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    Summary
    EudraCT Number:2020-000504-11
    Sponsor's Protocol Code Number:NN7415-4616
    National Competent Authority:Estonia - SAM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-08-10
    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 ConcernedEstonia - SAM
    A.2EudraCT number2020-000504-11
    A.3Full title of the trial
    Open-label study investigating efficacy, safety and pharmacokinetics of concizumab prophylaxis in children below 12 years with haemophilia A or B with or without inhibitors
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study on how well concizumab works for you if you have haemophilia A or B with or without inhibitors (explorer10)
    A.4.1Sponsor's protocol code numberNN7415-4616
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1247-7330
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/371/2021
    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 pointClinical Transparency (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.5.6E-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 numberEMA/OD/095/12, EMA/OD/116/17
    D.3 Description of the IMP
    D.3.1Product nameConcizumab C 10 mg/mL PDS290
    D.3.4Pharmaceutical form Solution for injection in pre-filled pen
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNConcizumab
    D.3.9.3Other descriptive nameConcizumab
    D.3.9.4EV Substance CodeSUB192373
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 Yes
    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 numberEMA/OD/095/12, EMA/OD/116/17
    D.3 Description of the IMP
    D.3.1Product nameConcizumab C 40 mg/mL PDS290
    D.3.4Pharmaceutical form Solution for injection in pre-filled pen
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNConcizumab
    D.3.9.3Other descriptive nameConcizumab
    D.3.9.4EV Substance CodeSUB192373
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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 Yes
    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: 3
    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 numberEMA/OD/095/12, EMA/OD/116/17
    D.3 Description of the IMP
    D.3.1Product nameConcizumab C 100 mg/mL PDS290
    D.3.4Pharmaceutical form Solution for injection in pre-filled pen
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNConcizumab
    D.3.9.3Other descriptive nameConcizumab
    D.3.9.4EV Substance CodeSUB192373
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 Yes
    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 with or without inhibitors

    Haemophilia B with our without inhibitors
    E.1.1.1Medical condition in easily understood language
    Bleeding disorder, inherited deficiency in clotting factor VIII or IX with or without antibodies to replacement therapy
    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 10053752
    E.1.2Term Hemophilia B with anti factor IX
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10053751
    E.1.2Term Hemophilia A with anti factor VIII
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10053753
    E.1.2Term Hemophilia A without inhibitors
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10053754
    E.1.2Term Hemophilia B without inhibitors
    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 establish superiority of concizumab prophylaxis in concizumab-naïve children less than 12 years with haemophilia A or B with inhibitors compared to their previous on demand treatment on the number of treated spontaneous and traumatic bleeding episodes.
    E.2.2Secondary objectives of the trial
    - To establish non-inferiority of concizumab prophylaxis in concizumab-naïve children less than 12 years with haemophilia A or B without inhibitors compared to their previous PPX treatment on the number of treated spontaneous and traumatic bleeding episodes

    - To evaluate the safety of concizumab administered once-daily in concizumab-naïve children less than 12 years with haemophilia A or B with or without inhibitors

    - To evaluate the safety of concizumab administered once-daily in patients previously treated with concizumab via compassionate use

    - To evaluate pharmacokinetics and pharmacodynamics of concizumab administered once-daily in concizumab-naïve children less than 12 years with haemophilia A or B with or without inhibitors
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Informed consent/assent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study.

    - Diagnosis of congenital severe haemophilia A (FVIII less than 1%) or moderate/severe congenital haemophilia B (FIX equal to or less than 2%), or congenital haemophilia with inhibitors.

    - For arm 1 only: Male aged less than 12 years of age at the time of signing informed consent.

    - For arm 1 only: Patients with medical records of a total of at least 26 weeks of treatment within the last 52 weeks prior to enrolment(a)
    * Patients with HAwI with medical records of a total of at least 26 weeks of on demand treatment within the last 52 weeks prior to enrolment.
    * Patients with HBwI with medical records of a total of at least 26 weeks of on demand treatment within the last 52 weeks prior to enrolment
    * Patients with HBwI regardless of the regimen and duration of previous haemophilia treatment
    * Patients without inhibitors with medical records of a total of at least 26 weeks of PPX treatment within the last 52 weeks prior to enrolment

    - For arm 2 only: Male patients (regardless of age) previously treated with concizumab via compassionate use.

