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    Summary
    EudraCT Number:2012-001711-23
    Sponsor's Protocol Code Number:NN7088-3885
    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:2012-12-13
    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 number2012-001711-23
    A.3Full title of the trial
    A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients with Severe Haemophilia A
    Studio clinico multinazionale, in aperto, non controllato sulla sicurezza, l'efficacia e la farmacocinetica di NNC 0129-0000-1003 in pazienti pediatrici con emofilia A grave precedentemente trattati
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients with Severe Haemophilia A
    Studio clinico multinazionale, in aperto, non controllato sulla sicurezza, l’efficacia e la farmacocinetica di NNC 0129-0000-1003 in pazienti pediatrici con emofilia A grave precedentemente trattati
    A.4.1Sponsor's protocol code numberNN7088-3885
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1129-6009
    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 SponsorNOVO NORDISK
    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 S.p.A.
    B.4.2CountryItaly
    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 (GCR,1452)
    B.5.3 Address:
    B.5.3.1Street AddressVandtaarnsvej 114,VTB
    B.5.3.2Town/ citySoeborg
    B.5.3.3Post codeDK-2860
    B.5.3.4CountryDenmark
    B.5.4Telephone numberN/A
    B.5.5Fax numberN/A
    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/3/12/995
    D.3 Description of the IMP
    D.3.1Product nameN8-GP rFVIII
    D.3.2Product code NA
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1309086-46-1
    D.3.9.2Current sponsor codeNNC129-1003
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2000
    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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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.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/3/12/995
    D.3 Description of the IMP
    D.3.1Product nameN8-GP 500U vial
    D.3.2Product code NA
    D.3.4Pharmaceutical form Powder and solvent for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 1309086-46-1
    D.3.9.2Current sponsor codeNNC129-1003
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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 A
    Emofilia A
    E.1.1.1Medical condition in easily understood language
    Bleeding disorder, inherited deficiency in clotting factor VIII
    Disordine della coagulazione di tipo ereditario a carico del fattore VIII
    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 14.1
    E.1.2Level LLT
    E.1.2Classification code 10018938
    E.1.2Term Haemophilia A (Factor VIII)
    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 NNC 0129-0000-1003 (hereafter referred
    to as N8-GP)
    Valutare l'immunogenicità di NNC 0129-0000-1003 (d'ora in poi chiamato N8-GP)
    E.2.2Secondary objectives of the trial
    To evaluate safety other than immunogenicity of N8-GP
    - To evaluate efficacy of N8-GP in prophylaxis and treatment of bleeding
    episodes
    - To evaluate pharmacokinetic properties of N8-GP and compare to
    previous FVIII product
    • Valutare la sicurezza oltre che l’immunogenicità di N8-GP

    • Valutare l’efficacia di N8-GP nella profilassi e nel trattamento degli episodi emorragici

    • Valutare le proprietà farmacocinetiche di N8-GP e confrontarle al precedente prodotto a base di FVIII (solo valutazioni PK)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male patients with severe congenital haemophilia A (FVIII activity level
    < 1%)
    - Age below 12 years
    - Weight ≥10 kg
    - Documented history of > 150 ED to FVIII products for patients aged 6-
    11 years and > 50 ED to FVIII products for patients aged 0-5 years
    • Pazienti di sesso maschile affetti da emofilia A congenita (livello di attività del FVIII &lt; 1%)
    • Età inferiore ai 12 anni
    • Peso &gt; 10 kg
    • Anamnesi documentata di &gt; 150 ED a prodotti a base di FVIII per i pazienti di età compresa tra 6 ed 11 anni e &gt; 50 ED a prodotti a base di FVIII per i pazienti di età compresa tra 0 e 5 anni
    E.4Principal exclusion criteria
    Anamnesi di sviluppo di inibitori del FVIII
    Any history of FVIII inhibitors
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of inhibitory antibodies against coagulation factor VIII (FVIII)
    ≥0.6 Bethesda units
    • Incidenza degli anticorpi inibitori anti-FVIII ≥ 0,6 unità Bethesda (BU)
    E.5.1.1Timepoint(s) of evaluation of this end point
    During the main phase of the trial (from 0-26 weeks of treatment)
