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    Summary
    EudraCT Number:2012-004867-38
    Sponsor's Protocol Code Number:NN7999-3895
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-02-15
    Trial 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 number2012-004867-38
    A.3Full title of the trial
    Safety and efficacy of nonacog beta pegol (N9-GP) in Previously Untreated Patients with Haemophila B.

    An open-label single-arm multicentre non-controlled phase 3a trial investigating safety and efficacy
    of nonacog beta pegol (N9-GP) in prophylaxis and treatment of bleeding episodes in previously
    untreated patients with haemophilia B (FIX activity ≤2%)
    Sicurezza ed efficacia di nonacog beta pegol (N9-GP) in pazienti con Emofilia B precedentemente non trattati.

    Studio di fase 3a, multicentrico in aperto, a singolo braccio non controllato, su efficacia e sicurezza di nonacog beta pegol (N9-GP) in profilassi e nel trattamento degli episodi di sanguinamento in pazienti con Emofilia B precedentemente non trattati (attività del FIX ≤2%).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and efficacy of nonacog beta pegol (N9-GP) in Previously Untreated Patients with Haemophila B
    Sicurezza ed efficacia di nonacog beta pegol (N9-GP) in pazienti con Emofilia B precedentemente non trattati
    A.3.2Name or abbreviated title of the trial where available
    An open-label single-arm multicentre non-controlled phase 3a trial investigating safety and efficacy
    Sicurezza ed efficacia di nonacog beta pegol (N9-GP) in pazienti con Emofilia B precedentemente non
    A.4.1Sponsor's protocol code numberNN7999-3895
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/731/2009
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNOVO NORDISK. 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 supportNovo Nordisk A/S
    B.4.2CountryDenmark
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovo Nordisk A/S
    B.5.2Functional name of contact pointGlobal Clinical Registry
    B.5.3 Address:
    B.5.3.1Street AddressNovo Allé
    B.5.3.2Town/ cityBagsvaerd
    B.5.3.3Post code2880
    B.5.3.4CountryDenmark
    B.5.4Telephone number.
    B.5.5Fax number.
    B.5.6E-mailclinicaltrials@novonordisk.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/3/09/640
    D.3 Description of the IMP
    D.3.1Product nameN9-GP 2000IU /fiala
    D.3.4Pharmaceutical form Powder and solvent for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNONACOG BETA PEGOL
    D.3.9.2Current sponsor codeNNC0156-009
    D.3.9.4EV Substance CodeSUB33409
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 Yes
    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 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/3/09/640
    D.3 Description of the IMP
    D.3.1Product nameN9-GP 500IU/ fiala
    D.3.4Pharmaceutical form Powder and solvent for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNONACOG BETA PEGOL
    D.3.9.2Current sponsor codeNNC0156-009
    D.3.9.4EV Substance CodeSUB33409
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125
    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
    Haemophilia B
    Emofilia B
    E.1.1.1Medical condition in easily understood language
    Bleeding disorder, inherited deficiency in clotting factor IX
    Disordine ereditario della coagulazione
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10018939
    E.1.2Term Haemophilia B (Factor IX)
    E.1.2System Organ Class 100000004850
    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 immunogenicity of N9-GP
    Valutare l'immunogenicità di N9-GP
    E.2.2Secondary objectives of the trial
    - To evaluate safety of N9-GP (nonacog beta pegol)
    - To evaluate efficacy of N9-GP (nonacog beta pegol)
    o in long-term prophylaxis treatment
    o in the treatment of bleeding episodes
    o through the surrogate marker: FIX activity
    o through monitoring of number of doses and consumption of N9-GP
    1.Valutare la sicurezza di N9-GP
    2.Valutare l'efficacia di N9-GP:
    - nel trattamento di profilassi a lungo termine
    -nel trattamento degli episodi di sanguinamento
    -attraverso un marker surrogato: attività del FIX
    -attraverso il monitoraggio del numero delle dosi necessarie ed il consumo di N9-GP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Informed consent obtained before any trial-related activities. Trialrelated
    activities are any procedures that are carried out as part of the
    trial, including activities to determine suitability for the trial
    - Male, < 6 years of age at the time of signing informed consent
    - Patients with the diagnosis of haemophilia B (FIX activity level ≤ 2%)
    based on medical records or central laboratory results
    - Previously untreated or exposed to FIX containing products less than
    or equal to 3 exposure days (5 previous exposure days to blood
    components are acceptable)
    -Consenso Informato ottenuto prima di qualsiasi attività specifica dello studio
    -Soggetto maschio di età inferiore ai 6 anni di età al momento della firma del consenso informato
    -Diagnosi di Emofilia B (Livello di attività del FIX ≤ 2%)
    -Pazienti precedentemente non trattati o con un massimo di 3 giorni di esposizione a prodotti ricombinanti contenenti FIX o di 5 giorni per prodotti plasma derivati
    E.4Principal exclusion criteria
    - Any history of FIX inhibitors (defined by medical records)
    - Known or suspected hypersensitivity to trial product or related
    products
    - Previous participation in this trial. Participation is defined as first dose
    administered of trial product
    - Receipt of any investigational medicinal product within 30 days before
    screening
    - Congenital or acquired coagulation disorder other than haemophilia B
    - Any chronic disorder or severe disease which, in the opinion of the
    Investigator, might jeopardise patient's safety or compliance with the
    protocol
    - Patient's parent(s)/LAR(s) mental incapacity, unwillingness to
    cooperate, or a language barrier precluding adequate understanding and
    cooperation
    -Anamnesi di inibitori al FIX ( da cartella medica)
    -Sospetta o riconosciuta ipersensività al prodotto sperimentale o a prodotti correlati
    -Trattamento con altri prodotti sperimentali fino a 30 giorni precedenti allo screening
    -Altre patologie della coagulazione oltre l'Emofilia B congenite od acquisite
    -Qualsiasi disordine cronico o malattia grave che nell'opinione dello sperimentatore potrebbe mettere a rischio la sicurezza o la compliance del protocollo del paziente
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of inhibitory antibodies against FIX
    Incidenza di Anticorpi inibitori contro il Fattore IX
    E.5.1.1Timepoint(s) of evaluation of this end point
    All objectives/endpoints will be evaluated when the first 20 PUPs have
    reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs,
    and at end of trial. End of trial will be up to 4 years after the patient has
    reached 100 EDs.
    Tutti gli obiettivi/endpoints verranno valutati quando i primi 20 pazienti PUP raggiungeranno un minimo di 50 giorni di esposizione, quando i primi 40 pazienti PUP raggiungeranno 100 giorni di esposizione e al termine della sperimentazione.
    E.5.2Secondary end point(s)
    - Number and frequency of adverse events, serious adverse events, and
    Medical Events of Special interest
    - Number of breakthrough bleeding episodes during prophylaxis
    (annualised bleeding rate)
    - Haemostatic effect by 4-point haemostatic response scale ("excellent",
    "good", "moderate" and "poor")
    -Numero e frequenza degli eventi avversi, degli eventi avversi seri e MESI (Medical Event of Special Interest)
    -Numero degli episodi di sanguinamento durante trattamento in profilassi
    -Valutazione dell'effetto emostatico secondo una scala a 4 punti
    E.5.2.1Timepoint(s) of evaluation of this end point
    All objectives/endpoints will be evaluated when the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial. End of trial will be up to 4 years after the patient has reached 100 EDs.
    Tutti gli obiettivi/endpoints verranno valutati quando i primi 20 pazienti PUP raggiungeranno un minimo di 50 giorni di esposizione, quando i primi 40 pazienti PUP raggiungeranno 100 giorni di esposizione e al termine della sperimentazione.
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Immunogenicity
    Immunogenicità
    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 Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    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.8.4.1Number 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 EEA10
    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
    Canada
    France
    Germany
    Israel
    Italy
    Japan
    Malaysia
    Spain
    Thailand
    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
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days28
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days28
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 1
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 8
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 36
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    F.4.2.2In the whole clinical trial 50
    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)
    N/A
    N/A
    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 Decision2015-10-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-11-12
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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