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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04944784




Registration number
NCT04944784
Ethics application status
Date submitted
16/06/2021
Date registered
30/06/2021

Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)
Scientific title
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)
Secondary ID [1] 0 0
2020-004040-29
Secondary ID [2] 0 0
CY 5031
Universal Trial Number (UTN)
Trial acronym
COURAGE-ALS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Amyotrophic Lateral Sclerosis 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Reldesemtiv
Treatment: Drugs - Placebo

Experimental: 300 mg reldesemtiv twice daily for a 600 mg total daily dose, from Day 1 until Week 24 - Patients in this arm take 2 reldesemtiv 150 mg oral tablets twice a day for a 600 mg total daily dose from Day 1 until Week 24.

Placebo comparator: Placebo twice daily, from Day 1 until Week 24 - Patients in this arm take 2 placebo oral tablets twice a day from Day 1 until Week 24.

Experimental: 300 mg reldesemtiv twice daily for a 600 mg total daily dose, from Week 24 until Week 48 - Patients in this arm take 2 reldesemtiv 150 mg oral tablets twice a day for a 600 mg total daily dose from Week 24 until Week 48 for patients who were not down titrated during the 24 weeks of blinded dosing.

Experimental: 150 mg reldesemtiv twice daily for a 300 mg total daily dose, from Week 24 until Week 48 - Patients in this arm take 1 reldesemtiv 150 mg oral tablet twice a day for a 300 mg total daily dose from Week 24 until Week 48 for patients who were down titrated for any reason during the 24 weeks of blinded dosing.


Treatment: Drugs: Reldesemtiv
Reldesemtiv Oral Tablet

Treatment: Drugs: Placebo
Placebo Oral Tablet
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Effect of reldesemtiv versus placebo on functional outcomes in ALS
Timepoint [1] 0 0
Baseline to Week 24
Secondary outcome [1] 0 0
Effect of reldesemtiv versus placebo on combined functional and survival outcomes in ALS
Timepoint [1] 0 0
24 Weeks
Secondary outcome [2] 0 0
Effect of reldesemtiv versus placebo on ventilatory function
Timepoint [2] 0 0
Baseline to Week 24
Secondary outcome [3] 0 0
Effect of reldesemtiv versus placebo on quality of life
Timepoint [3] 0 0
Baseline to Week 24
Secondary outcome [4] 0 0
Effect of reldesemtiv versus placebo on handgrip strength
Timepoint [4] 0 0
Baseline to Week 24

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/08/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
18/07/2023
Sample size
Target
Accrual to date
Final
486
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,WA
Recruitment hospital [1] 0 0
Brain and Mind Centre - Camperdown
Recruitment hospital [2] 0 0
Concord Repatriation General Hospital - Concord
Recruitment hospital [3] 0 0
Royal Brisbane and Women's Hospital - Herston
Recruitment hospital [4] 0 0
Flinders Medical Centre - Bedford Park
Recruitment hospital [5] 0 0
The Perron Institute - Nedlands
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2139 - Concord
Recruitment postcode(s) [3] 0 0
4029 - Herston
Recruitment postcode(s) [4] 0 0
5042 - Bedford Park
Recruitment postcode(s) [5] 0 0
6009 - Nedlands
Recruitment outside Australia
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United States of America
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Arizona
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California
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Colorado
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District of Columbia
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Florida
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Illinois
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Kansas
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Massachusetts
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Michigan
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Missouri
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Nebraska
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Texas
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Vermont
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Virginia
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Wisconsin
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Belgium
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Leuven
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Canada
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Ontario
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Quebec
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Saskatchewan
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Copenhagen
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France
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Bron
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France
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Lille
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France
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France
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Marseille
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France
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Nice
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France
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Paris
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France
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Tours
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Germany
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Berlin
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Bonn
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Germany
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Hanover
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Germany
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Jena
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Lübeck
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Germany
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Ulm
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Ireland
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Dublin
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Italy
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Milano
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Italy
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Milan
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Italy
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Turin
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Netherlands
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Utrecht
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Poland
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Warsaw
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Portugal
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Lisboa
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Barcelona
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Bilbao
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Madrid
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Valencia
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Malmö
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Stockholm
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Switzerland
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Saint Gallen
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Liverpool
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United Kingdom
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London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Cytokinetics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Cytokinetics, MD
Address 0 0
Cytokinetics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04944784