Clinical trial NCT05442775: A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)
Official title: A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Keywords: Amyotrophic Lateral Sclerosis
ALS
CK-2127107
Reldesemtiv
Lead sponsor: Cytokinetics
Sponsor class: INDUSTRY
First posted: 2022-07-05
Enrollment: 71
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Long-term Safety and Tolerability
timeFrame: Baseline to Week 34 (time the study was terminated prematurely)
Secondary outcomes:
measure: Long-term Effect of Reldesemtiv on ALSFRS-R Functional Outcomes
timeFrame: Baseline to Week 32 (last timepoint before study was terminated prematurely)
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: St. Joseph's Hospital & Medical Center - Barrow Neurological Institute
state: Arizona
city: Orange
country: United States
facility: University of California Irvine - ALS & Neuromuscular Center
state: California
city: San Francisco
country: United States
facility: California Pacific Medical Center - Forbes Norris MDA/ALS Research Center
state: California
city: Aurora
country: United States
facility: University of Colorado Hospital Anschutz Outpatient Pavilion
state: Colorado
city: Washington
country: United States
facility: George Washington Medical Faculty Associates
state: District of Columbia
city: Jacksonville
country: United States
facility: University of Florida
state: Florida
city: Jacksonville
country: United States
facility: Mayo Clinic Jacksonville
state: Florida
city: Tampa
country: United States
facility: University of South Florida - Carol and Frank Morsani Center for Advanced Health Care
state: Florida
city: Indianapolis
country: United States
facility: Indiana University IU Health Neuroscience Center
state: Indiana
city: Kansas City
country: United States
facility: The University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins Outpatient Center
state: Maryland
city: Ann Arbor
country: United States
facility: Michigan Medicine
state: Michigan
city: Detroit
country: United States
facility: Henry Ford Hospital
state: Michigan
city: Lincoln
country: United States
facility: Neurology Associates
state: Nebraska
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical University Institute for Human Performance
state: New York
city: Charlotte
country: United States
facility: Atrium Health Neuroscience Institute
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Dallas
country: United States
facility: Texas Neurology, P.A.
state: Texas
city: Henrico
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Milwaukee
country: United States
facility: Froedtert Hospital - Department of Neurology
state: Wisconsin
city: Concord
country: Australia
facility: Concord Repatriation General Hospital
state: New South Wales
city: Herston
country: Australia
facility: Royal Brisbane and Women's Hospital, Neurology Department
state: Queensland
city: Nedlands
country: Australia
facility: The Perron Institute
city: Leuven
country: Belgium
facility: Uz Leuven Gasthuisberg Department of Neurology
city: Calgary
country: Canada
facility: University of Calgary - Heritage Medical Research Clinic
state: Alberta
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Fredericton
country: Canada
facility: Stan Cassidy Centre for Rehabilitation
state: New Brunswick
city: Hamilton
country: Canada
facility: McMaster University
state: Ontario
city: Ottawa
country: Canada
facility: Ottawa Hospital Research Institute
state: Ontario
city: Toronto
country: Canada
facility: Sunnybrook Health Sciences Centre
state: Ontario
city: Montréal
country: Canada
facility: Montreal Neurological Institute and Hospital
state: Quebec
city: Saskatoon
country: Canada
facility: University of Saskatchewan
state: Saskatchewan
city: Quebec
country: Canada
facility: CHU de Quebec-Universite Laval
city: Dublin
country: Ireland
facility: RSCI Education and Research Center Beaumount Hospital
city: Milan
country: Italy
facility: IRCCS Istituto Auxologico Italiano Ospedale San Luca U.O. Neurologia e Stroke Unit
city: Torino
country: Italy
facility: AOU Citta della Salute e della Scienza di Torino Universita degli Studi di Torino P.O. Mollinette - Dipartimento de Neuroscienze "Rita Levi Montalcini"
city: Utrecht
country: Netherlands
facility: UMC Utrecht Department of Neurology, ALS Center
city: Madrid
country: Spain
facility: Hospital San Rafael
city: Valencia
country: Spain
facility: Hospital Universitario y Politecnico La Fe
city: Stockholm
country: Sweden
facility: Studieenheten, Akademiskt Specialistcentrum
Study officials:
affiliation: Scientific Leadership at Cytokinetics
name: Cytokinetics MD
role: STUDY_CHAIR
References: