Study Purpose:
The purpose of this study is to assess the effect of reldesemtiv versus placebo on functional outcomes in ALS.Study Status:
Not recruiting
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
Reldesemtiv, Placebo
Placebo:
Yes
Phase:
Phase 3
Study Chair(s)/Principal Investigator(s):
Cytokinetics, MD, Cytokinetics
Clinicaltrials.gov ID:
Neals Affiliated?
Yes
Coordinating Center Contact Information
United States
Full Study Summary:
COURAGE-ALS is a Phase 3, double-blind, randomized, placebo-controlled trial of reldesemtiv in patients aged 18 to 80 with ALS.
The screening and qualification period for the trial will be no more than 14 days in duration. Approximately 555 eligible ALS patients will be randomized (2:1) to receive the following dose of reldesemtiv or placebo (stratified by riluzole use/non-use and edaravone use/non-use) for the first 24 weeks (double-blind, placebo-controlled period):
- 300 mg reldesemtiv twice a day for a 600 mg total daily dose (TDD)
- Placebo twice daily
At the end of the 24-week double-blind, placebo-controlled period, patients will transition to the active drug period, where all patients will receive the following dose of reldesemtiv for the next 24 weeks:
- 300 mg reldesemtiv twice a day for a 600 mg TDD for patients who were not down titrated during the 24 weeks of blinded dosing
- 150 mg reldesemtiv twice a day for a 300 mg TDD for patients who were down titrated during the 24 weeks of blinded dosing
The screening and qualification period for the trial will be no more than 14 days in duration. Approximately 555 eligible ALS patients will be randomized (2:1) to receive the following dose of reldesemtiv or placebo (stratified by riluzole use/non-use and edaravone use/non-use) for the first 24 weeks (double-blind, placebo-controlled period):
- 300 mg reldesemtiv twice a day for a 600 mg total daily dose (TDD)
- Placebo twice daily
At the end of the 24-week double-blind, placebo-controlled period, patients will transition to the active drug period, where all patients will receive the following dose of reldesemtiv for the next 24 weeks:
- 300 mg reldesemtiv twice a day for a 600 mg TDD for patients who were not down titrated during the 24 weeks of blinded dosing
- 150 mg reldesemtiv twice a day for a 300 mg TDD for patients who were down titrated during the 24 weeks of blinded dosing
Study Sponsor:
Cytokinetics
Estimated Enrollment:
486
Estimated Study Start Date:
08 / 16 / 2021
Estimated Study Completion Date:
03 / 31 / 2023
Posting Last Modified Date:
04 / 14 / 2023
Date Study Added to neals.org:
06 / 30 / 2021
Minimum Age:
18 Years
Maximum Age:
80 Years
Can participants use Riluzole?
Yes
Key Inclusion Criteria:- Males or Females between the ages of 18 and 80 years of age, inclusive
- Diagnosis of familial or sporadic ALS (defined as meeting the laboratory-supported probable, probable, or definite criteria for ALS according to the World Federation of Neurology El Escorial criteria). Patients who meet the possible criteria are eligible if they have lower motor neuron findings; those who have purely upper motor neuron findings are ineligible.
- First symptom of ALS ≤ 24 months prior to screening. The qualifying first symptoms of ALS are limited to manifestations of weakness in extremity, bulbar, or respiratory muscles.
- ALSFRS-R total score ≤ 44 at screening. Patients with a total score of 45 or higher may be rescreened 60±7 days following the original screening date.
- Upright FVC ≥ 65.0% of predicted for age, height, sex and ethnicity at screening according to Global Lung Initiative equation
- Must be either on riluzole for ≥ 30 days prior to screening or have not taken it for at least 30 days prior to screening
- Must have completed at least 2 cycles of edaravone at the time of screening or have not received it for at least 30 days prior to screening
- Able to swallow whole tablets
Exclusion Criteria:
- eGFRCysC < 45.0 mL/min/1.73 m2 at screening
- Urine protein/creatinine ratio > 1 mg/mg (113 mg/mmol) at screening
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 3-times the upper limit of normal (ULN)
- Total bilirubin (TBL), direct or indirect bilirubin above the ULN.
- Cognitive impairment, related to ALS or otherwise that impairs the patient's ability to understand and/or comply with study procedures and provide informed consent
- Other medically significant neurological conditions that could interfere with the assessment of ALS symptoms, signs or progression.
- Has a tracheostomy
St. Joseph's Hospital & Medical Center - Barrow Neurological Institute
Phoenix, Arizona
85013
United States
Cedars-Sinai Medical Center
Los Angeles, California
90048
United States
University of California Irvine - ALS & Neuromuscular Center
Orange, California
92868
United States
California Pacific Medical Center - Forbes Norris MDA/ALS Research Center
San Francisco, California
94109
United States
Stanford Hospital and Clinics
Stanford, California
94305
United States
University of Colorado Hospital Anschutz Outpatient Pavilion
Aurora, Colorado
80045
United States
GW Medical Faculty Associates
Washington, District of Columbia
20037
United States
University of Florida Jacksonville
Jacksonville, Florida
32209
United States
Mayo Clinic Florida
Jacksonville, Florida
32224
United States
University of South Florida - Carol and Frank Morsani Center for Advanced Health Care
Tampa, Florida
33612
United States
Duchossois Center for Advanced Medicine
Chicago, Illinois
60637
United States
Indiana University
Indianapolis, Indiana
46202
United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa
52242
United States
The University of Kansas Medical Center
Kansas City, Kansas
66160
United States
Johns Hopkins Outpatient Center
Baltimore, Maryland
21287
United States
Massachusetts General Hospital - Neurological Clinical Research Institute
Boston, Massachusetts
02114
United States
University of Massachusetts Memorial Medical Center/Medical School
Worcester, Massachusetts
01655
United States
Michigan Medicine
Ann Arbor, Michigan
48109
United States
Henry Ford Health System
Detroit, Michigan
48202
United States
Washington University School of Medicine - Center for Advance Medicine
Saint Louis, Missouri
63108
United States
Neurology Associates, PC
Lincoln, Nebraska
68506
United States
Hospital for Special Surgery
New York, New York
10021
United States
Columbia University Medical Center
New York, New York
10032
United States
University of Rochester Medical Center
Rochester, New York
14642
United States
SUNY Upstate Medical University
Syracuse, New York
13210
United States
Atrium Health Neuroscience Institute - Charlotte
Charlotte, North Carolina
28207
United States
Cleveland Clinic
Cleveland, Ohio
44195
United States
Providence ALS Center
Portland, Oregon
97213
United States
Oregon Health and Science University
Portland, Oregon
97239
United States
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania
17033
United States
Lewis Katz School of Medicine at Temple University
Philadelphia, Pennsylvania
19140
United States
Vanderbilt University Medical Center - Clinical Research Center
Nashville, Tennessee
37232
United States
Texas Neurology, P.A.
Dallas, Texas
75206
United States
University of Vermont Medical Center
Burlington, Vermont
05401
United States
VCU Neuroscience Orthopaedic and Wellness Center (NOW)
Henrico, Virginia
23233
United States
Froedtert Hospital
Milwaukee, Wisconsin
53226
United States
Brain and Mind Centre
Camperdown, New South Wales
2050
Australia
Concord Repatriation General Hospital
Concord, New South Wales
2139
Australia
Royal Brisbane and Women's Hospital
Herston, Queensland
4029
Australia
Flinders Medical Centre
Bedford Park, South Australia
5042
Australia
The Perron Institute
Nedlands, Western Australia
6009
Australia
UZ Leuven Gasthuisberg, Department of Neurology
Leuven 3000
Belgium
University of Calgary - Heritage Medical Research Clinic
Calgary, Alberta
T2N 4Z6
Canada
University of Alberta
Edmonton, Alberta
T6G 2B7
Canada
Stan Cassidy Centre for Rehabilitation
Fredericton, New Brunswick
E3B 0C7
Canada
McMaster University Medical Centre
Hamilton, Ontario
L8N 3Z5
Canada
London Health Sciences Centre
London, Ontario
N6A 5A5
Canada
Ottawa Hospital Research Institute - Civic Campus
Ottawa, Ontario
K1Y 4E9
Canada
Sunnybrook Research Institute
Toronto, Ontario
M4N 3M5
Canada
Centre de recherche du CHUM
Montréal, Quebec
H2X 0A9
Canada
McGill University, Montreal Neurological Institute & Hospital
Montréal, Quebec
H3A 2B4
Canada
CHU de Quebec-Université Laval
Québec, Quebec
G1J 1Z4
Canada
Saskatoon City Hospital
Saskatoon, Saskatchewan
S7K 0M7
Canada
Deparment of Neurology Bispebjerg University Hospital
Copenhagen 2400
Denmark
CRC SLA de Lyon
Bron 69677
France
CHRU de Lille Hopital Roger Salengro
Lille 59037
France
CHU de Limoges - Hopital Dupuytren
Limoges 87 042
France
CHU de la Timone
Marseille 13005
France
CHU de Nice - Hôpital Pasteur 2
Nice 06 001
France
Hopital La Pitie Salpetriere
Paris 75013
France
CHRU de Tours, Hopital Bretonneau, Clinical Research Center
Tours 37044
France
Charité - Universitätsmedizin Berlin
Berlin 13353
Germany
Universitatsklinikum Bonn
Bonn 53127
Germany
Medical School Hannover - Department of Neurology
Hanover 30625
Germany
Universitatsklinikum Jena
Jena 07747
Germany
Universitätsklinikum Schleswig Holstein
Lübeck 23538
Germany
Universitatsklinikum Ulm
Ulm 89081
Germany
RSCI Education and Research Centre, Beaumont Hospital
Beaumont, Dublin
9
Ireland
Instituti Clinici Scientifici Maugeri
Milano 20138
Italy
Ospedale San Luca
Milan 20149
Italy
Centro Clinical Nemo - Fondazione Serena Onlus
Milan 20162
Italy
AOU Città della Salute e Scienza (Molinette),
Turin 10126
Italy
UMC Utrecht, Department of Neurology, ALS Center
Utrecht 3584 CX
Netherlands
City Clinic Research
Warsaw 02-473
Poland
Centro Hospitalar Universitario Lisboa Norte, Department of Neurology
Lisboa 1649-035
Portugal
Hospital Universitari de Bellvitge
Barcelona 08907
Spain
Hospital Universitario Basurto
Bilbao 48013
Spain
Hospital San Rafael
Madrid 28016
Spain
Hospital Universitari i Politecnic La Fe
Valencia 46026
Spain
Neurologimottagningen Skane University Hospital
Malmö 21428
Sweden
Studieenheten Akademiskt Specialistcentrum, Sabbatsberg Hospital
Stockholm 11361
Sweden
Muskelzentrum/ALS Clinic
Saint Gallen 9007
Switzerland
The Walton Centre NHS Foundation Trust
Liverpool L9 7LJ
United Kingdom
Maurice Wohl Clinical Neuroscience Institute
London SE5 9RX
United Kingdom