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    Summary
    EudraCT Number:2018-000550-21
    Sponsor's Protocol Code Number:20170534
    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:2021-06-15
    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 number2018-000550-21
    A.3Full title of the trial
    Multicenter, Single-arm Open-label Extension Study to Assess Long term Safety and Efficacy of Current or Prior Treatment with Denosumab in Children/Young Adults with Osteogenesis Imperfecta
    Studio di estensione multicentrico, in aperto, a braccio singolo, per valutare la sicurezza e l’efficacia a lungo termine del trattamento precedente o attuale con denosumab in bambini/giovani adulti con osteogenesi imperfetta
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Assess Long term Safety and Efficacy of Current or Prior
    Treatment with Denosumab in Children/Young Adults with Osteogenesis
    Imperfecta
    Estensione in aperto dello studio 20130173 su denosumab in bambini e giovani adulti con osteogenesi imperfetta
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code number20170534
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/058/2016
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAMGEN 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 supportAmgen Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmgen S.r.l.
    B.5.2Functional name of contact pointMedical Information
    B.5.3 Address:
    B.5.3.1Street AddressVia Tazzoli 6
    B.5.3.2Town/ cityMilano
    B.5.3.3Post code20154
    B.5.3.4CountryItaly
    B.5.4Telephone number0039026241121
    B.5.5Fax number0039026241121
    B.5.6E-mailmedicalinformationitaly@amgen.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 Yes
    D.2.1.1.1Trade name XGEVA
    D.2.1.1.2Name of the Marketing Authorisation holderAmgen Europe B.V.
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameDenosumab
    D.3.2Product code [AMG 162]
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDenosumab
    D.3.9.1CAS number 615258-40-7
    D.3.9.2Current sponsor codeAMG 162
    D.3.9.3Other descriptive nameDenosumab - Immunoglobulin G2 Human Monoclonal Antibody to RANK Ligand
    D.3.9.4EV Substance CodeSUB29173
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number70
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Osteogenesis Imperfecta (OI)
    Osteogenesi Imperfetta
    E.1.1.1Medical condition in easily understood language
    Osteogenesis Imperfecta (OI)
    Osteogenesi Imperfetta
    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 10031243
    E.1.2Term Osteogenesis imperfecta
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic 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 long-term safety of denosumab in subjects with pediatric OI completing Study 20130173
    Valutare la sicurezza a lungo termine di denosumab in soggetti pediatrici con osteogenesi imperfetta (OI) che completeranno lo studio 20130173
    E.2.2Secondary objectives of the trial
    -To describe changes in bone mineral density (BMD) of lumbar spine and proximal femur (total hip and femoral neck) from baseline to 6, 12 and 24 months
    -To describe the incidence of X-ray confirmed long bone and new and worsening vertebral fractures from baseline to 12 and 24 months
    -To describe the incidence of vertebral and non-vertebral fractures from baseline to 12 months and 24 months
    -To describe the change in growth velocity (determined by calculating age-adjusted Z-scores for height, weight, and body mass index [BMI]) at 12 and 24 months
    -Descrivere le variazioni della densità minerale ossea (BMD) della colonna lombare e del femore prossimale (femore totale e collo femorale) dal basale a 6, 12 e 24 mesi
    -Descrivere l’incidenza di fratture delle ossa lunghe confermate dall’esame radiografico e di fratture vertebrali nuove o più gravi dal basale a 12 e 24 mesi
    -Descrivere l’incidenza di fratture vertebrali e non vertebrali dal basale a 12 e 24 mesi.
    -Descrivere la variazione della velocità di crescita (determinata calcolando lo Z-score in base all'età per altezza, peso e indice di massa corporea [BMI]) a 12 e 24 mesi
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects are eligible to be included in the study only if all of the following criteria apply:
    -Subject has provided informed consent/assent prior to initiation of any Study 20170534 specific activities/procedures.
    Subject's legally acceptable representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.
    - Subject was enrolled in Study 20130173 and:
    - Completed the 20130173 EOS visit (regardless of completing or ending investigational product early).
    OR
    - Subjects who do not reconsent to transition to 3-Month Dosing Regimen on Study 20130173 are also eligible for enrollment
    OR
    - Early terminated from Study 20130173 as a result of meeting BMD Z score IP stopping criteria and was required to early terminate from the study.
    I soggetti possono essere inclusi nello studio solo se rispettano tutti i seguenti criteri:
    -Il soggetto ha fornito il consenso informato/assenso prima di iniziare qualsiasi specifica attività/procedure nello Studio 20170534.
