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    Summary
    EudraCT Number:2020-003467-26
    Sponsor's Protocol Code Number:NN7769-4516
    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:2022-01-20
    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 number2020-003467-26
    A.3Full title of the trial
    Safety, efficacy and exposure of subcutaneously administered NNC0365-3769
    (Mim8) prophylaxis in children with haemophilia A with or without FVIII
    inhibitors
    Seguridad, eficacia y exposición de la profilaxis con NNC0365-3769 (Mim8) administrado por vía subcutánea a niños con hemofilia A con o sin inhibidores del FVIII.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study investigating Mim8 in children with haemophilia A with or without inhibitors
    Estudio de investigación de Mim8 en niños con hemofilia A con o sin inhibidores
    A.4.1Sponsor's protocol code numberNN7769-4516
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1255-1540
    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.3.9.3Other descriptive nameNNC0365-3769
    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
    Hemofilia A,

    Hemofilia A con inhibidores
    E.1.1.1Medical condition in easily understood language
    Bleeding disorder, inherited deficiency in clotting factor VIII

    Bleeding disorder, inherited deficiency in clotting factor VIII with antibodies to clotting factor replacement therapy
    Desorden coagulación, deficiencia hereditaria factor VIII coagulación
    Desorden coagulación, deficiencia hereditaria factor VIII coagulación con anticuerpos frente a terapia reemplazo factores coag
    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 investigate the safety of Mim8 prophylaxis in children with haemophilia A with or without
    FVIII inhibitors.
    Investigar la seguridad de la profilaxis con Mim8 en niños con hemofilia A con o sin inhibidores del FVIII
    E.2.2Secondary objectives of the trial
    Investigate the efficacy of Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.
    Evaluate the consumption of coagulation factor replacement product per bleed treatment (number of injections) with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.
    Evaluate the development of anti-Mim8 antibodies with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.
    Investigate exposure of Mim8 after once-weekly and once-monthly subcutaneous dosing in children with haemophilia A with or without FVIII inhibitors.
    Evaluate treatment burden with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors, as reported by their caregivers.
    Investigate treatment preference among caregivers of previously treated children with haemophilia A with or without FVIII inhibitors.
    Evaluate aspects of physical functioning with Mim8 prophylaxis in children with haemophilia A with or without FVIII inhibitors.
    - Investigar eficacia de profilax con Mim8 en niños con hemofilia A con/sin inhibidores del FVIII
    - Evaluar utilización de productos de reposición de factores de coagulación para el tto de hemorragias (nº inyecciones) en niños con hemofilia A con/sin inhibidores del FVIII en profilax con Mim8
    - Evaluar desarrollo de anticuerpos frente a Mim8 en niños con hemofilia A con/sin inhibidores del FVIII en profilax con Mim8
    - Investigar exposición a Mim8 después de la administración subcutánea una vez a la semana y una vez al mes en niños con hemofilia A con/sin inhibidores del FVIII
    - Evaluar molestias derivadas del tto en niños con hemofilia A con o sin inhibidores del FVIII en profilax con Mim8, en base a lo comunicado por sus cuidadores
    - Investigar preferencia de tto entre cuidadores de niños con hemofilia A tratados previamente con/sin inhibidores del FVIII
    - Evaluar aspectos del funcionamiento físico en niños con hemofilia A con/sin inhibidores del FVIII en profilax con Mim8
    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 and female participants with the diagnosis of congenital haemophilia A of any severity
    based on medical records.
    3. Aged 1−11 years (both inclusive) at the time of signing informed consent.
    4. For previously treated participants :
    a. Participant has been prescribed treatment with FVIII concentrate or bypassing agent in the last 26 weeks prior to screening.
    b. Participants with endogenous FVIII activity above or equal to 1%, based on medical records, must have at least 1 treated bleed during the previous 26 weeks before screening for which factor VIII concentrate or bypassing agent has been prescribed (No requirements for participants with FVIII activity below 1%).
    5. For previously untreated participants:
    a. Diagnosis of severe haemophilia A (endogenous FVIII activity below 1%) based on medical records.
