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    Summary
    EudraCT Number:2015-001411-12
    Sponsor's Protocol Code Number:CBYM338B2203E1
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-02-27
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2015-001411-12
    A.3Full title of the trial
    Extension of the CBYM338B2203 phase IIb/III study to evaluate the longterm
    efficacy, safety and tolerability of intravenous BYM338 in patients
    with sporadic inclusion body myositis
    Estensione dello studio di fase IIb/III CBYM338B2203 per valutare l’efficacia, la sicurezza e la tollerabilità a lungo termine di BYM338 somministrato per via endovenosa in pazienti con miosite sporadica da corpi inclusi
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Extension study to evaluate the long-term efficacy, safety and tolerability
    of BYM338 in patients with sporadic inclusion body myositis
    Estensione dello studio di fase IIb/III CBYM338B2203 per valutare l’efficacia, la sicurezza e la tollerabilità a lungo termine di BYM338 in pazienti con miosite sporadica da corpi inclusi
    A.3.2Name or abbreviated title of the trial where available
    Extension study to evaluate the long-term efficacy, safety and tolerability
    Estensione dello studio per valutare l'efficacia, la sicurezza e la tollerabilità a lungo termine
    A.4.1Sponsor's protocol code numberCBYM338B2203E1
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN12345678
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT12345678
    A.5.3WHO Universal Trial Reference Number (UTRN)U1234-1234-1234
    A.5.4Other Identifiers
    Name:NANumber:CBYM338B2203E1
    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 SponsorNOVARTIS FARMA S.P.A.
    B.1.3.4CountryItaly
    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 supportNovartis Pharma A.G
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNOVARTIS FARMA S.p.A
    B.5.2Functional name of contact pointDrug Regulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressLargo Umberto Boccioni, 1
    B.5.3.2Town/ cityORIGGIO
    B.5.3.3Post code21040
    B.5.3.4CountryItaly
    B.5.4Telephone number+39 02 96541
    B.5.5Fax number+39 02 9659066
    B.5.6E-mailinfo.studiclinici@novartis.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 numberEMA/OD/046/12
    D.3 Description of the IMP
    D.3.1Product nameBimagrumab
    D.3.2Product code BYM338
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee 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 Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Sporadic Inclusion Body Myositis

    Miosite sporadica da corpi inclusi
    E.1.1.1Medical condition in easily understood language
    Myositis
    Miosite
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10066407
    E.1.2Term Inclusion body myositis
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10066407
    E.1.2Term Inclusion body myositis
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue 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 evaluate the long-term safety and tolerability of BYM338
    Valutare la sicurezza e la tollerabilità a lungo termine di BYM338
    E.2.2Secondary objectives of the trial
    - To describe the long-term evolution of quadriceps muscle strength
    using quadriceps Quantitative Muscle Testing (QMT)
    - To describe physical function reported by patients using the Sporadic
    Inclusion Body Myositis Functional Assessment (sIFA)
    - To report the incidence of self-reported falls and self-reported injurious
    falls (falls that result in any subject injury) which are a subset of all selfreported
    falls
    - To describe physical performance using the Short Physical Performance
    Battery (SPPB)
    - To characterize muscle changes using magnetic resonance imaging
    (MRI) from a subset of patients
    - To investigate the development of immunogenicity against BYM338
    - Descrivere i cambiamenti sul lungo periodo della forza dei quadricipiti mediante il test muscolare quantitative (QMT)
    - Descrivere la funzionalità fisica riferita dai pazienti mediante il Sporadic Inclusion Body Myositis Functional Assessment (sIFA)
    - Riportare l’incidenza delle cadute riferite dai pazienti e delle cadute e delle cadute “dannose” riferite dai pazienti (cadute che hanno portato ad un danno del paziente)
    - Descrivere la prestazione fisica mediante la Short Physical Performance Battery (SPPB)
    - Caratterizzare i cambiamenti muscolari tramite la risonanza magnetica nucleare in un sottogruppo di pazienti
    - Indagare lo sviluppo dell’immunogenicità verso bimagrumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Patients who completed the core study
    - Written informed consent must be obtained before any extension study
    assessment is performed
    - Able to communicate well with the investigator
    - Willing to participate for the entire duration of the extension study with
    commitment to follow study requirements and procedures.
    Pazienti di sesso maschile e femminile che hanno completato lo studio principale, ossia che hanno ricevuto la dose di farmaco alla Settimana 48 e hanno completato la visita di fine studio e di follow-up dello studio principale.
    - Consenso informato scritto ottenuto prima dell’effettuazione di qualsiasi valutazione
    - Pazienti in grado di comunicare bene con lo sperimentatore
    - Pazienti disponibili a partecipare all’intera durata dello studio di estensione con impegno a seguire tutti i requisiti e le procedure dello studio.
