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Quick Info
Status
This study has been completed
Estimated Enrollment
458
Phase
2
Treatment Type
Drug: CK-2127107
Trial Type
Randomized. Parallel Assignment
Sponsor
Primary Investigator
Study Director MD Cytokinetics
Contact Information
Locations
Australia, Other
Flinders Medical Centre, Bedford Park, 5042, Australia
Brain and Mind Centre, The University of Sydney, Camperdown, 2050, Australia
Royal Brisbane and Women's Hospital, Herston, 4029, Australia
The Perron Institute for Neurological and Translation Science, Nedlands, 6009, Australia
Department of Neurology, Westmead Hospital, Westmead, 2145, Australia
Canada, Alberta
University of Calgary, Heritage Medical Research Center, Calgary, AB, T2N 4Z6, Canada
Edmonton Kaye Clinic, Edmonton, AB, T6GT 1Z1, Canada
Canada, Ontario
McMaster University Medical Centre, Hamilton, ON, L8N 3Z5, Canada
London Health Sciences Centre University Hospital, London, ON, N6A 5A5, Canada
Sunnybrook Health Science Centre, Toronto, ON, M4N 3M5, Canada
Canada, Other
CHU de Quebec-Universite Laval, Hopital de l'Enfant Jesus, Quebec, G1J 1Z4, Canada
Canada, Quebec
Montreal Neurological Institute and Hospital, Montreal, QC, H3A 2B4, Canada
Centre de recherche du Centre Hospitalier de l'Universite de Montreal, Montréal, QC, H2X 0A9, Canada
Canada, Saskatchewan
Saskatoon City Hospital, Saskatoon, SK, S7H 0G9, Canada
Ireland, Other
Beaumont Hospital, Dublin, Dublin 9, Ireland
Netherlands, Other
University Medical Center Utrecht, Utrecht, 3584 CX, Netherlands
Spain, Other
Hospital San Rafael Servicio de Neurologia, Madrid, 28016, Spain
United States, Arizona
St. Joseph's Hospital and Medical Center - Barrow Neurological Clinics, Phoenix, AZ, 85013, United States
United States, California
Cedars-Sinai Medical Center, Los Angeles, CA, 90048, United States
University of California Irvine, Orange, CA, 92868, United States
Forbes Norris MDA/ALS Research Center, San Francisco, CA, 94115, United States
Stanford Hospital and Clinics, Stanford, CA, 94305, United States
United States, Colorado
University of Colorado Hospital Anschutz Outpatient Pavilion, Aurora, CO, 80045, United States
United States, Connecticut
Hospital for Special Care, New Britain, CT, 06053, United States
United States, District of Columbia
George Washington University Medical Faculty Associates, Washington, DC, 20037, United States
United States, Florida
University of Florida, Gainesville, FL, 32610, United States
Mayo Clinic, Jacksonville, FL, 32224, United States
Carol & Frank Morsani Center for Advanced Healthcare - University of South Florida, Tampa, FL, 33612, United States
United States, Georgia
Emory Clinic, Atlanta, GA, 30322, United States
United States, Illinois
Duchossois Center for Advanced Medicine, Chicago, IL, 60637, United States
United States, Indiana
IU Health Neuroscience Center of Excellence, Indianapolis, IN, 46202, United States
United States, Iowa
University of Iowa Hospitals and Clinics, Iowa City, IA, 52242, United States
United States, Kansas
University of Kansas Medical Center, Kansas City, KS, 66160, United States
United States, Maryland
Johns Hopkins University - Outpatient Center, Baltimore, MD, 21287, United States
United States, Massachusetts
University of Massachusetts Memorial Medical Center/University of Massachusetts Medical School, Worcester, MA, 01655, United States
United States, Michigan
Michigan Medicine, Ann Arbor, MI, 48109, United States
Henry Ford Health System, Detroit, MI, 48202, United States
United States, Minnesota
Hennepin County Medical Center, Minneapolis, MN, 55415, United States
Mayo Clinic, Rochester, MN, 55905, United States
United States, Missouri
Saint Louis University, Department of Neurology, Saint Louis, MO, 63104, United States
Washington University School of Medicine, Saint Louis, MO, 63110, United States
United States, Nebraska
Neurology Associates, P.C., Lincoln, NE, 68506, United States
United States, New York
Hospital For Special Surgery, New York, NY, 10021, United States
Neurological Institute, Columbia University Medical Center, New York, NY, 10032, United States
SUNY Upstate Medical University, Syracuse, NY, 13210, United States
United States, North Carolina
Neurosciences Institute, Neurology - Charlotte, Charlotte, NC, 28207, United States
Duke Neurological Disorders Clinic, Durham, NC, 27705, United States
Wake Forest School of Medicine, Winston-Salem, NC, 27157, United States
United States, Ohio
Cleveland Clinic, Cleveland, OH, 44195, United States
The Ohio State University Wexner Medical Center, Columbus, OH, 43210, United States
United States, Oregon
Providence Brain and Spine Institute ALS Center, Portland, OR, 97213, United States
Oregon Health & Science University, Portland, OR, 97239, United States
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center, Hershey, PA, 17033, United States
Temple University School of Medicine, Philadelphia, PA, 19140, United States
United States, Tennessee
Vanderbilt University Medical Center - Clinical Research Center, Nashville, TN, 37232, United States
United States, Texas
Texas Neurology, Dallas, TX, 75214, United States
Houston Methodist Hospital, Houston, TX, 77030, United States
UTHSCSA Medical Arts and Research Center, San Antonio, TX, 78229, United States
United States, Vermont
University of Vermont Medical Center, Burlington, VT, 05405, United States
United States, Virginia
University of Virginia Health System, Charlottesville, VA, 22908, United States
VCU Health - Ambulatory Care Center (ACC), Richmond, VA, 23298, United States
United States, Washington
University of Washington Medical Center, Seattle, WA, 98195, United States
United States, West Virginia
West Virginia University, Dept. of Neurology, Morgantown, WV, 26506-9180, United States
United States, Wisconsin
Froedtert Memorial Lutheran Hospital, Milwaukee, WI, 53226, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
SVC ≥ 65%
Months Since Onset
Number of months since first symptoms of ALS.
≤ 24 months from diagnosis (not onset)
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
No
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
No
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Yes
Update Notes
Quality control complete, results posted
9/14/2020
No significant updates
2/18/2020
Trial complete
4/8/2019
No significant updates
3/11/2019
Recruitment status updated
2/15/2019
Location updated
10/24/2018
Location updated
9/25/2018
Trial details updated
9/10/2018
Location updated
4/2/2018
Trial name updated
4/2/2018
Location updated
3/23/2018
Location updated
3/15/2018
Location updated
3/6/2018
No significant updates
3/1/2018
Location updated
2/20/2018
No significant updates
2/9/2018
Location updated
2/6/2018
Location updated
1/31/2018
Location updated
1/24/2018
Locations updated
1/10/2018
Locations updated
1/3/2018
Locations updated
12/20/2017
Locations updated
12/4/2017
Locations updated
11/27/2017
Locations updated
11/7/2017
Location updated
10/27/2017
10/23/2017
Location updated
10/5/2017
Location updated
9/28/2017
Location updated
9/15/2017
No significant updates
9/6/2017
Recruitment status and location updated
8/1/2017
New trial added
5/22/2017

Other Information

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.
Eligibility
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
Details
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.
Trial Protocol as Published on Clinicaltrials.gov
NCT03160898 (First Published: 5/12/2017)