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:

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:

NCT04944784

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

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

Isabel Cepeda / 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