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    Summary
    EudraCT Number:2020-005092-13
    Sponsor's Protocol Code Number:MO42623
    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:2021-10-13
    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 number2020-005092-13
    A.3Full title of the trial
    A MULTICENTER, OPEN-LABEL PHASE IV STUDY TO EVALUATE OVERALL HEALTH, PHYSICAL ACTIVITY, AND JOINT OUTCOMES, IN PARTICIPANTS AGED ≥ 13 AND < 70 YEARS WITH SEVERE OR MODERATE HEMOPHILIA A WITHOUT FVIII INHIBITORS ON EMICIZUMAB PROPHYLAXIS
    ESTUDIO DE FASE IV, MULTICÉNTRICO Y ABIERTO PARA EVALUAR LA SALUD GENERAL, LA ACTIVIDAD FÍSICA Y LOS RESULTADOS ARTICULARES EN PARTICIPANTES DE 13 AÑOS O MÁS Y MENOS DE 70 AÑOS CON HEMOFILIA A GRAVE O MODERADA SIN INHIBIDORES DEL FVIII QUE RECIBEN PROFILAXIS CON EMICIZUMAB
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Evaluate Overall Health, Physical Activity and Joint Outcomes, in Participants with Severe or Moderate Hemophilia A without FVIII Inhibitors on Emicizumab Prophylaxis
    Un estudio para evaluar la salud general, la actividad física y los resultados de las articulaciones en participantes con hemofilia A grave o moderada sin inhibidores del FVIII que reciben profilaxis con emicizumab
    A.4.1Sponsor's protocol code numberMO42623
    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 SponsorRoche Farma S. A. U. que realiza el ensayo en España y que actúa como representante F. Hoffmann-La Roche Ltd
    B.1.3.4CountrySwitzerland
    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 supportF. Hoffman-La Roche Ltd.
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationF. Hoffmann-La Roche Ltd
    B.5.2Functional name of contact pointTrial Information Support Line-TISL
    B.5.3 Address:
    B.5.3.1Street AddressGrenzacherstrasse 124
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4070
    B.5.3.4CountrySwitzerland
    B.5.6E-mailspain.start_up_unit@roche.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 Hemlibra
    D.2.1.1.2Name of the Marketing Authorisation holderRoche Registration Limited
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameHemlibra/Emicizumab
    D.3.2Product code Ro 553-4262/F03-01
    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 INNEmicizumab
    D.3.9.1CAS number 1610943-06-0
    D.3.9.2Current sponsor codeRO553-4262
    D.3.9.3Other descriptive nameHEMLIBRA
    D.3.9.4EV Substance CodeSUB182706
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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 No
    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 Yes
    D.3.11.13.1Other medicinal product typeRO5534262 (ACE910) is a recombinant humanized anti-activated blood coagulation Factor IX (anti-FIXa) and anti-blood coagulation Factor X (anti-FX) bispecific monoclonal antibody.
    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
    Severe or Moderate Hemophilia A
    Hemofilia A severa o moderada
    E.1.1.1Medical condition in easily understood language
    Hemophilia A is an inherited bleeding disorder in which blood does not clot normally. People with hemophilia A will bleed more than normal for example after an injury, surgery, or dental procedure.
    La hemofilia A es trastorno hemorrágico hereditario en el que la sangre no se coagula normalmt. Personas con hemofilia A sangrarán más de lo normal, por ej, después de lesión, cirugía o proced dental.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    • To evaluate the impact of emicizumab treatment on joint health and health-related quality of life (HRQoL) outcomes of participants with hemophilia A as well as their physical activity
    - Evaluar el impacto del tratamiento con emicizumab en los resultados de la salud de las articulaciones y la calidad de vida relacionada con la salud (CVRS) de los participantes con hemofilia A, así como su actividad física.
    E.2.2Secondary objectives of the trial
    • To evaluate the safety of emicizumab
    • To evaluate the immune response to emicizumab
    • Evaluar la seguridad de emicizumab
    • Evaluar la respuesta inmune al emicizumab
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Age >=13 and <70 years at time of signing Informed Consent Form
    • Diagnosis of severe congenital hemophilia A (intrinsic FVIII level <1%) or moderate congenital hemophilia A (intrinsic FVIII level <=5%) if previously prescribed prophylaxis
    • A negative test for FVIII inhibitor (i.e., <0.6 BU) within 8 weeks of enrollment
    • Participants who completed successful immune tolerance induction (ITI) at least 5 years before screening are eligible, provided they have had no evidence of inhibitor recurrence (permanent or temporary) as may be indicated by detection of an inhibitor, FVIII half-life < 6 hours, or FVIII recovery < 66% since completing ITI
    • Participants who were on standard FVIII prophylaxis, defined as the regular administration of FVIII to prevent bleeding, for at least the last 24 weeks, can be enrolled regardless of the number of bleeds during this period
    • Adequate hematologic, hepatic and renal function
    • For women of childbearing potential: agreement to remain abstinent (refrain from heterosexual intercourse) or use contraception during the treatment period and for at least 24 weeks after the final dose of emicizumab
    - Edad ≥ 13 y < 70 años en el momento de firmar el documento de consentimiento
    informado.
    -Diagnóstico de hemofilia A congénita grave (nivel de FVIII intrínseco < 1 %) o hemofilia A
    congénita moderada (nivel de FVIII intrínseco ≤ 5 %) si se había prescrito previamente profilaxis.
    - Resultado negativo en la prueba de inhibidores del FVIII (es decir, < 0,6 UB) en las 8 semanas previas a la inclusión.
    - Los participantes sometidos con éxito a una inducción de tolerancia inmunitaria (ITI) al menos 5 años antes de la selección podrán participar en el estudio siempre que no haya habido indicios de reaparición (permanente o transitoria) de los inhibidores, como la detección de un inhibidor, una semivida del FVIII < 6 horas o una recuperación del FVIII < 66%, desde que finalizó la ITI.
    - Los participantes con profilaxis habitual con FVIII, definida como la administración
    habitual de FVIII para prevenir las hemorragias, durante al menos las últimas 24 semanaspodrán participar con independencia del número de hemorragias ocurridas durante este período. Se deben documentar los detalles del tratamiento profiláctico con FVIII y el número de episodios hemorrágicos durante al menos 24 semanas antes de la entrada en el estudio.
    - Función hematológica, hepática y renal adecuada
    - Para mujeres en edad fértil: acuerdo para permanecer en la abstinencia (abstenerse de tener relaciones heterosexuales) o usar anticonceptivos durante el período de tratamiento y durante al menos 24 semanas después de la dosis final de emicizumab.
    E.4Principal exclusion criteria
    • Inherited or acquired bleeding disorder other than severe congenital hemophilia A (intrinsic FVIII level <1%) or moderate congenital hemophilia A (intrinsic FVIII level <=5%) without FVIII inhibitors who were previously prescribed prophylaxis for at least 24 weeks
    • Participants who have previously received emicizumab prophylaxis
    • Participants who had joint replacement, joint procedure, synovectomy or synoviorthesis less than 5 years ago, or participants who had joint replacement, joint procedure, synovectomy or synoviorthesis more than 5 years ago but are still experiencing pain in the joint (only the specific joint will be excluded from the study), or participants that plan to have joint replacement, joint procedure, synovectomy or synoviorthesis, or participants that are deemed suitable candidates for joint replacement, joint procedure, synovectomy or synoviorthesis at screening
    • Participants who have conditions other than hemophilia A that can affect joint health and structure (e.g., osteoarthritis) or with severely impaired mobility due to conditions other than hemophilia A
    • Participants with reduced bone mineral density defined as clinically relevant vitamin D deficiency
    • Participants with pre-existing cardiovascular disease not receiving controlled and targeted medication or in a stable condition
    • Participants not eligible for MRI
    • History of illicit drug or alcohol abuse within 48 weeks prior to screening
    • Participants who are at high risk for thrombotic microangiopathy (TMA)
    • Previous (within the last 12 months) 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
    • Other conditions (e.g., certain autoimmune diseases) that may currently increase the risk of bleeding or thrombosis
    • History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
    • Planned surgery during the emicizumab loading dose phase. Surgeries in participants on emicizumab from Week 5 onwards are allowed
    • Known HIV infection not controlled by medication
    • Concomitant disease, condition, significant abnormality on screening evaluation or laboratory tests, or treatment that could interfere with the conduct of the study, or that would in the opinion of the investigator, pose an additional unacceptable risk in administering study drug to the participant
    • Receipt of any of the following:
    o An investigational drug to treat or reduce the risk of hemophilic
    bleeds within 5 half-lives of last drug administration at screening
    o A non-hemophilia-related investigational drug within last 30 days
    or 5 half-lives at screening, whichever is shorter
    o Any other investigational drug currently being administered or
    planned to be administered
    • Inability to comply with the study protocol
    • Pregnant or breastfeeding, or intending to become pregnant during the study
    - Trastorno hemorrágico hereditario o adquirido distinto de la hemofilia A congénita grave
    (nivel de FVIII intrínseco < 1%) o hemofilia A congénita moderada (nivel de FVIII
    intrínseco ≤ 5%) sin inhibidores del FVIII a los que se había prescrito previamente
    profilaxis durante al menos 24 semanas.