    (a) For patients that have been diagnosed with haemophilia less than 1 year prior to enrolment, historical medical records from time of diagnosis will suffice as long as medical records of a total of at least 26 weeks of relevant treatment is available
    E.4Principal exclusion criteria
    - Known or suspected hypersensitivity to study intervention or related products.
    - Known inherited or acquired coagulation disorder other than congenital haemophilia.
    - Ongoing or planned Immune Tolerance Induction treatment.
    - History of thromboembolic disease(a). Current clinical signs of or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events(b)

    (a) Includes arterial and venous thrombosis including myocardial infarction, pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion.
    (b) Thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events.
    E.5 End points
    E.5.1Primary end point(s)
    For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment: The number of treated spontaneous and traumatic bleeding episodes
    E.5.1.1Timepoint(s) of evaluation of this end point
    From start of treatment (week 0) up until the analysis cut-off (a)

    (a) The analysis cut-off is defined as the point in time when all inhibitor patients in arm 1 have completed the week 32 visit (or withdrawn) and all non-inhibitor patients in arm 1 have completed the week 40 visit (or withdrawn). The individual patient’s cut-off will be the last completed visit before the cut-off date.
    E.5.2Secondary end point(s)
    1. For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment: The number of all bleeding episodes (spontaneous and traumatic)
    2. For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment: Number of treated spontaneous bleeding episodes
    3. For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment: Number of treated joint bleeding episodes
    4. For inhibitor patients with at least 26 weeks on demand treatment during the last year before enrolment: Number of treated bleeding episodes in baseline target joints
    5. For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment: Number of treated spontaneous and traumatic bleeding episodes
    6. For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment: The number of all bleeding episodes (spontaneous and traumatic)

    7. For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment: Number of treated spontaneous bleeding episodes
    8. For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment: Number of treated joint bleeding episodes
    9. For non-inhibitor patients with at least 26 weeks PPX treatment during the last year before enrolment: Number of treated bleeding episodes in baseline target joints
    10. Number of treatment emergent adverse events
    11. Number of thromboembolic events
    12. Number of hypersensitivity type reactions
    13. Number of injection site reactions
    14. Number of patients who develop antibodies to concizumab – yes/no
    15. Number of treatment emergent adverse events
    16. Concizumab plasma concentrations prior to dosing
    17. Peak thrombin generation prior to dosing
    18. Free TFPI concentration prior to dosing
    19. Pre-dose (trough) concizumab plasma concentration (Ctrough)
    20. Maximum concizumab plasma concentration (Cmax)
    21. Area under the concizumab plasma concentration-time curve (AUC)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.-4 From start of treatment (week 0) up until the analysis cut-off(a)
    5.-9 From the point in time when the concizumab maintenance dose is confirmed, decreased or increased and up until the analysis cut-off(a)
    10.-15 From start of treatment (week 0) up until the analysis cut-off(a)
    16.-18 Week 32
    19. Prior to the concizumab administration at week 20
    20.-21 From 0 to 24 hours where 0 is the time of the concizumab dose at week 20

    (a) The analysis cut-off is defined as the point in time when all inhibitor patients in arm 1 have completed the week 32 visit (or withdrawn) and all non-inhibitor patients in arm 1 have completed the week 40 visit (or withdrawn). The individual patient’s cut-off will be the last completed visit before the cut-off date.
    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 Yes
    E.7.1.3.1Other trial type description
    PK/PD
    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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA23
    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
    India
    Japan
    Thailand
    United States
    European Union
    Bosnia and Herzegovina
    North Macedonia
    Norway
    Russian Federation
    Turkey
    Ukraine
    United Kingdom
    E.8.7Trial has a data monitoring committee Yes
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days14
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial days27
    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: 95
    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: 3
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 90
    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) Yes
    F.1.2.1Number of subjects for this age range: 1
    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 32
    F.4.2.2In the whole clinical trial 96
    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)
    None
    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 Decision2022-09-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-10-03
    P. End of Trial
    P.End of Trial StatusOngoing
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