Study Purpose:
The purpose of this study is to assess the effect of reldesemtiv versus placebo on functional outcomes in ALS.Study Status:
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
Cytokinetics, MD / email hidden; JavaScript is required / 650-624-2929
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:
555
Estimated Study Start Date:
08 / 16 / 2021
Estimated Study Completion Date:
03 / 01 / 2024
Posting Last Modified Date:
10 / 25 / 2022
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 | Recruiting
Annalee Boyle, MD / 602-406-7773 / email hidden; JavaScript is required
Phoenix, Arizona
85013
United States
Cedars-Sinai Medical Center | Recruiting
Richard Lewis, MD
Sophia Mostowy / 310-423-0827 / email hidden; JavaScript is required
Los Angeles, California
90048
United States
University of California Irvine - ALS & Neuromuscular Center | Recruiting
Namita Goyal, MD
Cindy Vo / 714-456-6192 / email hidden; JavaScript is required
Orange, California
92868
United States
California Pacific Medical Center - Forbes Norris MDA/ALS Research Center | Recruiting
Jonathan Katz, MD
Henry Chen / email hidden; JavaScript is required
San Francisco, California
94109
United States
Stanford Hospital and Clinics | Recruiting
Yuen T. So, MD, PhD / 650-725-4341 / email hidden; JavaScript is required
Stanford, California
94305
United States
University of Colorado Hospital Anschutz Outpatient Pavilion | Recruiting
/ 303-724-4644 / email hidden; JavaScript is required
Aurora, Colorado
80045
United States
GW Medical Faculty Associates | Recruiting
Elham Bayat, MD
Anosha Khan / 202-741-2745 / email hidden; JavaScript is required
Washington, District of Columbia
20037
United States
University of South Florida - Carol and Frank Morsani Center for Advanced Health Care | Recruiting
Tuan H. Vu, MD
Jessica Shaw / (813) 974-9413 / email hidden; JavaScript is required
Tampa, Florida
33612
United States
Mayo Clinic Florida | Recruiting
Bjorn E. Oskarsson, MD
Colette McHugh-Strong / (904) 953-4965 / email hidden; JavaScript is required
Jacksonville, Florida
32224
United States
University of Florida Jacksonville | Recruiting
Michael Pulley, MD, PhD
Deepa Nagaraju / (904) 244-9480 / email hidden; JavaScript is required
Jacksonville, Florida
32209
United States
Duchossois Center for Advanced Medicine | Recruiting
Kourosh Rezania
Javaria Anwer / (908)392-8303 / email hidden; JavaScript is required
Chicago, Illinois
60637
United States
Indiana University | Recruiting
Cynthia Bodkin, MD
Indianapolis, Indiana
46202
United States
University of Iowa Hospitals and Clinics | Recruiting
Andrea Swenson
Heena Olalde / 319-356-8326 / email hidden; JavaScript is required
Iowa City, Iowa
52242
United States
The University of Kansas Medical Center | Recruiting
Jeffrey M. Statland, MD
Katie Lillig / 913-945-9932 / email hidden; JavaScript is required
Kansas City, Kansas
66160
United States
Johns Hopkins Outpatient Center | Recruiting
Jeffrey D. Rothstein, MD, PhD
Kristen Riley / email hidden; JavaScript is required
Baltimore, Maryland
21287
United States
Massachusetts General Hospital - Neurological Clinical Research Institute | Recruiting
Max Higgins / 617-643-2522 / email hidden; JavaScript is required
Sabrina Paganoni, MD, PhD
Boston, Massachusetts
02114
United States
University of Massachusetts Memorial Medical Center/Medical School | Recruiting
Margaret A. Owegi, DO
Catherine Douthwright / 774-441-7696 / email hidden; JavaScript is required
Worcester, Massachusetts
01655
United States
Henry Ford Health System | Recruiting
Ximena Arcila-Londono, MD
Kathryn Swiftney / 313-916-3501 / email hidden; JavaScript is required
Detroit, Michigan
48202
United States
Michigan Medicine | Recruiting
Stephen A. Goutman, MD, MS
Jayna Duell / 734-936-8776 / email hidden; JavaScript is required