    durante la fase principale dello studio (da 0 a 26 settimane di trattamento)
    E.5.2Secondary end point(s)
    1. Frequency of adverse events including serious adverse events
    reported during the trial period
    2. Haemostatic effect of N8-GP when used for treatment of bleeding
    episodes and assessed as: Excellent, Good, Moderate, or None
    3. Number of bleeding episodes during prophylactic treatment with N8-
    GP (annualised bleeding rate)
    4. Consumption of N8-GP per bleeding episode (number of injections and
    U/kg)
    5. Consumption of N8-GP during prophylaxis (number of injections and
    U/kg per month and year)
    6. Incremental recovery (defined as the peak level recorded 30 min after
    end of injection) evaluated for previous FVIII product and N8-GP
    7. Area under the curve evaluated for previous FVIII product and N8-GP
    8. Terminal half-life evaluated for previous FVIII product and N8-GP
    9. Clearance evaluated for previous FVIII product and N8-GP
    1.Frequenza degli eventi avversi inclusi gli eventi avversi gravi seri segnalati durante il periodo dello studio
    2. Effetto emostatico di N8-GP quando utilizzato per il trattamento di episodi emorragici e valutato come: Eccellente, Buono, Moderato o Nessuno
    3. Numero degli episodi emorragici durante il trattamento profilattico con N8-GP (tasso annuale di sanguinamento)
    4. Consumo di N8-GP per episodio emorragico (numero di iniezioni e U/kg)
    5. Consumo di N8-GP durante la profilassi (numero di iniezioni e U/kg per mese e per anno)
    6. recupero incrementale (definito come il livello massimo analizzato 30 minuti dopo l'iniezione) valutato sia per il precedente prodotto a base di fattore VIII che per N8-GP
    7. Area sotto la curva (AUC) valutata sia per il precedente prodotto a base di fattore VIII che per N8-GP
    8. Emivita terminale valutata sia per il precedente prodotto a base di fattore VIII che per N8-GP
    9. clearance valutata sia per il precedente prodotto a base di fattore VIII che per N8-GP
    E.5.2.1Timepoint(s) of evaluation of this end point
    The pharmacokinetic endpoints on previous FVIII product will be based
    on assessments performed 2-6 weeks prior to initial dosing with N8-GP
    and up to 30 hours after administration of previous FVIII product.
    The pharmacokinetic endpoints on N8-GP will be based on assessments
    performed from 1 hour prior to and up to 96 hours after initial
    administration of N8-GP.
    All secondary safety and efficacy endpoints will be analysed and
    reported separately for the main phase (from 0-26 weeks of treatment)
    and the extension phase of the trial (from 26 weeks to the last patient
    has completed the trial).
    Gli endpoint di farmacocinetica sul precedente prodotto a base di fattore VIII saranno basati su esami effettuati 2-6 settimane prima del dosaggio iniziale con N8-GP e fino a 30 ore dopo la somministrazione del precedente prodotto a base di fattore VIII.
    Gli endpoint di farmacocinetica su N8-GP saranno basati su esami effettuati da 1 ora prima della somministrazione di N8-GP fino a 96 ore dalla somministrazione di N8-GP.
    Tutti gli endpoint secondari di safety ed efficacia saranno analizzati e riportati separatamente per la fase principale (da 0 a 26 settimane di trattamento) e la fase di estensione (dalla settimana 26 fino al completamento del trial da parte dell'ultimo paziente).
    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 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 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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 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 EEA45
    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 No
    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
    Brazil
    Canada
    European Union
    Israel
    Japan
    Korea, Republic of
    Macedonia, the former Yugoslav Republic of
    Malaysia
    Switzerland
    Turkey
    Ukraine
    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 years0
    E.8.9.1In the Member State concerned months63
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months63
    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.1.1Number of subjects for this age range: 60
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 59
    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 No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 60
    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 Decision2013-01-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-12-10
    P. End of Trial
    P.End of Trial StatusOngoing
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