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
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
Reldesemtiv, Placebo
Placebo:
Yes
Phase:
Phase 2
Study Chair(s)/Principal Investigator(s):
MD Cytokinetics, Cytokinetics
Clinicaltrials.gov ID:
Neals Affiliated?
Yes
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.
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:
Cytokinetics
Participant Duration:
12 weeks
Estimated Enrollment:
458
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?
Yes
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
Australia
Royal Brisbane and Women's Hospital
Herston, Queensland
4029
Australia
Flinders Medical Centre
Bedford Park, South Australia
5042
Australia
The Perron Institute for Neurological and Translation Science
Nedlands, Western Australia
6009
Australia
Department of Neurology, Westmead Hospital
Westmead, New South Wales
2145
Australia
Montreal Neurological Institute and Hospital
Montreal, Quebec
H3A 2B4
Canada
CHU de Quebec-Universite Laval, Hopital de l'Enfant Jesus
Quebec G1J 1Z4
Canada
Saskatoon City Hospital
Saskatoon, Saskatchewan
S7H 0G9
Canada
Centre de recherche du Centre Hospitalier de l'Universite de Montreal
Montréal, Quebec
H2X 0A9
Canada
London Health Sciences Centre University Hospital
London, Ontario
N6A 5A5
Canada
Sunnybrook Health Science Centre
Toronto, Ontario
M4N 3M5
Canada
McMaster University Medical Centre
Hamilton, Ontario
L8N 3Z5
Canada
Edmonton Kaye Clinic
Edmonton, Alberta
T6GT 1Z1
Canada
University of Calgary, Heritage Medical Research Center
Calgary, Alberta
T2N 4Z6
Canada
Beaumont Hospital
Dublin Dublin 9
Ireland
University Medical Center Utrecht
Utrecht 3584 CX
Netherlands
Hospital San Rafael Servicio de Neurologia
Madrid 28016
Spain