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    Summary
    EudraCT Number:2012-004867-38
    Sponsor's Protocol Code Number:NN7999-3895
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2015-07-10
    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 ConcernedSpain - AEMPS
    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 Haemophilia 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%)
    Seguridad y eficacia de nonacog beta pegol (N9-GP) en pacientes con hemofilia B no tratados previamente

    Ensayo de fase 3a, no controlado, multicéntrico, de un solo grupo, abiertosin enmascaramiento, para investigar la seguridad y la eficacia de nonacog beta pegol (N9-GP) en la profilaxis y el tratamiento de los episodios hemorrágicos en pacientes con hemofilia B (actividad de FIX ? 2 %) no tratados previamente
    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 Haemophilia B
    Seguridad y eficacia de nonacog beta pegol (N9-GP) en pacientes con hemofilia B no tratados previamente
    A.3.2Name or abbreviated title of the trial where available
    paradigm?6
    A.4.1Sponsor's protocol code numberNN7999-3895
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1135-9557
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/329/2014
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNovo Nordisk A/S
    B.1.3.4CountryDenmark
    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 AddressNovo Allé
    B.5.3.2Town/ cityBagsværd
    B.5.3.3Post code2880
    B.5.3.4CountryDenmark
    B.5.4Telephone number+34913 349 800
    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/vial
    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.3.9.2Current 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 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 500IU/vial
    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.3.9.2Current 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 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
    Hemofilia B
    E.1.1.1Medical condition in easily understood language
    Bleeding disorder, inherited deficiency in clotting factor IX
    Desorden del sangrado, deficiencia hereditaria en el factor IX de couagulación
    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 18.0
    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 (nonacog beta pegol)
    Evaluar la inmunogeneicidad de N9-GP (nonacog beta pegol)
    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
    ?Evaluar la seguridad de N9-GP (nonacog beta pegol)
    ?Evaluar la eficacia de N9-GP (nonacog beta pegol)
    o en el tratamiento profiláctico a largo plazo
    o en el tratamiento de los episodios hemorrágicos
    o por medio del marcador indirecto: actividad del FIX
    o por medio del seguimiento del número de dosis y del consumo de N9-GP
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Informed consent obtained before any trial-related activities. Trial-related 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)
    -Obtención del consentimiento informado antes de realizar cualquier actividad relacionada con el ensayo. Se consideran actividades relacionadas con el ensayo todos los procedimientos que se lleven a cabo como parte del ensayo, incluidas las actividades para determinar la idoneidad para el mismo.
    -Pacientes varones < 6 años de edad en el momento de la firma del consentimiento informado.
    -Pacientes con un diagnóstico de hemofilia B (nivel de actividad del FIX ? 2 %) basado en la historia clínica o los resultados del laboratorio central.
    -Pacientes no tratados previamente o con una exposición a productos con FIX inferior o igual a 3 días de exposición (se aceptan 5 días de exposición previas a hemoderivados).
    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
    ?Antecedentes de uso de inhibidores del FIX (según la historia clínica).
    ?Hipersensibilidad conocida o presunta al producto del ensayo o productos relacionados.
    ?Participación previa en este ensayo. La participación se define como la administración de la primera dosis del producto del ensayo.
    ?Recepción de cualquier producto en investigación en los 30 días previos a la selección
    ?Trastornos de la coagulación congénitos o adquiridos distintos de la hemofilia B.
    ?Cualquier trastorno crónico o enfermedad importante que, en opinión del investigador, pueda poner en peligro la seguridad del paciente o el cumplimiento del protocolo.
    ?Incapacidad mental, falta de cooperación o barrera idiomática que impida una correcta comprensión y colaboración de los padres o el tutor legal del paciente.
    E.5 End points
    E.5.1Primary end point(s)
    Incidence of inhibitory antibodies against FIX
    Incidencia de anticuerpos inhibidores contra el FIX
    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.
    Todos los objetivos/criterios de valoración se evaluarán cuando los primeros 20 PNTP PSTP hayan alcanzado al menos 50 DE, cuando los primeros 40 PNTP PSTP hayan alcanzado 100 DE y al final del ensayo. El final del ensayo tendrá lugar 4 años como máximo después de que el paciente haya alcanzado 100 DE
    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")
    -Número y frecuencia de acontecimientos adversos, acontecimientos adversos graves y acontecimientos médicos graves de interés especial.
    -Número de episodios hemorrágicos intercurrentes durante la profilaxis (tasa anualizada de hemorragias).
    -Efecto hemostático mediante la escala de respuesta de 4 puntos ("excelente", "buena", "leve" o "pobre")
    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.
    Todos los objetivos/criterios de valoración se evaluarán cuando los primeros 20 PNTP PSTP hayan alcanzado al menos 50 DE, cuando los primeros 40 PNTP PSTP hayan alcanzado 100 DE y al final del ensayo. El final del ensayo tendrá lugar 4 años como máximo después de que el paciente haya alcanzado 100 DE
    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
    Inmunogeneicidad
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 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
    Australia
    Canada
    European Union
    Israel
    Japan
    Malaysia
    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
    UVUP
    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: 50
    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 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 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
    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 Decision2015-07-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-06-18
    P. End of Trial
    P.End of Trial StatusOngoing
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