Ann Arbor, Michigan
48109
United States
Washington University School of Medicine - Center for Advance Medicine | Recruiting
TImothy M. Miller, MD, PhD
/ 1-844-ALS-CARE / email hidden; JavaScript is required
Saint Louis, Missouri
63108
United States
Neurology Associates, PC | Recruiting
Gary L. Pattee, MD
Lincoln, Nebraska
68506
United States
Hospital for Special Surgery | Recruiting
Dale J. Lange
Shara Holzberg / 646-797-8592 / email hidden; JavaScript is required
New York, New York
10021
United States
Columbia University Medical Center | Recruiting
Jinsy Andrews, MD, MSc
New York, New York
10032
United States
University of Rochester Medical Center | Recruiting
Rochester, New York
14642
United States
SUNY Upstate Medical University | Recruiting
Deborah Bradshaw, MD
Sigiriya Smolen / 315-464-9767 / email hidden; JavaScript is required
Syracuse, New York
13210
United States
Atrium Health Neuroscience Institute - Charlotte | Recruiting
Leo McCluskey
Cynthia Lary / (704) 446-6063 / email hidden; JavaScript is required
Charlotte, North Carolina
28207
United States
Cleveland Clinic | Recruiting
Rebecca Kuenzler, MD
Cleveland, Ohio
44195
United States
Providence ALS Center | Recruiting
Nicholas T. Olney, MD
Ashley Adamo / (503) 962-1171 / email hidden; JavaScript is required
Portland, Oregon
97213
United States
Oregon Health and Science University | Not yet recruiting
Yvel Maspinas / email hidden; JavaScript is required
Portland, Oregon
97239
United States
Penn State Health Milton S. Hershey Medical Center | Recruiting
James Grogan, MD
Dodi Schaak / email hidden; JavaScript is required
Hershey, Pennsylvania
17033
United States
Lewis Katz School of Medicine at Temple University | Recruiting
Terry Heiman-Patterson, MD
Kathleen Hatala / (215) 707-4171 / email hidden; JavaScript is required
Philadelphia, Pennsylvania
19140
United States
Vanderbilt University Medical Center - Clinical Research Center | Recruiting
Amanda C. Peltier, MD
Nashville, Tennessee
37232
United States
Texas Neurology, P.A. | Recruiting
Daragh Heitzman, MD
Mohamad A. Nasri, MD / (214) 827-3610 Ext. 251 / email hidden; JavaScript is required
Dallas, Texas
75206
United States
University of Vermont Medical Center | Recruiting
Rup Tandan, MD
Avery St. Sauveur / email hidden; JavaScript is required
Burlington, Vermont
05401
United States
VCU Neuroscience Orthopaedic and Wellness Center (NOW) | Recruiting
Kelly Gwathmey, MD
Henrico, Virginia
23233
United States
Froedtert Hospital | Recruiting
Dominic Fee, MD
Taylor Aderman / 414-955-0667 / email hidden; JavaScript is required
Milwaukee, Wisconsin
53226
United States
Brain and Mind Centre | Recruiting
Matthew Kiernan
/ +61 2 9351 0976 / email hidden; JavaScript is required
Camperdown, New South Wales
2050
Australia
Royal Brisbane and Women's Hospital | Recruiting
Susan Heggie
/ +61 7 36468111 / email hidden; JavaScript is required
Herston, Queensland
4029
Australia
Flinders Medical Centre | Recruiting
Eldira Dishnica / +61 (08) 8204 5168
Bedford Park, South Australia
5042
Australia
The Perron Institute | Recruiting
Merrilee Needham
/ +61 (08) 6457 0312 / email hidden; JavaScript is required
Nedlands, Western Australia
6009
Australia
Concord Repatriation General Hospital | Recruiting
Steve Vucic
/ +61 (02) 9767 8461 / email hidden; JavaScript is required
Concord, New South Wales
2139
Australia
UZ Leuven Gasthuisberg, Department of Neurology | Recruiting
Philip Van Damme
Leuven 3000
Belgium
Sunnybrook Research Institute | Recruiting
Lorne Zinman
Anita Seghatoleslam / (416) 480-6100 Ext. 87561 / email hidden; JavaScript is required
Toronto, Ontario
M4N 3M5
Canada
Saskatoon City Hospital | Recruiting
Kerri Schellenberg
Saskatoon, Saskatchewan
S7K 0M7
Canada
CHU de Quebec-Université Laval | Recruiting
Annie Dionne