    E.4Principal exclusion criteria
    - Women who are pregnant
    - Women of child-bearing potential unless they are using highly effective
    methods of contraception during dosing and for 6 months after the last
    BYM338 dose
    - Current use of prohibited treatments
    - History of severe hypersensitivity reaction in the core study
    - History of adverse event(s) (including those from the core study) prior
    to the start of study drug in the extension study that, in the judgment of
    the investigator, taking into account the subject's overall status, prevent
    the subject from entering the extension study
    - Clinically significant abnormal liver function tests
    - Any medical condition or laboratory finding which, in the opinion of the
    investigator may interfere with participation in the study, might
    confound the results of the study, or pose an additional safety risk in
    administering BYM338
    Other protocol-defined exclusion criteria may apply.
    - Gravidanza: test di gravidanza sul siero positivo (hcG)
    - Pazienti di sesso femminile potenzialmente in grado di avere figli, definiti come tutti i soggetti di sesso femminile fisiologicamente in grado di iniziare una gravidanza, a meno che stiano usando metodi di contraccezione altamente efficaci durante il trattamento e per 6 mesi dopo l’ultima dose di BYM338
    - Uso di farmaci concomitanti non consentiti
    - Anamnesi positiva per ipersensibilità severa durante lo studio principale
    - Storia di eventi avversi (inclusi quelli occorsi nello studio principale) prima dell’inizio del farmaco in studio nello studio di estensione, che, a giudizio dello sperimentatore, considerando lo stato generale del paziente, impedisca l’ingresso nello studio di estensione.
    - Anomalie significative nei parametri di funzionalità epatica, come SGOT (AST), SGPT (ALT), fosfatasi alcalina, o bilirubina (ad eccezione della sindrome di Gilbert), che avrebbero portato all’interruzione del trattamento durante lo studio principale secondo il protocollo
    - Qualsiasi condizione medica o anomalia negli esami di laboratorio (per esempio valori di laboratorio inaspettati o clinicamente significativi, ECG, ecc), che, a giudizio dello sperimentatore, potrebbe interferire con la partecipazione allo studio, potrebbe confonderne i risultati, o rappresentare un rischio aggiuntivo nella somministrazione di BYM338.
    E.5 End points
    E.5.1Primary end point(s)
    - Safety and tolerabilty of different i.v. BYM338 doses
    - Change from baseline in 6 Minute Walking Distance Test (6MWD)
    - Sicurezza e tollerabilità di dose diverse di BYM338 e.v.
    - Test del cammino in 6 minuti (6MWD)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline, 1 and 2 years
    Basale, 1 e 2 anni
    E.5.2Secondary end point(s)
    - Change from baseline in quadriceps muscle strength
    - Change from baseline in patient-reported physical performance
    - Incidence of patients with self-reported falls and self reported injurious falls
    - Change from baseline in physical performance
    - Change in muscles of the thigh
    - Number of patients who develop immunogenicity against BYM338
    - Descrivere i cambiamenti sul lungo periodo della forza dei quadricipiti mediante il test muscolare quantitative (QMT)
    - Descrivere la funzionalità fisica riferita dai pazienti mediante il Sporadic Inclusion Body Myositis Functional Assessment (sIFA)
    - Riportare l’incidenza delle cadute riferite dai pazienti e delle cadute e delle cadute “dannose” riferite dai pazienti (cadute che hanno portato ad un danno del paziente)
    - Descrivere la prestazione fisica mediante la Short Physical Performance Battery (SPPB)
    - Caratterizzare i cambiamenti muscolari tramite la risonanza magnetica nucleare in un sottogruppo di pazienti
    - Indagare lo sviluppo dell’immunogenicità verso bimagrumab
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Change from baseline in quadriceps muscle strength: Baseline, 1 and 2
    years
    - Change from baseline in patient-reported physical performance:
    Baseline, 1 and 2 years
    - Incidence of patients with self-reported falls and self reported injurious
    falls: Baseline, 1 and 2 years
    - Change from baseline in physical performance: Baseline, 1 and 2 years
    - Change in muscles of the thigh: 1 and 2 years
    - Number of patients who develop immunogenicity against BYM338: 2
    years
    - Change from baseline in quadriceps muscle strength: Baseline, 1 and 2
    years
    - Change from baseline in patient-reported physical performance:
    Baseline, 1 and 2 years
    - Incidence of patients with self-reported falls and self reported injurious
    falls: Baseline, 1 and 2 years
    - Change from baseline in physical performance: Baseline, 1 and 2 years
    - Change in muscles of the thigh: 1 and 2 years
    - Number of patients who develop immunogenicity against BYM338: 2
    years
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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 No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Periodo di Trattamento 2 epoch dello studio in aperto senza placebo
    Treatment Period 2 epoch of the study is open-label without placebo
    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 trial4
    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 EEA15
    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
    Australia
    Belgium
    Denmark
    France
    Italy
    Japan
    Netherlands
    Switzerland
    United Kingdom
    United States
    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
    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 months2
    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 months2
    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.1.1Number of subjects for this age range: 1
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 72
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 168
    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 No
    F.3.3.1Women of childbearing potential not using contraception Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 94
    F.4.2.2In the whole clinical trial 240
    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)
    None
    Nessuno
    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 Decision2015-10-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-09-08
    P. End of Trial
    P.End of Trial StatusCompleted
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