    - Participantes que hayan recibido previamente profilaxis con emicizumab.
    - Participantes sometidos a una artroplastia, intervención articular, sinonoviectomía o
    sinoviortesis hace menos de 5 años, o participantes sometidos a una artroplastia,
    intervención articular, sinonoviectomía o sinoviortesis hace más de 5 años pero que
    siguen experimentando dolor en la articulación (solo se excluirá del estudio la articulación concreta), o participantes que tengan previsto someterse a una artroplastia, intervención articular, sinonoviectomía o sinoviortesis, o participantes que se consideren candidatos adecuados para una artroplastia, intervención articular, sinonoviectomía o sinoviortesis en la selección.
    - Participantes con enfermedades distintas de la hemofilia A que puedan afectar a la salud y la estructura de las articulaciones (p. ej., artrosis) o con deterioro grave de la movilidad debido a enfermedades distintas de la hemofilia A.
    - Participantes con disminución de la densidad mineral ósea, definida como una deficiencia clínicamente relevante de vitamina D.
    - Participantes con enfermedad cardiovascular preexistente que no reciban medicación controlada y dirigida o que se encuentren en situación estable.
    - Participantes no candidatos para RM
    - Antecedentes de alcoholismo o toxicomanía en las 48 semanas previas a la selección.
    - Participantes con alto riesgo de MAT (p. ej., con antecedentes médicos o familiares de
    MAT-
    - Tratamiento previo (en los últimos 12 meses) o actual por enfermedad tromboembólica(excepto en caso de antecedentes de trombosis sin tratamiento antitrombótico en la actualidad) o signos de enfermedad tromboembólica.
    - Otras enfermedades que puedan aumentar actualmente el riesgo de hemorragia o trombosis (por ejemplo, algunas enfermedades autoinmunitarias).
    - Antecedentes de hipersensibilidad clínicamente significativa asociada al tratamiento con anticuerpos monoclonales o componentes de la inyección de emicizumab
    - Intervención quirúrgica programada durante la fase de dosis de carga de emicizumab. Se permiten las intervenciones quirúrgicas en pacientes en tratamiento con emicizumabdesde la semana 5 en adelante.
    - Infección conocida por el VIH no controlada con medicación
    - Enfermedad, trastorno, anomalía importante en la evaluación de selección o en los
    análisis clínicos o tratamiento concomitantes que podrían interferir en la realización delestudio o que, en opinión del investigador, supondrían un riesgo inaceptable adicional en la administración del fármaco del estudio al participante.
    - Recepción de cualquiera de los siguientes tratamientos:
    . Medicamento en investigación para tratar o reducir el riesgo de hemorragias
    hemofílicas en un período de 5 semividas desde la administración de la última dosis del fármaco en el momento de la selección.
    . Tratamiento con un medicamento en investigación no relacionado con la hemofilia en los últimos 30 días o 5 semividas antes de la selección, lo que más corto sea
    . Cualquier otro fármaco en investigación administrado actualmente o programado para administrarse.
    - Incapacidad para cumplir el protocolo del estudio.