    6. Child and parent/caregiver willingness and ability to comply with scheduled visits and study procedures, including the completion of diary and patient-reported outcomes questionnaires.
    1. Obtención del consentimiento informado antes de cualquier actividad relacionada con el estudio. Se consideran actividades relacionadas con el estudio todos los procedimientos que se lleven a cabo como parte del estudio, incluidas las actividades para determinar la idoneidad para el mismo.
    2. Varones o mujeres con diagnóstico de hemofilia A congénita de cualquier gravedad según la historia clínica.
    3. Edad comprendida entre 1 y 11 años (ambos inclusive) en el momento de firmar el consentimiento informado.
    4. En los participantes tratados previamente:
    a. Al participante se le ha prescrito tratamiento con concentrado de FVIII o un agente bypass en las 26 semanas previas a la selección.
    b. Los participantes con una actividad del FVIII endógeno mayor o igual al 1 %, según la historia clínica, deben tener al menos una hemorragia tratada en las 26 semanas previas a la selección para la que se haya prescrito concentrado de factor VIII o un agente bypass (sin requisitos para los participantes con una actividad del FVIII menor del 1 %).
    5. En los participantes no tratados previamente:
    c. Diagnóstico de hemofilia A grave (actividad del factor VIII endógeno menor del 1 %) según la historia clínica.
    6. Disposición y capacidad del niño y los padres o el cuidador para cumplir las visitas programadas y los procedimientos del estudio, incluida la cumplimentación del diario y de cuestionarios de resultados comunicados por los pacientes.
    E.4Principal exclusion criteria
    1. Known or suspected hypersensitivity to study product or related products.
    2. Previous participation in this study. Participation is defined as signed informed consent.
    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. Known congenital or acquired coagulation disorders other than haemophilia A.
    5. Other conditions (e.g. autoimmune disease) or laboratory abnormality that may increase risk of
    bleeding or thrombosis, as evaluated by the investigator.
    6. Any disorder, except for conditions associated with haemophilia A, that in the investigator’s
    opinion might jeopardise the participant’s safety or compliance with the protocol.
    7. Mental incapacity, unwillingness to cooperate or a language barrier precluding adequate
    understanding and cooperation.
    8. Lack of adequate parental/caregiver support to enter accurately and timely information
    regarding treatment and bleeding episodes into an (electronic) diary.
    9. Previous or current treatment for thromboembolic disease (with the exception of previous
    catheter-associated thrombosis for which anti-thrombotic treatment is not currently ongoing) or
    signs of thromboembolic disease.
    10. Major surgery planned to take place after screening.
    11. Immune tolerance induction planned to take place after treatment initiation.
    12. Hepatic dysfunction defined as aspartate aminotransferase (AST) and/or alanine
    aminotransferase (ALT) above 3 times the upper limit of normal combined with total bilirubin above 1.5 times the upper limit of normal measured at screening.
    13. Serum creatinine above 1.5 x upper limit of normal (ULN), measured at screening.
    14. Pregnancy (female participants).
    1. Hipersensibilidad conocida o sospechada a los productos del estudio o a productos relacionados.
    2. Participación previa en este estudio. La participación se define como la firma del consentimiento informado.
    3. Exposición a productos hemostáticos que no sean factores para la profilaxis de hemorragias en los 6 meses (o 5 semividas del medicamento, lo que sea más corto) previos a la primera dosis prevista, para participantes no incluidos en el periodo de pre-inclusión.
    4. Trastornos congénitos o adquiridos conocidos de la coagulación distintos de la hemofilia A.
    5. Otras enfermedades (p. ej., enfermedad autoinmunitaria) o anomalías analíticas que puedan aumentar el riesgo de hemorragia o trombosis, según la evaluación del investigadora.
    6. Cualquier trastorno, excepto los procesos asociados a la hemofilia A, que en opinión del investigador podría poner en peligro la seguridad del participante o el cumplimiento del protocolo.
    7. Incapacidad mental, falta de cooperación o barrera idiomática que impida la debida comprensión y colaboración.
    8. Falta de apoyo por parte de los padres o el cuidador para registrar información exacta y en el momento requerido sobre el tratamiento y los episodios hemorrágicos en un diario (electrónico)
    9. Tratamiento previo o actual por enfermedad tromboembólica (con la excepción de trombosis previa asociada a un catéter para la que no esté recibiendo tratamiento antitrombótico) o signos de enfermedad tromboembólica.
