6GT 1Z1
Canada
McMaster University Medical Centre
Daniela Trapsa / .(JavaScript must be enabled to view this email address) / Hamilton, Ontario L8N 3Z5
Canada
Montreal Neurological Institute and Hospital
Montreal, Quebec H3A 2B4
Canada
Sunnybrook Health Science Centre
Toronto, Ontario M4N 3M5
Canada
University of Calgary, Heritage Medical Research Center
Jose Antonio Martinez / .(JavaScript must be enabled to view this email address) / Calgary, Alberta T2N 4Z6
Canada