Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43865   clinical trials with a EudraCT protocol, of which   7286   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2010-023072-17
    Sponsor's Protocol Code Number:NN7999-3775
    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:2012-03-07
    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 number2010-023072-17
    A.3Full title of the trial
    Safety and Efficacy of NNC-0156-0000-0009 after Long-Term Exposure in Patients with Haemophilia B
    Sicurezza ed efficacia di NNC 0156 0000 0009 dopo esposizione a lungo termine in pazienti con emofilia B
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy of NNC-0156-0000-0009 after Long-Term Exposure in Patients with Haemophilia B
    Sicurezza ed efficacia di NNC 0156 0000 0009 dopo esposizione a lungo termine in pazienti con emofilia B
    A.3.2Name or abbreviated title of the trial where available
    PARADIGM 4
    PARADIGM 4
    A.4.1Sponsor's protocol code numberNN7999-3775
    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
    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/3/09/640
    D.3 Description of the IMP
    D.3.1Product nameN9-GP
    D.3.2Product code NNC-0156-0000-0009
    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 INNNA
    D.3.9.1CAS number 117551271-6
    D.3.9.2Current sponsor codeNNC 0156-0000-009
    D.3.9.3Other descriptive nameNA
    D.3.9.4EV Substance CodeNA
    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
    D.3.2Product code NNC-0156-0000-009
    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 INNNA
    D.3.9.1CAS number 117551271-6
    D.3.9.2Current sponsor codeNNC 0156-0000-009
    D.3.9.3Other descriptive nameNA
    D.3.9.4EV Substance CodeNA
    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, caused by mutations in the coagulation factor nine (FIX) gene, located in the distal part on the long arm of the X-chromosome. Haemophilia B is caused by any of a variety of genetic anomalies distributed throughout the gene, with almost every family.
    L’emofilia B è una malattia emorragica congenita recessiva legata al cromosoma X, causata da mutazioni nel gene del fattore di coagulazione IX (FIX), situato nella parte distale sul braccio lungo del cromosoma X. L’emofilia B è causata da una serie di anomalie genetiche distribuite per tutto il gene, di cui quasi ogni famiglia manifesta la propria configurazione unica.
    E.1.1.1Medical condition in easily understood language
    Haemophilia B is a recessive X-linked congenital bleeding disorder, caused by mutations in the coagulation factor nine (FIX) gene.
    L’emofilia B è una malattia emorragica congenita recessiva legata al cromosoma X, causata da mutazioni nel gene del fattore di coagulazione IX (FIX)
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    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 the immunogenicity of NNC-0156-0000-0009
    Valutare l’immunogenicità di NNC-0156-0000-0009
    E.2.2Secondary objectives of the trial
    • To evaluate clinical efficacy of haemostasis (treatment of bleeding episodes) of N9-GP • To evaluate clinical efficacy of N9-GP in long term bleeding prophylaxis (number of bleeding episodes during prophylaxis) • To evaluate the efficacy of N9-GP by the surrogate marker for efficacy, FIX activity • To evaluate general safety of N9-GP • To evaluate Patient Reported Outcomes (PRO), including health-related and disease-specific quality of life and patient treatment satisfaction • To evaluate the health economic impact of N9-GP treatment
    •Valutare l’efficacia clinica dell’emostasi (trattamento degli episodi di sanguinamento) di N9-GP
    •Valutare l’efficacia clinica di N9-GP nella profilassi a lungo termine dei sanguinamenti (numero di episodi di sanguinamento durante la profilassi)
    •Valutare l’efficacia di N9-GP tramite il marker surrogato per l’efficacia, l’attività di FIX
    •Valutare la sicurezza generale di N9-GP
    •Valutare gli esiti riportati dal paziente (PRO), incluse la qualità della vita in relazione alla salute, quella collegata allo stato specifico della malattia e la soddisfazione del paziente per il trattamento
    •Valutare l’impatto farmaco-economico per il trattamento con N9-GP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Informed consent obtained before any trial-related activities. Trial-related activities are any procedure that would not have been performed during normal management of the patient 2. Previous participation in NN7999-3747 and/or NN7999-3773 3. The patient and/or LAR is capable of assessing a bleeding episode, keeping a diary, capable of home treatment of bleeding episodes and otherwise capable of following the trial procedures
    1.Il consenso informato ottenuto prima dell’inizio di qualsiasi attività connessa allo studio. Le attività connesse allo studio sono tutte quelle procedure che non sarebbero state effettuate durante la normale gestione clinica del soggetto
    2.Precedente partecipazione agli studi NN7999-3747 e/o al NN7999-3773
    3.Il paziente e/o LAR sono in grado di valutare un episodio di sanguinamento, tenere un diario, eseguire il trattamento a casa degli episodi di sanguinamento e altrimenti in grado di seguire le procedure dello studio
    E.4Principal exclusion criteria
