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    Summary
    EudraCT Number:2017-004972-74
    Sponsor's Protocol Code Number:20160227
    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:2021-01-21
    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 number2017-004972-74
    A.3Full title of the trial
    An Open-label, Ascending Multiple-dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
    Studio in aperto a dosi multiple ascendenti volto a valutare la sicurezza, la tollerabilità, la farmacocinetica e la farmacodinamica di romosozumab in bambini e adolescenti con osteogenesi imperfetta.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-label, Ascending Multiple dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents with Osteogenesis Imperfecta
    Studio in aperto a dosi multiple ascendenti volto a valutare la sicurezza, la tollerabilità, la farmacocinetica e la farmacodinamica di romosozumab in bambini e adolescenti con osteogenesi imperfetta.
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code number20160227
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/066/2016
    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 Evenity 105mg
    D.2.1.1.2Name of the Marketing Authorisation holderUCB Pharma S.A.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameRomosozumab
    D.3.2Product code [AMG 785]
    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 INNROMOSOZUMAB
    D.3.9.1CAS number 909395-70-6
    D.3.9.2Current sponsor codeAMG 785
    D.3.9.4EV Substance CodeSUB187492
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number90
    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 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.IMP: 2
    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 CALCIUM D3 SANDOZ - 1000 MG + 880 U.I. COMPRESSE EFFERVESCENTI 30 COMPRESSE DIVISIBILI
    D.2.1.1.2Name of the Marketing Authorisation holderSANDOZ S.P.A.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameCalcium D3
    D.3.2Product code [vitamina D e calcio]
    D.3.4Pharmaceutical form Effervescent 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 INNCALCIO CARBONATO/COLECALCIFEROLO
    D.3.9.2Current sponsor codevitamina D e calcio
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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 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 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
    Osteogenesis imperfecta (OI) is a group of genetic skeletal disorders characterized by increased bone fragility, low bone mass, and increased bone turnover contributing to osteoporosis, fractures, and other conditions. OI is the most common form of primary osteoporosis in children with an estimated incidence of 1 per 25,000 live births.
    L’osteogenesi imperfetta (OI) rappresenta un gruppo di disturbi genetici dello scheletro caratterizzati da una maggiore fragilità ossea, da una bassa massa ossea e da un aumentato turnover delle ossa che contribuiscono all’osteoporosi, alle fratture e ad altre condizioni. OI è la forma più comune di osteoporosi primaria nei bambini con una incidenza stimata di 1 ogni 25000 nati vivi.
    E.1.1.1Medical condition in easily understood language
    Osteogenesis Imperfecta (Brittle bone disease)
    Osteogenesi imperfetta (malattia delle ossa fragili)
    E.1.1.2Therapeutic area Body processes [G] - Bones and nerves physological processes [G11]
    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 the pharmacokinetics (PK) profile following multiple SC doses of romosozumab in children and adolescents with OI
    Valutare il profilo farmacocinetico (PK) dopo la somministrazione di dosi sottocutanee (sc) multiple di romosozumab in bambini e adolescenti con OI
    E.2.2Secondary objectives of the trial
    - To evaluate the safety, tolerability, and immunogenicity profile following multiple SC doses of romosozumab in children and adolescents with OI
    - To evaluate the pharmacodynamic (PD) profile following multiple SC doses of romosozumab in children and adolescents with OI
    - Valutare il profilo di sicurezza, tollerabilità e immunogenicità dopo la somministrazione di dosi sc multiple di romosozumab in bambini e adolescenti con OI
    - Valutare il profilo farmacodinamico (PD) dopo la somministrazione di dosi sc multiple di romosozumab in bambini e adolescenti con OI
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Subject's legally acceptable representative has provided informed consent and the subject has provided written assent based on local regulations and/or guidelines prior to initiation of any study-specific activities/procedures
    - Ambulatory male or female children 5 to less than 12 years of age (cohorts 2,4, and 6) or adolescents 12 to less than 18 years of age (cohorts 1, 3 and 5) upon entry into screening
    - Clinical diagnosis of OI defined as a clinical history consistent with type I-IV OI as determined by presence of expected phenotype (eg, facial shape, voice, blue sclera, dentinogenesis imperfecta, typical radiographic features, fracture pattern) and lack of additional features unrelated to type I-IV OI (eg, blindness, mental retardation, neuropathy, craniosynostosis, premature exfoliation of deciduous teeth)
    - If familial, also must be autosomal dominant
    - Il rappresentante legalmente riconosciuto del soggetto ha fornito il consenso informato e il soggetto ha fornito il proprio consenso scritto secondo i regolamenti e/o linee guida locali prima dell'inizio di qualsiasi attività/procedure studio specifica
    - Bambini ambulatoriali di sesso maschile o femminile da 5 a meno di 12 anni (coorti 2,4 e 6) o adolescenti da 12 a meno di 18 anni
    (coorti 1, 3 e 5) all'inizio dello screening
