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    EudraCT Number:2021-004727-33
    Sponsor's Protocol Code Number:CY5032
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-11-18
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2021-004727-33
    A.3Full title of the trial
    A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)
    Una Fase 3, di Estensione in Aperto di COURAGE-ALS (CY 5031)
    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.
    Studio Clinico per pazienti con diagnosi di Sclerosi Laterale Amiotrofica (SLA) e che hanno già partecipato allo studio clinico CY 5031.
    A.3.2Name or abbreviated title of the trial where available
    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 Plan
    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 code2132
    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. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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)
    Sclerosi Laterale Amiotrofica (SLA)
    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.
    La SLA (nota anche come malattia di Lou Gehrig o malattia dei motoneuroni) è una degenerazione progressiva e fatale del 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.
    Valutare nel lungo termine la sicurezza e tollerabilità di reldesemtiv in pazienti affetti da SLA.
    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
    Valutare nel lungo termine l’effetto di reldesemtiv sugli esiti funzionali in base allo strumento ALSFRS-R e sulle
    ospedalizzazioni mediante il confronto fra gruppo con inizio precoce del trattamento rispetto al gruppo con inizio ritardato nell’ambito dello studio 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
    -Soggetto in grado di comprendere, e disposto a firmare, un modulo di consenso informato (ICF). Qualora il consenso venga rilasciato non per iscritto, il testimone imparziale del paziente dovrà sottoscrivere il modulo di consenso informato
    -Soggetto che ha completato la somministrazione nell’ambito dello studio 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
    - Soggetto che ha assunto un medicinale sperimentale (diverso da reldesemtiv) nei 30 giorni precedenti il trattamento, o un periodo corrispondente a cinque emivite dell’agente pregresso, quale dei due periodi sia il più lungo
    E.5 End points
    E.5.1Primary end point(s)
    The incidence of adverse events (AEs) in the patient population
    Incidenza di eventi avversi (AE) nella popolazione di pazienti
    E.5.1.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 settimane
    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 al primo manifestarsi di insufficienza respiratoria (quale tracheotomia per qualsiasi
    motivo oppure uso di ventilazione non invasiva (non-invasive ventilation, NIV) per =22 ore al
    giorno per =10 giorni consecutivi) oppure al decesso nell’intervallo fra la data di randomizzazione nello studio CY 5031 e la Settimana 48 dello studio CY 5032
    - Tempo alla prima ospedalizzazione nell’intervallo fra il Giorno 1 dello studio CY 5031 fino alla Settimana 48 dello studio CY 5032
    - Valutazione combinata delle variazioni nel punteggio totale ALSFRS-R, nel tempo all’insorgenza di insufficienza respiratoria e nella durata della sopravvivenza nell’intervallo fra il baseline dello studio CY 5031 fino alla Settimana 48 dello studio CY 5032, e nell’intervallo fra la Settimana 24 dello studio CY 5031 fino alla Settimana 48 dello studio CY 5032
    - Variazioni del punteggio totale ALS Functional Rating Scale – Revised (ALSFRS-R) nell’intervallo fra il baseline di CY 5031 fino alla Settimana 48 dello studio CY 5032, e nell’intervallo fra la Settimana 24 dello studio CY 5031 fino alla Settimana 48 dello studio CY 5032
    - Pendenza delle variazioni del punteggio totale ALSFRS-R nell’intervallo fra il baseline dello
    studio CY 5031 fino alla Settimana 48 dello studio CY 5032, e nell’intervallo fra la Settimana
    24 dello studio CY 5031 fino alla Settimana 48 dello studio CY 5032
    E.5.2.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 settimane
    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. 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 No
    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 Yes
    E. trial design description
    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.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned5
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    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
    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 state25
    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
    Alla fine delle 48 settimane, i pazienti potranno entrare nel programma di accesso gestito (Managed Access Program, MAP) per reldesemtiv. Se il medico curante acconsente a partecipare al programma, il/i suo/suoi paziente/i potrebbe/potrebbero essere idoneo/i a passare dallo studio OLE al programma 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 Decision2023-01-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-12-12
    P. End of Trial
    P.End of Trial StatusOngoing
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