    1. Known or suspected hypersensitivity to the trial product or related products 2. Dosing of any investigational drug within the last 30 days prior to the present trial (excluding N9-GP) 3. Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR interviews 4. Current FIX inhibitors ≥0.6 BU (central laboratory) 5. Congenital or acquired coagulation disorders other than haemophilia B 6. Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records) 7. Any disease (liver, kidney, inflammatory and mental disorders included) or condition which, according to the Investigator’s judgement, could imply a potential hazard to the patient, interfere with trial participation, or interfere with trial outcome
    1.Ipersensibilità nota o sospetta al prodotto sperimentale o ad altri prodotti ad esso correlati
    2.Somministrazione di qualsiasi prodotto sperimentale entro gli ultimi 30 giorni prima dello studio presente (escluso N9-GP)
    3.Storia nota d’inibitori contro il FIX basata sulle cartelle cliniche esistenti, sugli esami di laboratorio e sui colloqui con il paziente e/o con il rappresentante legale
    4.Inibitori di FIX presenti ≥0,6 BU (laboratorio centrale)
    5.Disordini della coagulazione congeniti o acquisiti diversi dall’emofilia B
    6.Eventi trombotici arteriosi precedenti (p.es. infarto del miocardio e trombosi intracranica) o precedente trombosi venosa profonda o embolia polmonare (come definiti dalle cartelle cliniche disponibili)
    7. Qualsiasi disturbo (inclusi i disturbi a livello epatico, renale, o anche disturbi infiammatori e mentali) o condizione che, a giudizio dello Sperimentatore, potrebbero implicare un potenziale rischio per il paziente, interferire con la partecipazione allo studio o con il risultato dello studio
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of inhibitory antibodies against FIX defined as titre ≥0.6 BU
    Incidenza di anticorpi inibitori contro il FIX definita come titolo ≥0,6 BU.
    E.5.1.1Timepoint(s) of evaluation of this end point
    End of trial
    Conclusione dello studio
    E.5.2Secondary end point(s)
    Secondary Efficacy Endpoints • Haemostatic effect of N9-GP when used for treatment of bleeding episodes, assessed on a fourpoint scale for haemostatic response (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure • Number of bleeding episodes per patient during routine prophylaxis • FIX trough levels • The number of injections of N9-GP required per bleeding episode • Amount of N9-GP required per bleeding episode (U/kg BW/bleeding episodes Secondary Safety Endpoints • Adverse Events (AEs) and Serious Adverse Events (SAEs) • Host Cell Proteins (HCP)-antibodies • General safety endpoints including laboratory parameters, physical examination and vital signs
    Gli endpoint secondari dell’efficacia Effetto emostatico di N9-GP usato per il trattamento di episodi di sanguinamento, valutato secondo una scala di quattro punti (eccellente, buono, moderato e scarso) per il responso emostatico, dove eccellente e buono valgono come successo e moderato e scarso come fallimento
    • Numero di episodi di sanguinamento per paziente durante la profilassi di routine
    • Livelli minimi di FIX
    • Il numero d’iniezioni di N9-GP richieste per episodio di sanguinamento
    • Quantità di N9-GP richiesta per episodio di sanguinamento (U/kg BW/episodio di sanguinamento)
    Gli endpoint secondari sulla sicurezza
    • Eventi avversi (AE) ed eventi avversi gravi (SAE)
    • Anticorpi delle proteine della cellula ospite (HCP)
    • Endpoint generali sulla sicurezza inclusi i parametri di laboratorio, l’esame fisico e i segni vitali
    E.5.2.1Timepoint(s) of evaluation of this end point
    End of trial
    Conclusione dello studio
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    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 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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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 trial3
    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 Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA17
    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
    Canada
    Japan
    Macedonia, the former Yugoslav Republic of
    Malaysia
    Russian Federation
    South Africa
    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 years0
    E.8.9.1In the Member State concerned months55
    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 months55
    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: 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) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 21
    F.4.2.2In the whole clinical trial 75
    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)
    The patients involved in the trial NN7999-3775 will be treated with the trial product until it becomes commercially available.
    I pazienti partecipanti alla sperimentazione NN7999-3775 saranno trattati fino alla eventuale disponibilità commerciale del prodotto sperimentale.
    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 Decision2011-11-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-07-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-03-31
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Sat Apr 27 01:08:46 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA