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    Summary
    EudraCT Number:2020-004040-29
    Sponsor's Protocol Code Number:CY5031
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-09-01
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2020-004040-29
    A.3Full title of the trial
    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).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical Study in which patients diagnosed with Amyotrophic Lateral Sclerosis (ALS) will receive Reldesemtiv (study drug). The safety and efficacy of the drug will be tested.
    A.3.2Name or abbreviated title of the trial where available
    COURAGE-ALS
    A.4.1Sponsor's protocol code numberCY5031
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation PlanP/025/2021
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCytokinetics Inc
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportCytokinetics Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAtlantic Research Group BV
    B.5.2Functional name of contact pointVice President
    B.5.3 Address:
    B.5.3.1Street AddressAmsterdam Schiphol Tetra, Siriusdreef 17 - 27
    B.5.3.2Town/ city2132 WT Hoofddorp
    B.5.3.4CountryNetherlands
    B.5.6E-mailidigiovanna@atlanticresearchgroup.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/20/2256
    D.3 Description of the IMP
    D.3.1Product namereldesemtiv
    D.3.2Product code CK-2127107
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNreldesemtiv
    D.3.9.1CAS number 1345410-31-2
    D.3.9.2Current sponsor codeCK-2127107
    D.3.9.3Other descriptive nameRELDESEMTIV
    D.3.9.4EV Substance CodeSUB191996
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Amyotrophic Lateral Sclerosis (ALS)
    E.1.1.1Medical condition in easily understood language
    ALS (also known Lou Gehrig’s disease or motor neuron disease) is a progressive and fatal degeneration of the nervous system.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10002026
    E.1.2Term Amyotrophic lateral sclerosis
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the effect of reldesemtiv versus placebo on functional outcomes in ALS. To assess the safety and tolerability of reldesemtiv compared to placebo.
    E.2.2Secondary objectives of the trial
    • To assess the effect of reldesemtiv versus placebo on combined functional and survival outcomes in ALS
    • To assess the effect of reldesemtiv versus placebo on ventilatory function
    • To assess the effect of reldesemtiv versus placebo on quality of life
    • To assess the effect of reldesemtiv versus placebo on handgrip strength

    Exploratory Objective
    To assess the effect of reldesemtiv versus
    placebo on the progression of ALS
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Key Inclusion Criteria
    • 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 published in 2000 [Brooks 2000]). 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. Cramps, fasciculations, or fatigue should not be taken in isolation as a first symptom of ALS.
    • 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 and be deemed eligible if their ALSFRS-R total score is ≤ 44 or if their score is 2 or more points less than at initial screening.
    Such patients must continue to meet all other inclusion/exclusion criteria at the time of rescreening.
    • Upright FVC ≥ 65.0% of predicted for age, height, sex and ethnicity at screening according to Global Lung Initiative equation
    • Able to perform reproducible pulmonary function tests defined as being able to perform FVC at screening with variability of the 2 highest raw values of less than 10% with a maximum of 5 trials
    permitted. Screening FVC results must be reviewed and approved by the central review process prior to randomization.
    • 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 at the time of screening
    • Clinical laboratory findings within the normal range, or if outside the normal range, not deemed clinically significant by the Investigator, except as specifically indicated as laboratory exclusion
    E.4Principal exclusion criteria
    • eGFRCr andeGFRCysC < 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.
    • Presence at screening 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
    • Has a tracheostomy
    E.5 End points
    E.5.1Primary end point(s)
    • Change from baseline to Week 24 in ALSFRS-R total score.
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 weeks
    E.5.2Secondary end point(s)
    • Combined assessment of change in ALSFRS-R total score, time to onset of respiratory insufficiency, and survival time up to Week 24
    • Change from baseline to Week 24 in the percent predicted FVC
    • Change from baseline to Week 24 in the ALSAQ-40 total score
    • Change from baseline to Week 24 in handgrip strength (average of both hands)

    Exploratory Endpoints
    • Time to the patient being prescribed and patient agrees to it, time to first receipt, time to first use, time to daily use, time to dependence and number used of any of the following durable medical equipment items (manual wheelchair, power wheelchair, augmentative and alternative communication device, noninvasive ventilation (NIV) and/or gastrostomy tube) from randomization to the end of the 24-week double blind, placebo-controlled period and to the end of Week 48
    • Change from baseline to Week 24 in the four subdomain scores of the ALSFRS-R
    • Time spent in each MiToS stage and number of patients to transition stages from baseline to Week 24
    • Change from baseline to Week 48 in ALSFRS-R total score
    • Change from baseline to Week 48 in forced vital capacity (FVC)
    • Combined assessment of change in ALSFRS-R total score, time to onset of respiratory insufficiency, and survival time up to Week 48
    • Change from baseline to Week 24 in the megascore of muscle strength measured by hand-held dynamometry (HHD) in bilateral first dorsal interosseous muscles, abductor pollicus brevis muscles, and abductor digiti minimi muscles
    • Change from baseline to Week 24 and Week 48 in the EQ-5D-5L
    • Change from baseline to Week 24 and Week 48 in the EQ-Visual Analogue Scale (VAS)
    . Time to event for first hospitalization to Week 24 and Week 48
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 weeks for secondary objectives
    48 weeks for exploratory objectives
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA24
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Canada
    United States
    France
    Poland
    Sweden
    Netherlands
    Spain
    Switzerland
    Germany
    Italy
    Belgium
    Ireland
    Portugal
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 374
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 181
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 192
    F.4.2.2In the whole clinical trial 555
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    Not yet determined
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-09-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-25
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-07-18
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