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    Summary
    EudraCT Number:2018-002154-70
    Sponsor's Protocol Code Number:CP40559
    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:2019-07-04
    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
    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 number2018-002154-70
    A.3Full title of the trial
    A MULTICENTER, SINGLE-ARM, OPEN-LABEL STUDY TO ASSESS THE SAFETY, PHARMACOKINETICS, AND EFFICACY OF BALOXAVIR MARBOXIL IN OTHERWISE HEALTHY PEDIATRIC PATIENTS FROM BIRTH TO LESS THAN 1 YEAR WITH INFLUENZA-LIKE SYMPTOMS
    ESTUDIO ABIERTO, MULTICÉNTRICO Y DE UN SOLO GRUPO PARA EVALUAR LA SEGURIDAD, LA FARMACOCINÉTICA Y LA EFICACIA DE BALOXAVIR
    MARBOXIL EN PACIENTES PEDIÁTRICOS CON SÍNTOMAS SEUDOGRIPALES Y POR LO DEMÁS ANOS DESDE EL NACIMIENTO HASTA MENOS DE 1
    AÑO
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study to Assess the Safety, Pharmacokinetics (how the body affects the drug), and Efficacy (how well the drug works) of Baloxavir Marboxil in Otherwise Healthy Pediatric Patients from Birth to < 1 Year with Influenza-Like Symptoms
    Un estudio para evaluar la seguridad, la farmacocinética (cómo el cuerpo afecta al fármaco) y la eficacia (qué tan bien funciona el fármaco) de Baloxavir Marboxil en pacientes pediátricos que de otra manera están sanos desde el nacimiento hasta <1 año con síntomas similares a los de la influenza
    A.3.2Name or abbreviated title of the trial where available
    Ministone 1
    A.4.1Sponsor's protocol code numberCP40559
    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
    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. Hoffmann-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.5.4Telephone number34913257300
    B.5.5Fax number34913248196
    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 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.3.1Product nameBaloxavir marboxil
    D.3.2Product code RO7191686/F08
    D.3.4Pharmaceutical form Granules for oral suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBALOXAVIR MARBOXIL
    D.3.9.2Current sponsor codeRO7191686
    D.3.9.4EV Substance CodeSUB190816
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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 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
    Influenza
    Gripe
    E.1.1.1Medical condition in easily understood language
    Influenza is a viral infection that is highly contagious and attacks the respiratory system including the nose, throat and lungs. It is commonly called as flu.
    La influenza es una infección viral que es altamente contagiosa y ataca el sistema respiratorio, incluyendo la nariz, la garganta y los pulmones. Es comúnmente llamado como gripe
    E.1.1.2Therapeutic area Diseases [C] - Virus Diseases [C02]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10022001
    E.1.2Term Influenza (epidemic)
    E.1.2System Organ Class 100000004862
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10022003
    E.1.2Term Influenza B virus infection
    E.1.2System Organ Class 100000004862
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10022002
    E.1.2Term Influenza A virus infection
    E.1.2System Organ Class 100000004862
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10016790
    E.1.2Term Flu
    E.1.2System Organ Class 100000004862
    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 safety of a single dose of baloxavir marboxil
    -Evaluar la seguridad de una dosis única de baloxavir marboxil
    E.2.2Secondary objectives of the trial
    • To evaluate the efficacy of baloxavir marboxil
    • To evaluate the virological activity of baloxavir marboxil
    • To evaluate the pharmacokinetics (PK) of baloxavir marboxil after single dose administration
    -Evaluar la eficacia de baloxavir marboxil
    -Evaluar la actividad virológica de baloxavir marboxil
    -Evaluar la farmacocinética de baloxavir marboxil después de la
    administración de una dosis única
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Age from birth to < 1 year at screening
    - Parent/guardian willing and able to comply with study requirements, in the investigator’s judgment
    - Patients with a diagnosis of influenza virus infection confirmed by the presence of all of the following:
    o Fever >= 38°C (tympanic temperature) at screening
    o At least one respiratory symptom (either cough or coryza)
    - The time interval between the onset of symptoms and screening is <= 96 hours
    -Edad desde el nacimiento hasta < 1 año en la visita de selección
    -Disposición y capacidad de los padres o el tutor para cumplir los requisitos del estudio, a criterio del investigador
    -Pacientes con diagnóstico de infección por el virus de la gripe confirmada por la presencia de todo lo siguiente:
    - Fiebre ≥ 38 C (temperatura timpánica) en la visita de selección.
