A Phase 3, Multi-Center, Double-Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)

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


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

Reldesemtiv, Placebo




Phase 3

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

Cytokinetics, MD, Cytokinetics

Clinicaltrials.gov ID:


Neals Affiliated?


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

Study Sponsor:


Estimated Enrollment:


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?


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 South Florida - Carol and Frank Morsani Center for Advanced Health Care

Tampa, Florida 33612
United States

Mayo Clinic Florida

Jacksonville, Florida 32224
United States

University of Florida Jacksonville

Jacksonville, Florida 32209
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

Henry Ford Health System

Detroit, Michigan 48202
United States

Michigan Medicine

Ann Arbor, Michigan 48109
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

Royal Brisbane and Women's Hospital

Herston, Queensland 4029

Flinders Medical Centre

Bedford Park, South Australia 5042

The Perron Institute

Nedlands, Western Australia 6009

Concord Repatriation General Hospital

Concord, New South Wales 2139

UZ Leuven Gasthuisberg, Department of Neurology

Leuven 3000

Sunnybrook Research Institute

Toronto, Ontario M4N 3M5

Saskatoon City Hospital

Saskatoon, Saskatchewan S7K 0M7

CHU de Quebec-Université Laval

Québec, Quebec G1J 1Z4

McGill University, Montreal Neurological Institute & Hospital

Montréal, Quebec H3A 2B4

Centre de recherche du CHUM

Montréal, Quebec H2X 0A9

London Health Sciences Centre

London, Ontario N6A 5A5

Ottawa Hospital Research Institute - Civic Campus

Ottawa, Ontario K1Y 4E9

McMaster University Medical Centre

Hamilton, Ontario L8N 3Z5

Stan Cassidy Centre for Rehabilitation

Fredericton, New Brunswick E3B 0C7

University of Alberta

Edmonton, Alberta T6G 2B7

University of Calgary - Heritage Medical Research Clinic

Calgary, Alberta T2N 4Z6

Deparment of Neurology Bispebjerg University Hospital

Copenhagen 2400

CHU de Nice - Hôpital Pasteur 2

Nice 06 001

Hopital La Pitie Salpetriere

Paris 75013

CHRU de Tours, Hopital Bretonneau, Clinical Research Center

Tours 37044

CHU de la Timone

Marseille 13005

CHU de Limoges - Hopital Dupuytren

Limoges 87 042

CHRU de Lille Hopital Roger Salengro

Lille 59037

CRC SLA de Lyon

Bron 69677

Charité - Universitätsmedizin Berlin

Berlin 13353

Universitatsklinikum Bonn

Bonn 53127

Medical School Hannover - Department of Neurology

Hanover 30625

Universitatsklinikum Jena

Jena 07747

Universitätsklinikum Schleswig Holstein

Lübeck 23538

Universitatsklinikum Ulm

Ulm 89081

RSCI Education and Research Centre, Beaumont Hospital

Beaumont, Dublin 9

AOU Città della Salute e Scienza (Molinette),

Turin 10126

Centro Clinical Nemo - Fondazione Serena Onlus

Milan 20162

Ospedale San Luca

Milan 20149

Instituti Clinici Scientifici Maugeri

Milano 20138

UMC Utrecht, Department of Neurology, ALS Center

Utrecht 3584 CX

City Clinic Research

Warsaw 02-473

Centro Hospitalar Universitario Lisboa Norte, Department of Neurology

Lisboa 1649-035

Hospital Universitari de Bellvitge

Barcelona 08907

Hospital Universitario Basurto

Bilbao 48013

Hospital San Rafael

Madrid 28016

Hospital Universitari i Politecnic La Fe

Valencia 46026

Neurologimottagningen Skane University Hospital

Malmö 21428

Studieenheten Akademiskt Specialistcentrum, Sabbatsberg Hospital

Stockholm 11361

Muskelzentrum/ALS Clinic

Saint Gallen 9007

The Walton Centre NHS Foundation Trust

Liverpool L9 7LJ
United Kingdom

Maurice Wohl Clinical Neuroscience Institute

London SE5 9RX
United Kingdom