A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)

NCT05442775 | Terminated Phase 3 Results FDA Drug

• 2 other identifiers: CY 50322021-004727-33
• Study Type: interventional
• Target: 71
• Locations: 9 countries
• Sites: 41

Brief Summary

The purpose of this study is to assess the long-term safety and tolerability of reldesemtiv in patients with ALS who have successfully completed dosing in the Phase 3 clinical trial, CY 5031 (also known as COURAGE-ALS)

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic Lateral Sclerosis; ALS; CK-2127107; Reldesemtiv

Outcome Measures

Primary Outcomes (1)

• Long-term Safety and Tolerability

  Incidence of treatment-emergent adverse events

  Baseline to Week 34 (time the study was terminated prematurely)

Secondary Outcomes (1)

• Long-term Effect of Reldesemtiv on ALSFRS-R Functional Outcomes

  Baseline to Week 32 (last timepoint before study was terminated prematurely)

Study Arms (1)

Reldesemtiv 300 mg twice daily

EXPERIMENTAL

Patients in this arm take 1 reldesemtiv 300 mg oral tablet twice a day for a 600 mg total daily dose (TDD). Participants who had been down titrated during the parent trail take 150 mg by mouth twice a day.

Drug: Reldesemtiv

Interventions

Reldesemtiv DRUG

Oral tablet

Reldesemtiv 300 mg twice daily

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Able to comprehend and willing to sign an ICF and willing to comply with all study procedures and restrictions for the duration specified in the Schedule of Activities. If non-written consent is given, a Legal Designee of the patient must sign the ICF form.
• Completed dosing in CY 5031

You may not qualify if:

• Has taken investigational study drug (other than reldesemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater
• Presence on Day 1 of any medically significant cardiac, pulmonary, gastrointestinal, 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.
• Use of a strong cytochrome P450 (CYP) 3A4 inhibitor within 7 days prior to first dose of reldesemtiv in CY 5032 or a strong CYP3A4 inducer within 14 days prior to first dose of reldesemtiv in CY 5032
• Use of a medication that is an OCT1/OCT2 substrate within 7 days prior to first dose of reldesemtiv in CY 5032
• Currently participating in another trial, managed access program, open label extension, early access program, or through the right to try act is receiving an investigational drug or received an investigational drug or device within 30 days (or 5 half-lives for drugs, whichever is longer) prior to Day 1. Patients also cannot be taking outside of a clinical trial certain investigational drugs (which includes drugs, supplements, and nutraceuticals) that are currently being studied or have been studied for the treatment of ALS.

Sponsors & Collaborators

• Cytokinetics: lead

Study Sites (41)

St. Joseph's Hospital & Medical Center - Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

Location

University of California Irvine - ALS & Neuromuscular Center

Orange, California, 92868, United States

Location

California Pacific Medical Center - Forbes Norris MDA/ALS Research Center

San Francisco, California, 94109, United States

Location

University of Colorado Hospital Anschutz Outpatient Pavilion

Aurora, Colorado, 80045, United States

Location

George Washington Medical Faculty Associates

Washington D.C., District of Columbia, 20037, United States

Location

University of Florida

Jacksonville, Florida, 32209, United States

Location

Mayo Clinic Jacksonville

Jacksonville, Florida, 32224, United States

Location

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

Tampa, Florida, 33612, United States

Location

Indiana University IU Health Neuroscience Center

Indianapolis, Indiana, 43202, United States

Location

The University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins Outpatient Center

Baltimore, Maryland, 21287, United States

Location

Michigan Medicine

Ann Arbor, Michigan, 48109, United States

Location

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

Neurology Associates

Lincoln, Nebraska, 68506, United States

Location

Hospital for Special Surgery

New York, New York, 10021, United States

Location

SUNY Upstate Medical University Institute for Human Performance

Syracuse, New York, 13210, United States

Location

Atrium Health Neuroscience Institute

Charlotte, North Carolina, 28207, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Texas Neurology, P.A.

Dallas, Texas, 75206, United States

Location

Virginia Commonwealth University

Henrico, Virginia, 23233, United States

Location

Froedtert Hospital - Department of Neurology

Milwaukee, Wisconsin, 53226, United States

Location

Concord Repatriation General Hospital

Concord, New South Wales, 2139, Australia

Location

Royal Brisbane and Women's Hospital, Neurology Department

Herston, Queensland, 4029, Australia

Location

The Perron Institute

Nedlands, 6009, Australia

Location

Uz Leuven Gasthuisberg Department of Neurology

Leuven, 3000, Belgium

Location

University of Calgary - Heritage Medical Research Clinic

Calgary, Alberta, T2N 4Z6, Canada

Location

University of Alberta

Edmonton, Alberta, T6G1Z1, Canada

Location

Stan Cassidy Centre for Rehabilitation

Fredericton, New Brunswick, E3B 0C7, Canada

Location

McMaster University

Hamilton, Ontario, L8N4K1, Canada

Location

Ottawa Hospital Research Institute

Ottawa, Ontario, K1Y4E9, Canada

Location

Sunnybrook Health Sciences Centre

Toronto, Ontario, M4N 3M5, Canada

Location

Montreal Neurological Institute and Hospital

Montreal, Quebec, H3A 2B4, Canada

Location

University of Saskatchewan

Saskatoon, Saskatchewan, S7K0M7, Canada

Location

CHU de Quebec-Universite Laval

Québec, G1J1Z4, Canada

Location

RSCI Education and Research Center Beaumount Hospital

Dublin, 9, Ireland

Location

IRCCS Istituto Auxologico Italiano Ospedale San Luca U.O. Neurologia e Stroke Unit

Milan, 20149, Italy

Location

AOU Citta della Salute e della Scienza di Torino Universita degli Studi di Torino P.O. Mollinette - Dipartimento de Neuroscienze "Rita Levi Montalcini"

Torino, 10126, Italy

Location

UMC Utrecht Department of Neurology, ALS Center

Utrecht, 3584, Netherlands

Location

Hospital San Rafael

Madrid, 28016, Spain

Location

Hospital Universitario y Politecnico La Fe

Valencia, 46026, Spain

Location

Studieenheten, Akademiskt Specialistcentrum

Stockholm, 113 61, Sweden

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

reldesemtiv

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Limitations and Caveats

Based on interim analysis results of the Phase 3 parent trial CY 5031 which met the criteria for futility in participants with ALS, CY 5032 was prematurely terminated on 31 March 2023.

Results Point of Contact

• Title: Cytokinetics MD
• Organization: Cytokinetics

medicalaffairs@cytokinetics.com

650-624-2929

Study Officials

• Cytokinetics MD

  Scientific Leadership at Cytokinetics

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 16, 2022
• First Posted: July 5, 2022
• Study Start: August 4, 2022
• Primary Completion: June 7, 2023
• Study Completion: June 7, 2023
• Last Updated: August 13, 2024
• Results First Posted: August 13, 2024

Record last verified: 2024-07

Locations

NL (1); IE (1); AU (3); ES (2); SE (1); US (21); IT (2); BE (1); CA (9)