    - Al menos un síntoma respiratorio (tos o rinitis)
    -El intervalo entre el comienzo de los síntomas y la selección es <= 96 horas
    E.4Principal exclusion criteria
    - Hospitalized for complications of influenza or significant comorbidities
    - Concurrent infections requiring systemic antiviral therapy at screening
    - Require, in the opinion of the investigator, any of the prohibited medication during the study
    - Preterm neonates (born at < 37 weeks gestation)
    - Weigh < 2.5 kg at screening
    o Only children > 4.5 kg will be eligible to take part in countries that have volume limits for blood draws set at 1% of the total blood volume (maximum over 24 hour period) and 3% of the total blood volume (maximum over 30-day period)
    - Previous treatment with peramivir, laninamivir, oseltamivir, zanamivir, or amantadine within 2 weeks prior to screening
    - Immunization with a live/attenuated influenza vaccine during the 2 weeks prior to screening
    - Concomitant treatment with steroids or other immuno-suppressant therapy
    - Known HIV infection or other immunosuppressive disorder
    - Uncontrolled renal, vascular, neurologic or metabolic disease, hepatitis, cirrhosis, or pulmonary disease or patients with known chronic renal failure
    - Active cancer at any site
    - History of organ transplant
    - Known allergy to study drug or to acetaminophen
    - Participation in a clinical trial within 4 weeks or five half-lives of exposure to an investigational drug prior to screening, whichever is longer
    -Hospitalización por complicaciones de la gripe o enfermedades concomitantes importantes.
    -Infecciones concurrentes con necesidad de tratamiento antiviral sistémico en la fase de selección.
    -Necesidad, en opinión del investigador, de cualquiera de los medicamentos no permitidos durante el estudio.
    -Recién nacidos prematuros (nacidos con < 37 semanas de gestación)
    -Peso < 2,5 kg en la visita de selección
    -En los países donde se hayan fijado límites de volumen para las extracciones de sangre del
    1 % del volumen de sangre total (máximo durante un periodo de 24 horas) y el 3 % del volumen de sangre total (máximo durante un período de 30 días) solo podrán participar los niños que pesen > 4,5 kg
    -Tratamiento previo con peramivir, laninamivir, oseltamivir, zanamivir o amantadina en las dos semanas previas a la selección.
    -Vacunación con una vacuna antigripal de virus vivos/atenuados en las dos semanas previas a la visita de selección.
    -Tratamiento concomitante con esteroides u otros inmunodepresores.
    -Infección conocida por el VIH u otro trastorno inmunodepresor.
    - Enfermedad renal, vascular, neurológica o metabólica no controlada (p. ej., diabetes,
    trastornos tiroideos, enfermedad suprarrenal), hepatitis, cirrosis o neumopatía o pacientes
    con insuficiencia renal crónica conocida.
    Cáncer activo en cualquier localización.
    - Antecedentes de trasplante de órganos
    -Hipersensibilidad conocida al fármaco del estudio a los excipientes del medicamento
    -Participación en un ensayo clínico con un fármaco experimental en las 4 semanas, previas a la selección, lo que suponga más tiempo
    E.5 End points
    E.5.1Primary end point(s)
    1. Incidence, severity, and timing of adverse events, serious adverse events, vital sign measurements, and clinical laboratory tests
    1. Incidencia, gravedad y tiempo de los eventos adversos, eventos adversos graves, mediciones de signos vitales y pruebas de laboratorio clínico
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Up to Day 29 or early termination
    1. Hasta el día 29 o terminación anticipada
    E.5.2Secondary end point(s)
    1. Time to alleviation of influenza signs and symptoms
    2. Duration of fever
    3. Duration of symptoms
    4. Time to return to normal health and activity
    5. Frequency of influenza-related complications
    6. Proportion of patients requiring antibiotics
    7. Time to cessation of viral shedding by virus titer and by reverse transcription-polymerase chain reaction (RT PCR)
    8. Change from baseline in influenza virus titer and in the amount of virus RNA (RT-PCR) at each time point
    9. Proportion of patients with positive influenza virus titer and proportion of patients positive by RT-PCR at each time point
    10. Area under the curve in virus titer and in the amount of virus RNA (RT-PCR)
    11. Plasma concentrations of baloxavir marboxil (pro drug) and S-033447 (active metabolite) will be summarized by time C24 (plasma concentration 24 hours postdose) and C72 (plasma concentration 72 hours postdose) and cohort
    12. Population PK model derived PK parameters (e.g., apparent clearance [CL/F], apparent volume of distribution for the central compartment [Vc/F], area under the concentration–time curve from time 0 to infinity [AUCinf], maximum plasma concentration [Cmax])
