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    Summary
    EudraCT Number:2020-001048-24
    Sponsor's Protocol Code Number:NN7769-4514
    National Competent Authority:Latvia - SAM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-02-02
    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 ConcernedLatvia - SAM
    A.2EudraCT number2020-001048-24
    A.3Full title of the trial
    A multinational, open-label, randomised, controlled trial to investigate efficacy and safety of NNC0365-3769 (Mim8) in adults and adolescents with haemophilia A with or without inhibitors.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study investigating Mim8 in adults and adolescents with haemophilia A with or without inhibitors
    A.4.1Sponsor's protocol code numberNN7769-4514
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1249-4378
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/450/2020
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code NNC0365-3769 A
    D.3.4Pharmaceutical form Solution for injection
    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 INNN/A
    D.3.9.1CAS number *MASKED*
    D.3.9.2Current sponsor codeNNC0365-3769
    D.3.9.4EV Substance CodeSUB198393
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10 to 100
    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
    Haemophilia A with inhibitors
    E.1.1.1Medical condition in easily understood language
    Haemophilia A
    Haemophilia A with inhibitors
    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.1.2 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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To confirm the haemostatic effect of Mim8 as bleeding prophylaxis for adults and adolescents with haemophilia A with or without inhibitors.
    This will be done by:
    • Demonstrating superiority in number of bleeding episodes when treated with Mim8 once-weekly versus no prophylaxis for participants on no prophylaxis treatment prior to enrolment (Comparing Arm 1 and Arm 2 main treatment period)
    • Demonstrating non-inferiority in number of bleeding episodes when treated with either Mim8 once-weekly or once-monthly versus treatment with coagulation factor prophylaxis during run-in for participants on prophylaxis treatment prior to enrolment (Comparing Arm 3 and Arm 4 run-in with main treatment period).
    E.2.2Secondary objectives of the trial
    1. To investigate safety of Mim8 prophylaxis in adults and adolescents with haemophilia A with or without FVIII inhibitors.
    2. To evaluate the consumption of factor product per bleed treatment (number of injections) after Mim8 prophylaxis in adults and adolescents with haemophilia A with or without inhibitors.
    3. To evaluate the development of anti-Mim8 antibodies after Mim8 prophylaxis in adults and adolescents with haemophilia A with or without inhibitors.
    4. To evaluate treatment burden after Mim8 prophylaxis in adults and adolescents with haemophilia A with or without inhibitors.
    5. To evaluate aspects of patient’s physical functioning after Mim8 prophylaxis in adults and adolescents with haemophilia A with or without inhibitors.
    6. To evaluate joint pain intensity after Mim8 prophylaxis in adults and adolescents with haemophilia A with or without inhibitors.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Informed consent 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
    2. Male or female with diagnosis of congenital haemophilia A of any severity based on medical records
    3. Participants has been prescribed treatment with factor VIII concentrates or bypassing agent in the last 26 weeks prior to screening
    4. Age above or equal to 12 years at the time of signing informed consent.
    5. Body weight above or equal to 30 kg
    6. Applicable to participants treated with on-demand/no prophylaxis prior to enrolment: more than or equal to 5 bleeds in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed
    7. Applicable to participants with FVIII activity above or equal to 1% who are on prophylactic treatment: more than or equal to 1 bleed in the last 26 weeks prior to screening visit, for which factor VIII concentrates or bypassing agent has been prescribed
    8. Willingness and ability to comply with scheduled visits and study procedures, including the
    completion of diary and patient-reported outcomes questionnaires
    E.4Principal exclusion criteria
    1. Previous participation in this study. Participation is defined as signed informed consent
    2. Participation in any clinical study of an approved or non-approved investigational medicinal product, within 30 days (or 5 half-lives of the investigational medicinal product, whichever is greater) before screening
    3. Exposure to non-factor haemostatic products for bleeding prophylaxis within 6 months (or 5 half-lives of the medicinal product, whichever is shorter) prior to planned first dose, for participants not included in the run-in.
    4. Female who is pregnant, breast-feeding or intends to become pregnant or is of child-bearing
    potential and not using a highly effective contraceptive method. Breast feeding is allowed only during the run-in period
    5. Any disorder, except for conditions associated with haemophilia A, which in the investigator’s opinion might jeopardise participant’s safety or compliance with the protocol
    6. Known or suspected hypersensitivity to study product(s), any constituents of the product or to related products
    7. Receipt of gene therapy at any given time point
    8. Ongoing or planned ITI therapy
    9. Major surgery planned at the time of screening.
    10. Known congenital or acquired coagulation disorders other than haemophilia A
    11. Hepatic dysfunction defined as AST and/or ALT more than 3 times the upper limit combined with total bilirubin more than 1.5 times the upper limit measured at screening
    12. Renal impairment defined as estimated Glomerular Filtration Rate (eGFR) less than or equal to 30 ml/min/1.73 m^2 for serum creatinine measured at screening
    13. Previous or current thromboembolic disease or events (with the exception of previous
    catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or risk of thromboembolic disease, as evaluated by investigator
    14. Mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate
    understanding and cooperation
    15. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase
    risk of bleeding or thrombosis as evaluated by the investigator
    E.5 End points
    E.5.1Primary end point(s)
    Number of treated bleeds
    E.5.1.1Timepoint(s) of evaluation of this end point
    - No prophylaxis treatment (Arms 1 and 2): From randomisation (week 0) to end of main (week 26)
    - Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (week 26)
    E.5.2Secondary end point(s)
    1. Number of injection site reactions
    2. Occurrence of antiMim8 antibodies
    3. Number of treated spontaneous bleeds
    4. Number of treated joint bleeds
    5. Number of treated traumatic bleeds
    6. Number of treated target joint bleeds
    7. Consumption of factor product per bleed treatment (number of injections)
    8. Change in physical function domain of paediatric quality of life inventory (PEDS-QL)
    9. Change in participant’s treatment burden using the haemophilia treatment experience measure (Hemo-TEM)
    10. Change in participant’s joint pain score using Joint Pain Rating Scale
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. All participants receiving Mim8 (Arms 2, 3 and 4): From randomisation (week 0) to end of main (week 26)
    2. All participants receiving Mim8 (Arms 2, 3 and 4): From randomisation (week 0) to end of extension (week 52)
    3. – 7.:
    - No prophylaxis treatment (Arms 1 and 2): From randomisation (week 0) to end of main (week 26)
    - Prophylaxis treatment (Arms 3 and 4): From initiation of run-in (26-52 weeks prior to week 0) to week 0 and from randomisation (week 0) to end of main (week 26)
    8. – 10.:
    All participants (Arms 1, 2, 3 and 4): From randomisation (week 0) to the end of the main part (week 26)
    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 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    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 Yes
    E.8.1.7.1Other trial design description
    Intra-individual (within-patient) comparison
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Different treatment regimen of Mim8
    E.8.2.4Number of treatment arms in the trial4
    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 EEA53
    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
    China
    India
    Israel
    Japan
    Korea, Republic of
    Saudi Arabia
    South Africa
    Taiwan
    United States
    European Union
    Switzerland
    Russian Federation
    Serbia
    Turkey
    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 months3
    E.8.9.1In the Member State concerned days4
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days4
    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: 30
    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: 30
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 204
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 84
    F.4.2.2In the whole clinical trial 244
    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 Decision2021-04-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-29
    P. End of Trial
    P.End of Trial StatusOngoing
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