Alexandra Simard / email hidden; JavaScript is required
Québec, Quebec
G1J 1Z4
Canada
McGill University, Montreal Neurological Institute & Hospital | Recruiting
Angela Genge / email hidden; JavaScript is required
Montréal, Quebec
H3A 2B4
Canada
Centre de recherche du CHUM | Recruiting
Genevieve Matte
Montréal, Quebec
H2X 0A9
Canada
McMaster University Medical Centre | Recruiting
John Turnbull
Daniela Trapsa / 905-521-2100 Ext. 76368 / email hidden; JavaScript is required
Hamilton, Ontario
L8N 3Z5
Canada
Ottawa Hospital Research Institute - Civic Campus | Recruiting
Ariel Breiner
Ottawa, Ontario
K1Y 4E9
Canada
London Health Sciences Centre | Recruiting
Christen Shoesmith / email hidden; JavaScript is required
London, Ontario
N6A 5A5
Canada
Stan Cassidy Centre for Rehabilitation | Recruiting
Colleen O'Connell
Shane McCullum / email hidden; JavaScript is required
Fredericton, New Brunswick
E3B 0C7
Canada
University of Alberta | Recruiting
Wendy Johnston
Kelsey Tymkow / 780-492-7690 / email hidden; JavaScript is required
Edmonton, Alberta
T6G 2B7
Canada
University of Calgary - Heritage Medical Research Clinic | Recruiting
Li Pi Shan Rodney
Berchman Wong / 403-210-7009 / email hidden; JavaScript is required
Calgary, Alberta
T2N 4Z6
Canada
CHU de Nice - Hôpital Pasteur 2 | Recruiting
Marie-Helene Soriani
Nice 06 001
France
Hopital La Pitie Salpetriere | Recruiting
Francois Salachas
Paris 75013
France
CHRU de Tours, Hopital Bretonneau, Clinical Research Center | Recruiting
Philippe Corcia, Pr.
Tours 37044
France
CHU de la Timone | Recruiting
Shahram Attarian
Marseille 13005
France
CHU de Limoges - Hopital Dupuytren | Recruiting
Couratier Philippe
Limoges 87 042
France
CHRU de Lille Hopital Roger Salengro | Recruiting
Lille 59037
France
CRC SLA de Lyon | Recruiting
Bron 69677
France
Charité - Universitätsmedizin Berlin | Recruiting
Berlin 13353
Germany
Universitatsklinikum Bonn | Recruiting
Patrick Weydt
Bonn 53127
Germany
Medical School Hannover - Department of Neurology | Recruiting
Susanne Petri
Hanover 30625
Germany
Universitatsklinikum Jena | Recruiting
Annekathrin Rodiger
Jena 07747
Germany
Universitätsklinikum Schleswig Holstein | Recruiting
Grosskreutz Julian
Lübeck 23538
Germany
Universitatsklinikum Ulm | Recruiting
Albert C. Ludolph, Prof. Dr.
Ulm 89081
Germany
RSCI Education and Research Centre, Beaumont Hospital | Recruiting
Orla Hardiman
Beaumont, Dublin
9
Ireland
AOU Città della Salute e Scienza (Molinette), | Recruiting
Andrea Calvo
Turin 10126
Italy
Centro Clinical Nemo - Fondazione Serena Onlus | Recruiting
Christian Lunetta
Milan 20162
Italy
Ospedale San Luca | Recruiting
Vincenzo Silani / email hidden; JavaScript is required
Milan 20149
Italy
Instituti Clinici Scientifici Maugeri | Recruiting
Milano 20138
Italy
UMC Utrecht, Department of Neurology, ALS Center | Recruiting
L.H. van den Berg, Dr.
Utrecht 3584 CX
Netherlands
City Clinic Research | Recruiting
Magdalena Kuzma-Kozakiewicz
Warsaw 02-473
Poland
Centro Hospitalar Universitario Lisboa Norte, Department of Neurology | Recruiting
Mamede Carvalho
Lisboa 1649-035
Portugal
Hospital Universitari de Bellvitge | Recruiting
Monica Povedano
Barcelona 08907
Spain
Hospital Universitario Basurto | Recruiting
Luis Varona Franco
Bilbao 48013
Spain
Hospital San Rafael | Recruiting
Jesus Mora
Madrid 28016
Spain
Hospital Universitari i Politecnic La Fe | Recruiting
Juan Francisco Vazquez Costa
Valencia 46026
Spain
Studieenheten Akademiskt Specialistcentrum, Sabbatsberg Hospital | Recruiting
Caroline Ingre
Stockholm 11361
Sweden
Muskelzentrum/ALS Clinic | Recruiting
Markus Weber, MD
Saint Gallen 9007
Switzerland
The Walton Centre NHS Foundation Trust | Recruiting
Carolyn Young
Liverpool L9 7LJ
United Kingdom
Maurice Wohl Clinical Neuroscience Institute | Recruiting
Ammar Al-Chalabi
London SE5 9RX
United Kingdom