    - La diagnosi clinica di IO definita come un'anamnesi clinica coerente con il tipo I-IV OI come determinato dalla presenza del fenotipo atteso (ad esempio, forma facciale, voce, sclera blu, dentinogenesi imperfetta, tipiche caratteristiche radiografiche, schema di frattura) e la mancanza di caratteristiche aggiuntive non correlate a tipo I-IV OI (ad esempio, cecità, ritardo mentale, neuropatia,
    craniosinostosi, esfoliazione precoce dei denti decidui)
    - Se familiare, deve essere anche autosomico dominante
    E.4Principal exclusion criteria
    - History of an electrophoresis pattern inconsistent with type I to type IV OI
    - History of known mutation in a gene other than collagen type I alpha/collagen type I alpha 2 (COL1AI/COL1A2) causing OI or other
    metabolic bone disease
    - History of congenital dislocation of the radial head, interosseous membrane calcification, or exuberant callus formation
    - History of osteomalacia or rickets
    - Body weight less than 10 kg or greater than 90 kg

    *Please, refer to protocol for the full list
    - Storia di un modello di elettroforesi non coerente con il tipo da Ia IV di OI
    - Anamnesi di una mutazione nota in un gene diverso dal collagene di tipo I alfa/collagene di tipo I alfa 2 (COL1AI/COL1A2) che causa OI o altro malattia metabolica dell'osso
    - Anamnesi di dislocazione congenita della testa radiale, calcificazione della membrana interossea o formazione di calli esuberanti
    - Anamnesi di osteomalacia o di rachitismo
    - Peso corporeo inferiore a 10 kg o superiore a 90 kg

    *Fare riferimento al protocollo per la lista completa
    E.5 End points
    E.5.1Primary end point(s)
    Romosozumab serum PK parameters: maximum-observed concentration (Cmax), time to Cmax (tmax), area under the curve (AUC) and terminal half life (t1/2)
    Parametri di PK sierici di romosozumab: concentrazione massima osservata (Cmax), tempo alla Cmax (tmax), area sotto la curva (AUC) ed emivita terminale (t1/2)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Investigational product will be dosed on study days 1, 29 and 57 after completion of all pre-dose procedures. Safety assessments, including blood samples for anti-romosozumab antibodies, PK and PD measurements and time points are defined in the Schedule of
    Assessments as per the Protocol.
    Il prodotto sperimentale sarà dosato nei giorni di studio 1, 29 e 57 dopo il completamento di tutte le procedure di pre-dose. Le valutazioni di sicurezza, compresi i campioni di sangue per gli anticorpi anti-romosozumab, le misurazioni PK e PD e i punti temporali sono definiti nel Programma di Valutazioni secondo il Protocollo.
    E.5.2Secondary end point(s)
    • Treatment-emergent adverse events, including events of injection site reactions and changes in cranial nerve function.Vital signs, electrocardiograms, physical examinations, and safety laboratory tests, including serum calcium
    • Incidence of anti-romosozumab antibodies
    • Bone turnover markers including serum P1NP and serum CTX measurements.
    • Lumbar spine BMD, bone mineral content (BMC), bone area, and BMD Z-score as assessed by dual-energy X-ray absorptiometry (DXA)
    - Eventi avversi rilevanti per il trattamento, compresi gli eventi di reazione al sito di iniezione e cambiamenti nella funzione del nervo cranico. Segni vitali, elettrocardiogrammi, esami fisici e test di laboratorio sulla sicurezza, compreso il calcio sierico
    - Incidenza di anticorpi anti-romosozumab
    - Misurazione dei marker per il turnover osseo compresi P1NP sierico e CTX sierico
    - Colonna vertebrale lombare BMD, contenuto minerale osseo (BMC), area ossea e BMD Z-score come valutato dalla Densitometria DEXA
    E.5.2.1Timepoint(s) of evaluation of this end point
    Investigational product will be dosed on study days 1, 29 and 57 after completion of all pre-dose procedures. Safety assessments, including blood samples for anti-romosozumab antibodies, PK and PD measurements and time points are defined in the Schedule of Assessments as per the Protocol.
    Il prodotto sperimentale sarà dosato nei giorni di studio 1, 29 e 57 dopo il completamento di tutte le procedure di pre-dose. Le valutazioni di sicurezza, compresi i campioni di sangue per gli anticorpi anti-romosozumab, le misurazioni PK e PD e i punti temporali sono definiti nel Programma di Valutazioni secondo il Protocollo.
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase 1b
    Fase 1b
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    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 EEA13
    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
    Turkey
    France
    Germany
    Greece
    Hungary
    Italy
    Spain
    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
    Defined as the date when the last subject is assessed or receives an intervention for evaluation in the study (ie, last subject last visit),
    following any additional parts in the study (eg, long-term follow-up), as applicable
    Definita come la data in cui l'ultimo soggetto viene valutato o riceve un intervento per la valutazione nello studio (cioè, Lat subject last visit) a seguito di eventuali parti aggiuntive dello studio (ad esempio, follow-up a lungo termine), a seconda dei casi
    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 months6
    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 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: 12
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    F.1.2Adults (18-64 years) No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 16
    F.4.2.2In the whole clinical trial 24
    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)
    NA
    NA
    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 Decision2020-10-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-10-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-02-07
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