    10. Intervención de cirugía mayor prevista después de la selección.
    11. Inducción de inmunotolerancia prevista después del inicio del tratamiento.
    12. Disfunción hepática definida como un valor de aspartato aminotransferasa (AST) o alanina aminotransferasa (ALT) por encima de 3 veces el límite superior de la normalidad combinado con un valor de bilirrubina total por encima de 1,5 veces el límite superior de la normalidad medido en la selección.
    13. Creatinina sérica por encima de 1,5 veces el límite superior de la normalidad (LSN), medida en la selección.
    14. Embarazo (mujeres participantes)
    E.5 End points
    E.5.1Primary end point(s)
    1. Number of treatment emergent adverse events
    1. Número de acontecimientos adversos de aparición durante el tratamiento
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. From treatment initiation to follow up visit (week 0 to week 72)
    1- Desde el inicio del tratamiento hasta la visita de seguimiento (de la semana 0 a la semana 72)
    E.5.2Secondary end point(s)
    1. Number of treated bleeds
    2. Number of treated spontaneous bleeds
    3. Number of treated traumatic bleeds
    4. Number of treated joint bleeds
    5. Number of treated target joint bleeds
    6. Number of injection site reactions
    7. Consumption of factor product per bleed treatment
    8. Occurrence of anti-Mim8 antibodies
    9. Mim8 plasma concentration
    10. Change in physical function domain of Paediatric Quality of Life inventory (PEDS-QL) Generic Core Scales
    11. Treatment preference for Mim8 versus previous treatment using Caregiver Haemophilia Patient Preference Questionnaire (Caregiver H-PPQ)
    12. Change in participants’ treatment burden using the Haemophilia treatment experience measure (Hemo TEM)
    1. Número de hemorragias tratadas
    2. Número de hemorragias espontâneas tratadas
    3. Número de hemorragias traumáticas tratadas
    4. Número de hemorragias articulares tratadas
    5. Número de hemorragias en articulaciones diana tratadas
    6. Número de reacciones en el lugar de inyección
    7. Utilización de productos con factor para el tratamiento de las hemorragias
    8. Aparición de anticuerpos frente a Mim8
    9. Concentración plasmática de Mim8
    10. Variación del módulo de función física de las escalas básicas genéricas del cuestionario de calidad de vida pediátrica (PEDS-QL)
    11. Preferencia por Mim8 en comparación con el tratamiento previo mediante el cuestionario H PPQ del cuidador
    12. Variación de las molestias al paciente derivadas del tratamiento mediante el cuestionario de medida de la experiencia del tratamiento de la hemofilia (Hemo-TEM)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-6 . From treatment initiation to end of treatment (week 0 to week 52)
    7. From run-in initiation to end of treatment (week -26 to week 52)
    8-10. From treatment initiation to end of treatment (week 0 to week 52)
    11. Once during treatment (week 26)
    12. From treatment initiation to end of treatment (week 0 to week 52)
    1-6. Desde el inicio del tratamiento hasta el final del tratamiento (de la semana 0 a la semana 52)
    7. Desde el inicio del periodo de pre-inclusion hasta el final de tratamiento (de la semana -26 a la semana 52)
    8-10. Desde el inicio del tratamiento hasta el final del tratamiento (de la semana 0 a la semana 52)
    11. Una vez durante el tratamiento (semana 26)
    12. Desde el inicio del tratamiento hasta el final del tratamiento (de la semana 0 a la semana 52)
    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 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 EEA22
    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
    European Union
    India
    Israel
    Japan
    Korea, Republic of
    Russian Federation
    Switzerland
    Taiwan
    United Kingdom
    United States
    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
    UVUP
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months11
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months11
    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: 70
    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: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 69
    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 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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 23
    F.4.2.2In the whole clinical trial 70
    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 Decision2022-04-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-04-06
    P. End of Trial
    P.End of Trial StatusOngoing
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