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    Summary
    EudraCT Number:2011-000826-31
    Sponsor's Protocol Code Number:NN7999-3774
    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:2012-10-09
    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 number2011-000826-31
    A.3Full title of the trial
    Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children with Haemophilia B
    Sicurezza, Efficacia e Farmacocinetica di NNC-0156-0000-0009 in bambini con emofilia B precedentemente trattati
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 (N9-GP) in Previously Treated Children with Haemophilia B
    Sicurezza, Efficacia e Farmacocinetica di NNC-0156-0000-0009 (N9-GP) in bambini con emofilia B precedentemente trattati
    A.3.2Name or abbreviated title of the trial where available
    paradigm™5
    paradigm™5
    A.4.1Sponsor's protocol code numberNN7999-3774
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1119-5013
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/292/2010
    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 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 (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 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 2000U/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.3.8INN - Proposed INNnonacog beta pegol
    D.3.9.1CAS number 117551271-6
    D.3.10 Strength
    D.3.10.1Concentration unit U/ml 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 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/3/09/640
    D.3 Description of the IMP
    D.3.1Product nameN9-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.3.8INN - Proposed INNnonacog beta pegol
    D.3.9.1CAS number 117551271-6
    D.3.10 Strength
    D.3.10.1Concentration unit U/ml 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 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 B is a recessive X-linked congenital bleeding disorder characterised by increased bleeding tendency due to either a partial or complete deficiency or dysfunction of the essential blood coagulation factor IX. It is caused by mutations in the F9 gene, located in the distal part on the long arm of the X-chromosome.
    L’emofilia B è una malattia emorragica congenita recessiva legata al cromosoma X, caratterizzata da un’aumentata tendenza al sanguinamento dovuta sia a una completa o parziale deficienza del fattore di coagulazione IX (FIX) sia a una sua alterazione. E’ causata da mutazioni nel gene del FIX, situato nella parte distale del braccio lungo del cromosoma X.
    E.1.1.1Medical condition in easily understood language
    Haemophilia B is a congenital bleeding disorder caused by mutations in the coagulation Factor IX (F9) gene.
    L’emofilia B è una malattia emoraggica congenita, causata da mutazioni nel gene del fattore di coagulazione IX (FIX).
    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 15.0
    E.1.2Level LLT
    E.1.2Classification code 10018939
    E.1.2Term Haemophilia B (Factor IX)
    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 immunogenicity of glycopegylated recombinant coagulation factor IX (NNC-0156-0000-0009; hereafter referred to as N9-GP).
    Valutare l'immunogenicità del fattore IX di coagulazione ricombinante glicopeghilato (NNC-0156-0000-0009; da qui in poi definito come N9-GP).
    E.2.2Secondary objectives of the trial
    - Valutare la sicurezza oltre all’immunogenicità di N9-GP - Valutare l’efficacia di N9-GP nella profilassi a lungo termine e nel trattamento degli episodi di sanguinamento - Valutare l’efficacia di N9-GP mediante il controllo dell’attività del FIX, marker surrogato per l’efficacia - Valutare le proprietà farmacocinetiche (PK) di N9-GP
    - To evaluate safety other than immunogenicity of N9-GP - To evaluate the efficacy of N9-GP in long-term prophylaxis and in the treatment of breakthrough bleeding episodes - To evaluate the efficacy of N9-GP through the surrogate marker for efficacy, FIX activity - To evaluate the pharmacokinetic (PK) properties of N9-GP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Male patients with moderately severe or severe congenital haemophilia B with a FIX activity level ≤2% according to medical records - Age ≤ 12 years (until patient turns 13 years, at time of inclusion) - Body weight ≥10 kg - History of at least 50 EDs to other FIX products - The patient and/or parent(s)/caregiver are capable of assessing a bleeding episode, keeping an eDiary, capable of conducting home treatment and otherwise able to follow trial procedures
    - Pazienti maschi con emofilia B congenita moderatamente grave o grave con un livello di attività di FIX ≤ 2% documentata in cartella clinica - Età ≤ 12 anni (prima del compimento dei 13 anni al momento dell’inclusione) - Peso ≥10 kg - Storia di almeno 50 ED con altri prodotti FIX - Il paziente e/o il/i genitore/i o chi si prende cura del paziente siano in grado di valutare un episodio di sanguinamento, gestire un diario elettronico, effettuare trattamenti a domicilio ed essere in grado di seguire le procedure dello studio
    E.4Principal exclusion criteria
    - Known history of FIX inhibitors - Current FIX inhibitors ≥0.6 BU (central laboratory) - Congenital or acquired coagulation disorder other than haemophilia B - Platelet count <50,000/µL at screening - Alanine aminotransferase (ALT) >3 times the upper limit of normal reference ranges at screening - Creatinine level ≥1.5 times above the upper normal limit of normal reference ranges at screening - Human immunodeficiency virus (HIV) positive, defined by medical records, and with a CD4+ lymphocyte count ≤200/µL - Immune modulating or chemotherapeutic medication (except single pulse treatment, inhaled and topical steroids) - Previous arterial thrombotic events (myocardial infarction and intracranial thrombosis, as defined by medical records)
    - Storia nota di inibitori contro il Fattore IX - Inibitori contro il FIX ≥0,6 BU (dato del laboratorio centrale) - Disturbi della coagulazione congeniti o acquisiti diversi dall’emofilia B - Conta piastrinica &lt;50.000/µL allo screening - Valori di ALT 3 volte maggiore rispetto al limite superiore degli intervalli normali di riferimento allo screening - Valori di creatinina 1,5 volte maggiori rispetto al limite superiore normale degli intervalli normali di riferimento allo screening - Positività all’HIV, definita dalle cartelle cliniche, e con una conta di linfociti CD4+ ≤200/µL
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of inhibitory antibodies against coagulation factor IX (FIX) defined as titre ≥0.6 BU
    Incidenza degli anticorpi inibitori contro il Fattore IX della coagulazione definito come ≥0,6 BU
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 52
    settimana 52
    E.5.2Secondary end point(s)
    1. Number of bleeding episodes during prophylaxis 2. Haemostatic effect of N9-GP in treatment of bleeding episodes by 4-point categorical scale for haemostatic response (excellent, good, moderate and poor) 3. Incremental recovery at 30 minutes (IR30min) 4. Trough level single-dose 5. Trough level steady state 6. Terminal half-life (t1/2)
    1. Numero di episodi di sanguinamento durante la profilassi 2. Effetto emostatico di N9-GP nel trattamento di episodi di sanguinamento su una scala a 4 punti per la valutazione della risposta emostatica (eccellente, buono, moderato, scarso) 3. Recovery incrementale a 30 minuti (IR30min) 4. Livello basale dose singola 5. Livello basale steady state 5. Emivita terminale (t½)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.Week 52 2.Week 52 3.Week 0 4.Week 0 5.Week 4-44 6.Week 0
    1.Settimana 52 2.Settimana 52 3.Settimana 0 4.Settimana 0 5.Settimane dalla 4 alla 44 6.Settimana 0
    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 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) 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 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 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
    Brazil
    Canada
    Croatia
    Israel
    Japan
    Malaysia
    Taiwan
    Thailand
    Turkey
    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 months39
    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 months45
    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: 24
    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: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    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 10
    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)
    Not Applicable
    Non Applicabile
    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 Decision2012-09-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-09-18
    P. End of Trial
    P.End of Trial StatusCompleted
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