    1. Es hora de aliviar los signos y síntomas de la gripe
    2. Duración de la fiebre
    3. Duración de los síntomas.
    4. Es hora de volver a la salud normal y la actividad.
    5. Frecuencia de complicaciones relacionadas con la influenza.
    6. Proporción de pacientes que requieren antibióticos.
    7. Tiempo hasta el cese de la propagación viral por título de virus y por reacción en cadena de la polimerasa de transcripción inversa (RT PCR)
    8. Cambio desde el inicio en el título del virus de la influenza y en la cantidad de ARN del virus (RT-PCR) en cada punto de tiempo
    9. Proporción de pacientes con valor positivo del virus de la influenza y proporción de pacientes positivos por RT-PCR en cada momento
    10. Área bajo la curva en el título del virus y en la cantidad de ARN del virus (RT-PCR)
    11. Las concentraciones plasmáticas de baloxavir marboxil (pro drogas) y S-033447 (metabolito activo) se resumirán por el tiempo C24 (concentración plasmática 24 horas después de la dosis) y C72 (concentración plasmática 72 horas después de la dosis) y cohorte
    12. Los parámetros de PK derivados del modelo PK de la población (por ejemplo, aclaramiento aparente [CL / F], volumen aparente de distribución para el compartimiento central [Vc / F], área bajo la curva de concentración-tiempo desde el tiempo 0 hasta el infinito [AUCinf], máximo concentración plasmática [Cmax]
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-4. Up to Day 15
    5-7. Up to Day 29
    8. From baseline (Day 1) to Day 29
    9-10. Up to Day 29
    11. 24 and 72 hours post dose of study drug
    12. Day 1, Day 2, Day 4, Day 6-10
    .1-4. Hasta el día 15
    5-7. Hasta el día 29
    8. Desde la línea de base (día 1) hasta el día 29
    9-10. Hasta el día 29
    11. 24 y 72 horas después de la dosis del fármaco del estudio.
    12. Día 1, Día 2, Día 4, Día 6-10
    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 Yes
    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 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    Costa Rica
    Finland
    Israel
    Mexico
    Panama
    Poland
    Russian Federation
    South Africa
    Spain
    Thailand
    United States
    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
    The end of this study is defined as the date when the last patient, last visit (LPLV) occurs or the date at which the last data point required for statistical analysis or safety follow-up is received from the last patient, whichever occurs later.
    El final de este estudio se define como la fecha en que se produzca la última visita del último paciente o la fecha en la que se reciban los últimos datos necesarios para los análisis estadísticos o el
    seguimiento de la seguridad del último paciente, lo que ocurra más tarde. Está previsto que el final
    el estudio tenga lugar 29 días después de la inclusión del último paciente.
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    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: 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) Yes
    F.1.1.3.1Number of subjects for this age range: 4
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 26
    F.1.1.5Children (2-11years) No
    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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Otherwise healthy pediatric patients from birth to less than 1 year with influenza-like symptoms.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 15
    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)
    Currently, the Sponsor does not have any plans to provide the Roche IMP (baloxavir marboxil) or interventions to patients who have completed the study
    Actualmente, el Sponsor no tiene planes de proporcionar el Roche IMP (baloxavir marboxil) o intervenciones a pacientes que hayan completado el estudio.
    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 Decision2019-09-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-24
    P. End of Trial
    P.End of Trial StatusOngoing
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