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    Summary
    EudraCT Number:2021-004727-33
    Sponsor's Protocol Code Number:CY5032
    National Competent Authority:Portugal - INFARMED
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2023-01-12
    Trial results View 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 ConcernedPortugal - INFARMED
    A.2EudraCT number2021-004727-33
    A.3Full title of the trial
    A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)
    Estudo de fase III, extensão de rótulo aberto do ensaio COURAGE-ALS (CY5031)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical Study for patients who are diagnosed with Amyotrophic Lateral Sclerosis (ALS) and who have already participated in clinical study CY 5031.
    Um estudo clínico para doentes que são diagnosticados com esclerose lateral amiotrófica (ELA) e que já participaram no estudo clínico CY 5031.
    A.3.2Name or abbreviated title of the trial where available
    COURAGE-ALS OLE
    COURAGE-ALS OLE
    A.4.1Sponsor's protocol code numberCY5032
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05442775
    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/ cityWT Hoofdorp
    B.5.3.3Post codeNetherlands
    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
    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)
    Esclerose Lateral Amiotrófica (ELA)
    E.1.1.1Medical condition in easily understood language
    ALS (also known as Lou Gehrig's disease or motor neuron disease) is a progressive and fatal degeneration of the nervous system.
    ALS (também conhecida como doença de Lou Gehrig ou doença do neurônio motor) é uma degeneração progressiva e fatal do sistema nervoso.
    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 long-term safety and tolerability of reldesemtiv in patients with ALS.
    Avaliar a segurança e tolerabilidade de reldesemtiv a longo prazo em doentes com ELA.
    E.2.2Secondary objectives of the trial
    To assess the long-term effect of reldesemtiv on ALSFRS-R functional outcomes and hospitalization by comparing early-start to delayed-start groups from CY 5031
    Avaliar o efeito de reldesemtiv a longo prazo nos resultados funcionais de ALSFRS-R e hospitalização comparando os grupos de início antecipado e início retardado do CY 5031
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Able to comprehend and willing to sign an Informed Consent Form (ICF). If patient is able to comprehend, but non-written consent is given, an impartial witness of the patient must sign the ICF form
    • Completed dosing in CY 5031
    • Ter capacidade para compreender e estar disposto(a) a assinar um Formulário de consentimento informado (FCI). Se for dado um consentimento não escrito, um representante legal do(a) doente tem que assinar o formulário FCI
    • Ter concluído a dosagem no CY 5031
    E.4Principal exclusion criteria
    • Has taken an investigational study drug (other than reldesemtiv) prior to dosing, within 30 days or five half-lives of the prior agent, whichever is greater
    • Ter tomado um medicamento experimental do estudo (que não reldesemtiv) antes da dosagem, nos 30 dias ou cinco semividas do agente anterior, o que for superior
    E.5 End points
    E.5.1Primary end point(s)
    The incidence of adverse events (AEs) in the patient population
    A incidência de acontecimentos adversos (AA) na população de doentes
    E.5.1.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 semanas
    E.5.2Secondary end point(s)
    • Time to the first occurrence of respiratory insufficiency (defined as tracheostomy for any reason or the use of non-invasive ventilation (NIV) for ≥22 hours per day for ≥10 consecutive days) or death from date of randomization in CY 5031 through Week 48 of CY 5032
    • Time to the first hospitalization from Day 1 in CY 5031 through Week 48 of CY 5032
    • Combined assessment of change in ALSFRS-R total score, time to onset of respiratory insufficiency, and survival time from baseline of CY 5031 through Week 48 of CY 5032 and from Week 24 of CY 5031 through Week 48 of CY 5032
    • Changes in ALS Functional Rating Scale – Revised (ALSFRS-R) total score from baseline of CY 5031 through Week 48 of CY 5032 and from Week 24 of CY 5031 through Week 48 of CY 5032
    • Slopes of the changes in ALSFRS-R total score from baseline of CY 5031 through Week 48 of CY 5032 and from Week 24 of CY 5031 through Week 48 of CY 5032
    • Tempo até à primeira ocorrência de insuficiência respiratória (definido como traqueostomia por qualquer motivo ou utilização de ventilação não invasiva (VNI) durante ≥22 horas por dia durante ≥10 dias consecutivos), ou morte desde a data da aleatorização no CY 5031 até à Semana 48 do CY 5032
    • Tempo até à primeira hospitalização desde o Dia 1 no CY 5031 até à Semana 48 do CY 5032
    • Avaliação combinada da alteração na classificação total de ALSFRS-R, tempo até ao início da insuficiência respiratória e tempo de sobrevida desde a situação basal do CY 5031 até à Semana 48 do CY 5032, assim como desde a Semana 24 do CY 5031 até à Semana 48 do CY 5032
    • Alterações na escala de classificação funcional de ALS – Classificação total revista (ALSFRS-R) desde a situação basal do CY 5031 até à Semana 48 do CY 5032 e desde a Semana 24 do CY 5031 até à Semana 48 do CY 5032
    • Declives das alterações na classificação total de ALSFRS-R desde a situação basal do CY 5031 até à Semana 48 do CY 5032 e desde a Semana 24 do CY 5031 até à Semana 48 do CY 5032
    E.5.2.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 semanas
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    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 EEA29
    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 No
    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
    Última visita do último paciente
    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: 270
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 130
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 135
    F.4.2.2In the whole clinical trial 400
    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)
    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, the treating physician’s patient(s) may be eligible to transition from the OLE to the MAP
    No final das 48 semanas, os doentes podem transitar para um Programa de acesso gerido (MAP) do reldesemtiv. Se o médico responsável pelo tratamento concordar em participar no programa, o(s) doente(s) do médico responsável pelo tratamento poderá(ão) ser elegível(eis) para a transição do OLE para o MAP
    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 Decision2023-03-15
    N.Ethics Committee Opinion of the trial applicationWithdrawn
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-11-14
    P. End of Trial
    P.End of Trial Status
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