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    Summary
    EudraCT Number:2020-003001-58
    Sponsor's Protocol Code Number:NN7088-4595
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2022-11-01
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2020-003001-58
    A.3Full title of the trial
    A multi-centre, open-label trial evaluating efficacy, safety and pharmacokinetics of turoctocog alfa pegol (N8-GP) when used for treatment and prophylaxis of bleeding episodes in previously treated Chinese patients with haemophilia A
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy and safety of turoctocog alfa pegol (N8-GP) for prophylaxis and treatment of bleeding episodes in previously treated Chinese patients with haemophilia A
    A.3.2Name or abbreviated title of the trial where available
    pathfinder10
    A.4.1Sponsor's protocol code numberNN7088-4595
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1235-5905
    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 Esperoct 2000 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/3/12/995
    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 INNTuroctocog alfa pegol
    D.3.9.3Other descriptive nameTuroctocog alfa pegol
    D.3.9.4EV Substance CodeSUB191764
    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.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
    E.1.1.1Medical condition in easily understood language
    Bleeding disorder, inherited deficiency in clotting factor VIII
    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 10018938
    E.1.2Term Haemophilia A (Factor VIII)
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the clinical efficacy of N8-GP in bleeding prophylaxis (number of bleeding episodes during prophylaxis) in Chinese adolescent and adult patients with severe haemophilia A previously treated with other FVIII products
    E.2.2Secondary objectives of the trial
    In Chinese adolescent and adult patients with severe haemophilia A previously treated with other FVIII products,
    • To evaluate the clinical efficacy of N8-GP when used for treatment of bleeding episodes
    • To evaluate the immunogenicity of N8-GP
    • To evaluate the general safety of N8-GP
    • To evaluate the consumption of N8-GP
    • To evaluate the pharmacokinetic properties of N8-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 procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
    2. Male Chinese patient with severe congenital haemophilia A with a FVIII activity <1% according to medical records.
    3. Aged ≥12 years at the time of signing informed consent.
    4. History of at least 150 exposure days (EDs) to other FVIII products.
    5. The patient and/or caregiver is capable of assessing a bleeding episode, keeping a diary, performing home treatment of bleeding episodes and otherwise following the trial procedures at the discretion of the investigator.
    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 FVIII inhibitors based on existing medical records, laboratory report reviews and patient and/or caregiver interviews.
    5. Current FVIII inhibitors ≥0.6 BU.
    6. Congenital or acquired coagulation disorder other than haemophilia According to medical records.
    7. HIV positive, defined by medical records, with CD4+ count below or equal to 200/microL 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 medical records within last 6 months, then the test must be performed at screening visit.
    8. Previous significant thromboembolic events (e.g. myocardial infarction, cerebrovascular disease or deep venous thrombosis) 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, as defined by central laboratory.
    10. Renal impairment defined as estimated glomerular filtration rate (eGFR) ≤30 mL/min/1.73 m^2 for serum creatinine measured at screening, as defined by central laboratory.
    11. Platelet count <50×109/L at screening based on central laboratory values at screening.
    12. Ongoing immune modulating or chemotherapeutic medication.
    13. Any disorder, except for conditions associated with haemophilia A, which in the investigator`s opinion might jeopardise the patient`s safety or compliance with the protocol.
    14. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
    E.5 End points
    E.5.1Primary end point(s)
    Number of bleeding episodes
    E.5.1.1Timepoint(s) of evaluation of this end point
    From start of treatment until visit 7
    E.5.2Secondary end point(s)
    Efficacy
    1. Haemostatic effect of N8-GP when used for treatment of bleeding episodes, assessed on a four-point scale for haemostatic response (excellent, good, moderate and none)
    2. Consumption of N8-GP for treatment of bleeding episodes
    3. Consumption of N8-GP for prophylaxis
    4. FVIII trough activity during prophylaxis

    Safety
    5. Incidence rate of confirmed FVIII inhibitors ≥0.6 BU
    6. Number of adverse events (AEs)
    7. Number of serious adverse events (SAEs)

    PK
    8. FVIII activity 30 min post-injection (C30min)
    9. Incremental recovery (IR)
    10. Area under the curve (AUC)
    11. Terminal half-life (t½)
    12. Clearance (CL)
    13. FVIII trough activity 96 h post-injection (C96h)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.-3., 5. From start of treatment until visit 7
    4. From start of treatment (excluding the first exposure) until visit 7
    6.-7. From start of treatment until end of trial
    8.-9. Single-dose: 30 min ± 5 min post-injection at visit 2a, Steady-state: 30 min ± 5 min post-injection at visit 7
    10.-12. Single-dose: 0−inf post-injection at visit 2a, Steady-state: 0−96 h post-injection at visit 7
    13. Single-dose: 96 h ± 8 h post-injection at visit 2a, Steady-state: 96 h ± 8 h post-injection at visit 7
    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) 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 years1
    E.8.9.2In all countries concerned by the trial months3
    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: 18
    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: 18
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 18
    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.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 36
    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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