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    Summary
    EudraCT Number:2021-004947-25
    Sponsor's Protocol Code Number:NN7999-4670
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2023-07-12
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2021-004947-25
    A.3Full title of the trial
    A multi-centre, open-label trial evaluating efficacy, safety and pharmacokinetics of nonacog beta pegol when used for treatment and prophylaxis of bleeding episodes in Chinese patients with haemophilia B
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study investigating nonacog beta pegol (N9-GP) for treatment and prevention of bleedings in Chinese people with haemophilia B
    A.3.2Name or abbreviated title of the trial where available
    paradigm9
    A.4.1Sponsor's protocol code numberNN7999-4670
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1260-0438
    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 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 (2834)
    B.5.3 Address:
    B.5.3.1Street AddressNovo Alle 1
    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 Yes
    D.2.1.1.1Trade name Refixia 2 000 IU powder and solvent for solution for injection
    D.2.1.1.2Name of the Marketing Authorisation holderNovo Nordisk A/S
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.4Pharmaceutical 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.3.9.3Other descriptive nameNonacog beta pegol
    D.3.9.4EV Substance CodeSUB180810
    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.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
    E.1.1.1Medical condition in easily understood language
    Bleeding disorder, inherited deficiency in clotting factor IX
    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 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 the clinical efficacy of nonacog beta pegol in haemostasis (treatment of bleeding episodes during on-demand and prophylaxis [PPX]) in Chinese patients aged 12-70 years with moderate to severe haemophilia B
    E.2.2Secondary objectives of the trial
    In Chinese patients aged 12-70 years with moderate to severe haemophilia B:
    • To evaluate the clinical efficacy of nonacog beta pegol in PPX treatment (number of treated bleeding episodes during PPX)
    • To evaluate the consumption of nonacog beta pegol
    • To evaluate the immunogenicity of nonacog beta pegol
    • To evaluate the general safety of nonacog beta pegol
    • To evaluate the pharmacokinetic properties of nonacog beta pegol
    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 procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
    2. Male Chinese patient with moderate to severe congenital haemophilia B with a FIX activity ≤2% according to medical records.
    3. Aged 12-70 years (both inclusive) at the time of signing informed consent.
    4. History of at least 100 EDs to products containing FIX.
    5. Patients currently on prophylaxis or patients currently treated on-demand with at least 6 bleeding episodes during the last 12 months or at least 3 bleeding episodes during the last 6 months.
    6. The patient, legally authorised representative (LAR) and/or caregiver are capable of assessing a bleeding episode, keeping a diary, performing home treatment of bleeding episodes and otherwise following the trial procedures.
    E.4Principal exclusion criteria
    1. Known or suspected hypersensitivity to trial product or related products.
    2. Previous participation in this trial. Participation is defined as signed informed consent.
    3. Participation in any clinical trial of an approved or non-approved investigational medicinal product within 5 half-lives or 30 days from screening, whichever is longer.
    4. Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR interviews.
    5. Current FIX inhibitors ≥0.6 BU.
    6. HIV positive, defined by medical records, with CD4+ count ≤200/μL and a viral load >200 particles/µL or >400000 copies/mL within 6 months of the trial entry. If the data are not available in the medical records within the last 6 months, then the test must be performed at the screening visit.
    7. Congenital or acquired coagulation disorder other than haemophilia B.
    8. 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).
    9. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) >3 times the upper limit of normal combined with total bilirubin >1.5 times the upper limit of normal at screening.
    10. Renal impairment defined as estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73 m^2 for serum creatinine measured at screening.
    11. Any disorder, except for conditions associated with haemophilia B, which in the investigator’s opinion might jeopardise the patient’s safety or compliance with the protocol.
    12. Platelet count <50×10*9/L at screening.
    13. Immune modulating or chemotherapeutic medication.
    14. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
    E.5 End points
    E.5.1Primary end point(s)
    Haemostatic effect of nonacog beta pegol when used for treatment of bleeding episodes during on demand and PPX
    E.5.1.1Timepoint(s) of evaluation of this end point
    From start of treatment (week 0) until end of treatment (up to week 50)
    E.5.2Secondary end point(s)
    Efficacy:
    1. Number of treated bleeding episodes during PPX treatment (Arm B only)
    2. Consumption of nonacog beta pegol for treatment of bleeding episodes
    3. Consumption of nonacog beta pegol for PPX treatment (Arm B only)
    4. FIX trough levels during PPX treatment (Arm B only)

    Safety:
    5. Number of patients with inhibitory antibodies against FIX defined as titre ≥0.6 Bethesda units (BU)
    6. Number of adverse events (AEs)
    7. Number of serious adverse events (SAEs)

    Pharmacodynamic:
    8. Incremental recovery (IR) (Arm B only)
    9. Terminal half-life (t½) (Arm B only)
    10. Clearance (CL) (Arm B only)
    11. Area under the curve (AUC) (Arm B only)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. - 7. From start of treatment (week 0) until end of treatment (week 50)
    8. Single-dose: 30±10 minutes post injection at week 0,
    Steady-state: 30±10 minutes post injection at week 12
    9. - 11. Single-dose: 0-168 hours post injection at week 0
    Steady-state: 0-168 hours post injection at week 12
    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 Yes
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    China
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    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: 15
    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: 15
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 13
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 30
    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
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: China
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