    -Il tutore legale rappresentante del soggetto ha fornito Consenso informato quando il soggetto è legalmente troppo giovane per fornire consenso e il soggetto ha fornito un assenso scritto sulla base di regolamenti e/o linee guida prima che qualunque attività/procedure specifica dello studio sia avviata.
    -Il soggetto è stato arruolato nello studio 20130173 e:
    - ha completatto la visita di EoS per lo studio 20130173 (indipendentemente dal completamento o dalla fine anticipata del prodotto sperimentale)
    O
    - sono anche eleggibili i soggetti che non hanno acconsentito al passaggio al regime posologico di 3 mesi nello studio 20130173
    O
    - lo studio 20130173 è terminato in anticipo in seguito al raggiungimento dei criteri di stop secondo BMD Z-score
    E.4Principal exclusion criteria
    Subjects are excluded from the study if any of the following criteria apply:
    Prior/Concomitant Therapy
    -Treatment with any prohibited proscribed medications during Study 20130173 while receiving denosumab. Eligibility into study treatment with alternative osteoporosis medication/s of investigator's choice, follow guidelines per the specific alternative osteoporosis medication/s selected. For subjects off-treatment (observation only), no prohibited medications apply.
    Prior/Concurrent Clinical Study Experience
    -Subjects currently receiving treatment in another investigational device or drug study other than Study 20130173. Other investigational procedures while participating in this study are excluded.
    Other Exclusions
    - For subjects expected to receive investigational product (denosumab)
    at study day 1:
    -Female subject is pregnant or breastfeeding or planning to become pregnant or breastfeed during treatment and for an additional 5 months after the last dose of denosumab. Females of childbearing potential (Tanner Stage > = 2) should only be included in the study after a negative highly sensitive urine or serum pregnancy test.
    - For subjects expected to receive investigational product (denosumab) at study day 1:
    -Female subjects of childbearing potential unwilling to practice true sexual abstinence (refrain from heterosexual intercourse) or use 1 highly effective method of contraception during treatment and for an additional 5 months after the last dose of investigational product (IP) (denosumab). For study treatment with alternative osteoporosis medication/s of investigator's choice, follow contraception guidelines per the specific alternative osteoporosis medication/s selected. For subjects not receiving any IP (observation only), no contraception required. Refer to Appendix 1 for additional contraceptive information.
    -History or evidence of any other clinically significant disorder, condition or disease (with the exception of those outlined above) that, in the opinion of the investigator or Amgen physician, if consulted, would pose a risk to subject safety or interfere with the study evaluation, procedures or completion.
    I soggetti sono esclusi dallo studio se uno dei seguenti criteri è rispettato:
    Precedente/ concomitante terapia
    - trattamento con qualsiasi farmaco prescrivibile proibito durante lo studio 20130173 mentre si riceve denosumab. L'eleggibilità per il trattamento in studio con farmaci alternativi per l'osteoporosi su scelta dello sperimentatore, segue le linee guida specifiche per il trattamento prescelto. Per i soggetti fuori trattamento (solo osservazionali), non c'è nessun divieto
    Esperienza di studio clinico precedente/concomitante
    -Soggetti che attualmente ricevono il trattamento per un altro dispositivo sperimentale
    o farmaco oltre che per lo studio 20130173. Altre procedure sperimentali durante la partecipazione a questo studio sono esclusi.
    Altre esclusioni
    - Per i soggetti che riceveranno il medicinale in sperimentazione (denosumab) al giorno 1:
    -Il soggetto femminile è incinta o sta allattando o sta pianificando gravidanza o allattare durante il trattamento e per ulteriori 5 mesi dopo l'ultima dose di denosumab. Soggetti Femminili potenzialmente fertili (Tanner stage > = 2) deve essere incluso nello studio solo dopo risultato negativo al test delle urine o test di gravidanza sierica. per il trattamento in studio con farmaci alternativi per l'osteoporosi su scelta dello sperimentatore, segue le linee guida specifiche per il trattamento prescelto. Per i soggetti fuori trattamento (solo osservazionali), non c'è nessun divieto.
    - Per i soggetti che riceveranno il medicinale in sperimentazione (denosumab) al giorno 1:
    -Soggetti femminili potenzialmente fertili che non vogliono praticare l'astinenza sessuale (astenersi da rapporti eterosessuali) o usare 1 Metodo contraccettivo altamente efficace durante il trattamento e per ulteriori 5 mesi dopo l'ultima somministrazione del farmaco sperimentale (denosumab).
    Per il trattamento di studio con osteoporosi alternativa farmaci / i della scelta dello sperimentatore, seguire le linee guida sulla contraccezione per il / i farmaco / i di osteoporosi alternativo / i selezionato / i. Per i soggetti che non ricevono alcun prodotto sperimentale ( studio solo osservazione), nessuna contraccezione è
    necessaria. Fare riferimento all'Appendice 1 per ulteriori informazioni contraccettive.