    - Mujer embarazada o en período de lactancia o con intención de quedarse embarazada durante el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    1. Joint status over time based on centrally reviewed Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) scores with a specific focus on the synovitis score in participants with synovitis
    2. Clinical joint status over time based on the Hemophilia Joint Health Score (HJHS v2.1), excluding gait assessment
    3. Joint status at screening and month 36 based on centrally reviewed International Prophylaxis Study Group (IPSG) score (with MRI)
    4. Number of problem joints and proportion of problem joints, defined as joints having chronic joint pain and/or limited range of movement due to compromised joint integrity (i.e., chronic synovitis and/or hemophilic arthropathy) with or without persistent bleeding, over time
    5. Number of target joint bleeds over time (target joints are defined as joints with >=3 bleeds occurring in the same joint during the last 24 weeks)
    6. HRQoL, as assessed through use of the Comprehensive Assessment Tool of Challenges in Hemophilia (CATCH) Questionnaire over time
    7. Change in the level of physical activity during the study as measured with a wearable activity tracker (Fitbit)
    8. Change in daily step count, active minutes metabolic equivalents of tasks (METs), moderate to vigorous physical activity (MVPA; as per activity tracker default categorization), and type of physical activities
    9. Change in the time and intensity level of physical activity as measured by the International Physical Activity Questionnaire Short Format (IPAQ-SF)
    10. Number of all bleeds (i.e., those treated and untreated with FVIII), treated bleeds, spontaneous bleeds, joint bleeds, treated joint bleeds, and target joint bleeds (i.e., bleed rate) over time (ABR) as assessed through use of the Bleed and Medication Questionnaire (BMQ)
    11. Participant and/or caregiver preference for emicizumab compared with previous FVIII regimen, as assessed through use of the Emicizumab Preference Survey (EmiPref) at month 6
    1. Estado de la articulación a lo largo del tiempo basado en puntuaciones de detección de artropatía temprana de hemofilia con ultrasonido (HEAD-US) revisadas centralmente con un enfoque específico en la puntuación de sinovitis en participantes con sinovitis
    2. Estado clínico de las articulaciones a lo largo del tiempo según la puntuación de salud de las articulaciones de la hemofilia (HJHS v2.1), excluida la evaluación de la marcha
    3. Estado de las articulaciones en el cribado y en el mes 36 según la puntuación del International Prophylaxis Study Group (IPSG) revisada centralmente (con RM)
    4. Número de articulaciones problemáticas y proporción de articulaciones problemáticas, definidas como articulaciones que tienen dolor articular crónico y / o rango de movimiento limitado debido a integridad articular comprometida (es decir, sinovitis crónica y / o artropatía hemofílica) con o sin hemorragia persistente, a lo largo del tiempo
    5. Número de hemorragias articulares objetivo a lo largo del tiempo (las articulaciones objetivo se definen como articulaciones con> = 3 hemorragias en la misma articulación durante las últimas 24 semanas)
    6. CVRS, evaluada mediante el uso del Cuestionario de evaluación integral de los desafíos en la hemofilia (CATCH) a lo largo del tiempo
    7. Cambio en el nivel de actividad física durante el estudio medido con un rastreador de actividad portátil (Fitbit)
    8. Cambio en el recuento de pasos diarios, minutos activos equivalentes metabólicos de tareas (MET), actividad física de moderada a vigorosa (MVPA; según la categorización predeterminada del rastreador de actividad) y tipo de actividades físicas
    9. Cambio en el tiempo y el nivel de intensidad de la actividad física medido por el formato breve del cuestionario internacional de actividad física (IPAQ-SF)
    10. Número de todas las hemorragias (es decir, aquellas tratadas y no tratadas con FVIII), hemorragias tratadas, hemorragias espontáneas, hemorragias articulares, hemorragias articulares tratadas y hemorragias articulares diana (es decir, tasa de hemorragias) a lo largo del tiempo (ABR) según se evaluó mediante el uso de el cuestionario de sangrado y medicación (BMQ)
    11. Preferencia del participante y / o cuidador por emicizumab en comparación con el régimen anterior de FVIII, según se evaluó mediante el uso de la Encuesta de preferencia de Emicizumab (EmiPref) en el mes 6
    E.5.1.1Timepoint(s) of evaluation of this end point
    1-2. At Screening (Day -28 to Day -1), Months 6, 12, 24 and 36
    3. At Screening (Day -28 to Day -1) and Month 36
    4. At Months 6, 12, 24 and 36
    5. Up to 36 months
