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

NCT ID: NCT05442775

Last Updated: 2024-08-13

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE3
• Total Enrollment: 71 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-08-04
• Study Completion Date: 2023-06-07

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)

Detailed Description

CY 5032 is an open-label extension (OLE) study of the selective fast skeletal muscle troponin activator, reldesemtiv, in patients with ALS who finished dosing (through Week 48) in CY 5031 (COURAGE-ALS). Approximately 400 patients from the sites that participated in CY 5031 are expected to be enrolled in the open-label extension, CY 5032.

Following enrollment, patients will continue dosing with reldesemtiv, 300 mg twice a day for a 600 mg total daily dose (TDD) for a period of 48 weeks.

At the end of 48 weeks, patients may transition to a reldesemtiv Managed Access Program (MAP) if the treating physician agrees to participate in the program.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Reldesemtiv 300 mg twice daily

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.

Group Type: EXPERIMENTAL

Reldesemtiv

Intervention Type: DRUG

Oral tablet

Interventions

Reldesemtiv

Oral tablet

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cytokinetics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Cytokinetics MD

Role: STUDY_CHAIR

Scientific Leadership at Cytokinetics

Locations

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

Phoenix, Arizona, United States

University of California Irvine - ALS & Neuromuscular Center

Orange, California, United States

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

San Francisco, California, United States

University of Colorado Hospital Anschutz Outpatient Pavilion

Aurora, Colorado, United States

George Washington Medical Faculty Associates

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

University of Florida

Jacksonville, Florida, United States

Mayo Clinic Jacksonville

Jacksonville, Florida, United States

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

Tampa, Florida, United States

Indiana University IU Health Neuroscience Center

Indianapolis, Indiana, United States

The University of Kansas Medical Center

Kansas City, Kansas, United States

Johns Hopkins Outpatient Center

Baltimore, Maryland, United States

Michigan Medicine

Ann Arbor, Michigan, United States

Henry Ford Hospital

Detroit, Michigan, United States

Neurology Associates

Lincoln, Nebraska, United States

Hospital for Special Surgery

New York, New York, United States

SUNY Upstate Medical University Institute for Human Performance

Syracuse, New York, United States

Atrium Health Neuroscience Institute

Charlotte, North Carolina, United States

Cleveland Clinic

Cleveland, Ohio, United States

Texas Neurology, P.A.

Dallas, Texas, United States

Virginia Commonwealth University

Henrico, Virginia, United States

Froedtert Hospital - Department of Neurology

Milwaukee, Wisconsin, United States

Concord Repatriation General Hospital

Concord, New South Wales, Australia

Royal Brisbane and Women's Hospital, Neurology Department

Herston, Queensland, Australia

The Perron Institute

Nedlands, , Australia

Uz Leuven Gasthuisberg Department of Neurology

Leuven, , Belgium

University of Calgary - Heritage Medical Research Clinic

Calgary, Alberta, Canada

University of Alberta

Edmonton, Alberta, Canada

Stan Cassidy Centre for Rehabilitation

Fredericton, New Brunswick, Canada

McMaster University

Hamilton, Ontario, Canada

Ottawa Hospital Research Institute

Ottawa, Ontario, Canada

Sunnybrook Health Sciences Centre

Toronto, Ontario, Canada

Montreal Neurological Institute and Hospital

Montreal, Quebec, Canada

University of Saskatchewan

Saskatoon, Saskatchewan, Canada

CHU de Quebec-Universite Laval

Québec, , Canada

RSCI Education and Research Center Beaumount Hospital

Dublin, , Ireland

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

Milan, , Italy

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

Torino, , Italy

UMC Utrecht Department of Neurology, ALS Center

Utrecht, , Netherlands

Hospital San Rafael

Madrid, , Spain

Hospital Universitario y Politecnico La Fe

Valencia, , Spain

Studieenheten, Akademiskt Specialistcentrum

Stockholm, , Sweden

Countries

United States; Australia; Belgium; Canada; Ireland; Italy; Netherlands; Spain; Sweden

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2021-004727-33

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CY 5032

Identifier Type: -

Identifier Source: org_study_id