    -Storia o evidenza di qualsiasi altro disturbo clinicamente significativo, condizione o malattia (con l'eccezione di quelli delineati sopra) che, nel l'opinione dello sperimentatore o del medico Amgen, se consultata, porrà un rischio per la sicurezza dei soggetti o interferire con la valutazione dello studio, procedure o completamento.
    E.5 End points
    E.5.1Primary end point(s)
    L’endpoint primario è il monitoraggio della sicurezza, che include incidenza per soggetto degli eventi avversi, degli eventi avversi seri e degli eventi avversi di speciale interesse, incidenza per soggetto degli anticorpi anti-denosumab, variazioni dal basale dei valori di laboratorio e dei segni vitali, incidenza per soggetto di Z-score relativo all’indice di metafisi superiore all'intervallo di normalità adeguato per l’età del paziente, eruzione molare anomala e conformazione mandibolare
    -The primary endpoint is safety monitoring, including subject incidence of adverse events, serious adverse events and adverse events of special interest, subject prevalence of immunogenicity, changes from baseline in laboratory values and vital signs, and subject incidence of metaphyseal index Z score above age-appropriate normal range, abnormal molar eruption, and mandibular shaping
    E.5.1.1Timepoint(s) of evaluation of this end point
    at 3, 12 and 24 months
    a 3, 12 e 24 mesi
    E.5.2Secondary end point(s)
    - Actual values and changes in BMD Z score of lumbar spine and proximal femur (total hip and femoral neck) from baseline and from Study 20130173 baseline of 3-Month Dosing Regimen, as assessed by dual X-ray absorptiometry (DXA), to 6, 12 and 24 months
    -Incidence of X-ray confirmed long bone and new and worsening vertebral fractures from baseline, and from Study 20130173 baseline of 3-Month Dosing Regimen, to 12 and 24 months
    - Incidence of vertebral and nonvertebral fractures from baseline and from Study 20130173 baseline of 3-Month Dosing Regimen to 12 and 24 months
    - Change from baseline and from Study 20130173 baseline of 3-Month Dosing Regimen in growth velocity (determined by calculating age adjusted Z-scores for height, weight, and BMI) at 12 and 24 months
    - Valori effettivi e variazioni del punteggio BMD Z della colonna lombare e del femore prossimale (anca totale e collo femorale) dal basale e dal basale del regime posologico a 3 mesi dello Studio 20130173 , valutato da doppia absorptiometry a raggi X (DXA), a 6, 12 e 24 mesi
    - Incidenza di fratture vertebrali delle ossa lunghe nuove e in peggioramento confermate ai raggi X dal basale e dallo studio 20130173 al basale del regime posologico di 3 mesi, a 12 e 24 mesi
    - Incidenza di fratture vertebrali e non vertebrali dal basale e dallo studio 20130173 al basale del regime posologico di 3 mesi, a 12 e 24 mesi
    - Variazione rispetto al basale e dallo studio 20130173 al basale del regime posologico di 3 mesi nella velocità di crescita (determinato calcolando l'età aggiustato Z-score per altezza, peso e BMI) a 12 e 24 mesi
    E.5.2.1Timepoint(s) of evaluation of this end point
    at 3, 12 and 24 months
    a 3, 12 e 24 mesi
    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 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 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.8.2.4Number of treatment arms in the trial1
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA23
    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
    Austria
    Belgium
    Bulgaria
    Canada
    Czechia
    France
    Germany
    Hungary
    Italy
    Poland
    Spain
    United Kingdom
    United States
    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
    The primary completion date is the date when the last subject has completed the assessments for EOS. EOS is anticipated to occur at Week 96, except for those subjects enrolled prior to this protocol
    amendment and transitioning to a 3-Month Dosing Regimen. These subjects will remain on 3-Month Dosing Regimen for a minimum of 12-Months.
    La data di conclusione dello studio è definita come la data nella quale l'ultimo paziente ha completato la visita per l'EoS. La EoS è anticipata alla settimana 96, eccetto per quei soggetti arruolati prima di questo emendamento al protocollo e che arrivano dal regime posologico di 3 mesi. Questi soggetti manterranno il regime posologico di 3 mesi per un minimo di 12 mesi.
    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 months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months1
    E.8.9.2In all countries concerned by the trial days5
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 49
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 65
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 36
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Subjects under age incapable of giving consent personally.
    Sogetti minorenni incapaci di dare il proprio consenso personale.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 105
    F.4.2.2In the whole clinical trial 150
    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 Decision2019-06-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-04-18
    P. End of Trial
    P.End of Trial StatusOngoing
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