    6. At Baseline (Day 1), Months 3, 6, 12, 18, 24, and 36
    7-8. Until Month 36
    9. At Baseline (Day 1), Months 3, 6, 12, 18, 24, and 36
    10. Until Month 36
    11. At Month 6
    1-2. En la selección (día -28 al día -1), meses 6, 12, 24 y 36
    3. En la selección (día -28 a día -1) y mes 36
    4. En los meses 6, 12, 24 y 36
    5. Hasta 36 meses
    6. Al inicio del estudio (día 1), meses 3, 6, 12, 18, 24 y 36
    7-8. Hasta el mes 36
    9. Al inicio del estudio (día 1), meses 3, 6, 12, 18, 24 y 36
    10. Hasta el mes 36
    11. En el mes 6
    E.5.2Secondary end point(s)
    1. Incidence and severity of adverse events, with severity determined according to World Health Organization (WHO) toxicity scale
    2. Incidence of thromboembolic events
    3. Incidence of thrombotic microangiopathy
    4. Incidence of severe hypersensitivity, anaphylaxis, and anaphylactoid events
    5. Incidence and severity of injection-site reactions
    6. Prevalence of anti-drug antibodies (ADAs) against emicizumab at baseline and incidence of ADAs against emicizumab during the study
    7. Number and proportion of participants who develop anti-FVIII inhibitors (titer >=0.6 BU/mL) at specified timepoints
    1. Incidencia y gravedad de los eventos adversos, cuya gravedad se determina según la escala de toxicidad de la Organización Mundial de la Salud (OMS)
    2. Incidencia de eventos tromboembólicos
    3. Incidencia de microangiopatía trombótica
    4. Incidencia de reacciones graves de hipersensibilidad, anafilaxia y anafilactoides
    5. Incidencia y gravedad de las reacciones en el lugar de la inyección
    6. Prevalencia de anticuerpos antidrogas (ADA) contra emicizumab al inicio del estudio e incidencia de ADA contra emicizumab durante el estudio
    7. Número y proporción de participantes que desarrollan inhibidores anti-FVIII (título> = 0,6 UB / ml) en momentos específicos
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-5. Until 24 weeks (Safety Follow-up) after the final dose of emicizumab
    6. At Baseline (Day 1), Months 6, 12, 24 and 36
    7. Up to 36 months
    1-5. Hasta 24 semanas (seguimiento de seguridad) después de la dosis final de emicizumab
    6. Al inicio del estudio (día 1), meses 6, 12, 24 y 36
    7. Hasta 36 meses
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    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 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.8.2.4Number of treatment arms in the trial1
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA27
    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
    Brazil
    Canada
    Morocco
    Russian Federation
    Serbia
    Tunisia
    Turkey
    United States
    Germany
    Hungary
    Ireland
    Italy
    Spain
    Switzerland
    United Kingdom
    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
    uvup
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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: 40
    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: 40
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 78
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Signed by patient's legally authorized representative for patients who
    have not attained the age of majority
    Firmado por el representante legalmente autorizado del paciente para pacientes que
    no han alcanzado la mayoría de edad
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 120
    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)
    The Sponsor will offer continued access to emicizumab free of charge to eligible patients in accordance with the Roche Global Policy on Continued Access to Investigational Medicinal Product available at the following website: http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf.

    A patient's eligibility to receive emicizumab after completing the study
    is detailed in the Protocol section 4.3.4 "Continued Access to Emicizumab"
    El promotor ofrecerá acceso continuo a emicizumab sin cargo a los pacientes elegibles de acuerdo con la Política global de Roche sobre acceso continuo a medicamentos en investigación disponible en el siguiente sitio web: http://www.roche.com/policy_continued_access_to_investigational_medicines.pdf.
    La elegibilidad de un paciente para recibir emicizumab después de completar el estudio se detalla en la sección 4.3.4 del Protocolo "Acceso continuo a emicizumab"
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Covance/Labcorp Clinical Development SARL
    G.4.3.4Network Country France
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-10-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-10-06
    P. End of Trial
    P.End of Trial StatusOngoing
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