A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)
- Registration Number
- NCT05442775
- Lead Sponsor
- Cytokinetics
- 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.
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 71
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Reldesemtiv 300 mg twice daily Reldesemtiv 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.
- Primary Outcome Measures
Name Time Method Long-term Safety and Tolerability Baseline to Week 34 (time the study was terminated prematurely) Incidence of treatment-emergent adverse events
- Secondary Outcome Measures
Name Time Method Long-term Effect of Reldesemtiv on ALSFRS-R Functional Outcomes Baseline to Week 32 (last timepoint before study was terminated prematurely) Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total scores. ALSFRS-R total score range is from 0 to 48. A score of 48 reflects normal function.
Trial Locations
- Locations (41)
University of California Irvine - ALS & Neuromuscular Center
🇺🇸Orange, California, United States
Mayo Clinic Jacksonville
🇺🇸Jacksonville, Florida, United States
Michigan Medicine
🇺🇸Ann Arbor, Michigan, United States
Hospital for Special Surgery
🇺🇸New York, New York, United States
Texas Neurology, P.A.
🇺🇸Dallas, Texas, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Atrium Health Neuroscience Institute
🇺🇸Charlotte, North Carolina, United States
SUNY Upstate Medical University Institute for Human Performance
🇺🇸Syracuse, New York, United States
The Perron Institute
🇦🇺Nedlands, Australia
Concord Repatriation General Hospital
🇦🇺Concord, New South Wales, Australia
Royal Brisbane and Women's Hospital, Neurology Department
🇦🇺Herston, Queensland, Australia
Uz Leuven Gasthuisberg Department of Neurology
🇧🇪Leuven, Belgium
Sunnybrook Health Sciences Centre
🇨🇦Toronto, Ontario, Canada
Ottawa Hospital Research Institute
🇨🇦Ottawa, Ontario, Canada
CHU de Quebec-Universite Laval
🇨🇦Quebec, Canada
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
The University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
University of Alberta
🇨🇦Edmonton, Alberta, Canada
George Washington Medical Faculty Associates
🇺🇸Washington, District of Columbia, United States
Montreal Neurological Institute and Hospital
🇨🇦Montréal, Quebec, Canada
RSCI Education and Research Center Beaumount Hospital
🇮🇪Dublin, Ireland
McMaster University
🇨🇦Hamilton, Ontario, Canada
Neurology Associates
🇺🇸Lincoln, Nebraska, United States
University of Calgary - Heritage Medical Research Clinic
🇨🇦Calgary, Alberta, Canada
University of Saskatchewan
🇨🇦Saskatoon, Saskatchewan, Canada
University of Florida
🇺🇸Jacksonville, Florida, United States
Stan Cassidy Centre for Rehabilitation
🇨🇦Fredericton, New Brunswick, Canada
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
St. Joseph's Hospital & Medical Center - Barrow Neurological Institute
🇺🇸Phoenix, Arizona, United States
California Pacific Medical Center - Forbes Norris MDA/ALS Research Center
🇺🇸San Francisco, California, United States
Indiana University IU Health Neuroscience Center
🇺🇸Indianapolis, Indiana, United States
Henry Ford Hospital
🇺🇸Detroit, Michigan, United States
Froedtert Hospital - Department of Neurology
🇺🇸Milwaukee, Wisconsin, United States
University of Colorado Hospital Anschutz Outpatient Pavilion
🇺🇸Aurora, Colorado, United States
University of South Florida - Carol and Frank Morsani Center for Advanced Health Care
🇺🇸Tampa, Florida, United States
Virginia Commonwealth University
🇺🇸Henrico, Virginia, United States
Johns Hopkins Outpatient Center
🇺🇸Baltimore, Maryland, United States