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    Summary
    EudraCT Number:2015-000075-27
    Sponsor's Protocol Code Number:HPN-100-021
    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:2016-01-13
    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 number2015-000075-27
    A.3Full title of the trial
    A Randomised, Controlled, Open-Label Parallel Arm study of the Safety, Pharmacokinetics and Ammonia Control of RAVICTI® (Glycerol Phenylbutyrate [GPB]) Oral Liquid and Sodium Phenylbutyrate (NaPBA) in Phenylbutyrate Treatment Naïve Patients with Urea Cycle Disorders (UCDs)
    Estudio abierto, aleatorizado, controlado en grupos paralelos para evaluar la seguridad, la farmacocinética y el control de amonio de RAVICTI® (fenilbutirato de glicerol [GPB]) líquido oral y del fenilbutirato de sodio (NaPBA) en pacientes que padecen trastornos del ciclo de la urea (TCU) y que no han recibido previamente tratamiento con fenilbutirato
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to compare RAVICTI® against Sodium Phenylbutyrate (NaPBA) in the treatment of Urea Cycle Disorders (UCDs) in patients who have not received Phenylbutyrate treatment before.
    Estudio para comparar RAVICTI® versus fenilbutirato de sodio (NaPBA) en el tratamiento de los trastornos del ciclo de la urea (TCU) en los pacientes que no han sido previamente tratados con sodio Fenilbutirato
    A.4.1Sponsor's protocol code numberHPN-100-021
    A.5.4Other Identifiers
    Name:IND NumberNumber:073480
    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 SponsorHorizon Therapeutics, LLC
    B.1.3.4CountryUnited States
    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 supportHorizon Therapeutics, LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHorizon Therapeutics, LLC
    B.5.2Functional name of contact pointDirector, Clinical Drug Development
    B.5.3 Address:
    B.5.3.1Street Address150 South Saunders Road
    B.5.3.2Town/ cityLake Forest
    B.5.3.3Post codeIL, 60045
    B.5.3.4CountryUnited States
    B.5.4Telephone number001224804 3202
    B.5.5Fax number001224241 3151
    B.5.6E-mailccanavan@horizonpharma.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 RAVICTI
    D.2.1.1.2Name of the Marketing Authorisation holderHorizon Pharma Ireland 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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/733 to 739
    D.3 Description of the IMP
    D.3.1Product nameRAVICTI
    D.3.2Product code N/A
    D.3.4Pharmaceutical form Oral liquid
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPNasogastric use (Noncurrent)
    Gastroenteral use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNGlycerol phenylbutyrate
    D.3.9.1CAS number 611168-24-2
    D.3.9.2Current sponsor codeHPN-100
    D.3.9.3Other descriptive nameGLYCEROL PHENYLBUTYRATE
    D.3.9.4EV Substance CodeSUB96043
    D.3.10 Strength
    D.3.10.1Concentration unit g/ml gram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.1
    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.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 AMMONAPS Tablets
    D.2.1.1.2Name of the Marketing Authorisation holderSwedish Orphan Biovitrum International AB
    D.2.1.2Country which granted the Marketing AuthorisationSweden
    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 nameAMMONAPS Tablets
    D.3.4Pharmaceutical form Tablet
    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 INNsodium phenylbutyrate
    D.3.9.1CAS number 1716-12-7
    D.3.9.2Current sponsor codeNaPBA
    D.3.9.3Other descriptive nameSODIUM PHENYLBUTYRATE
    D.3.9.4EV Substance CodeSUB12586MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 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.IMP: 3
    D.1.2 and D.1.3IMP RoleComparator
    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 AMMONAPS Granules
    D.2.1.1.2Name of the Marketing Authorisation holderSwedish Orphan Biovitrum International AB
    D.2.1.2Country which granted the Marketing AuthorisationSweden
    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 nameAMMONAPS Granules
    D.3.4Pharmaceutical form Granules
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPNasogastric use (Noncurrent)
    Gastroenteral use
    Oral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNsodium phenylbutyrate
    D.3.9.1CAS number 1716-12-7
    D.3.9.2Current sponsor codeNaPBA
    D.3.9.3Other descriptive nameSODIUM PHENYLBUTYRATE
    D.3.9.4EV Substance CodeSUB12586MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/g milligram(s)/gram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number940
    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
    Urea Cycle Disorders (UCDs)
    trastornos del ciclo de la urea (TCU)
    E.1.1.1Medical condition in easily understood language
    Genetic disease that affects how the body gets rid of waste from excess protein in the diet
    Enfermedad genética que afecta la manera cómo el cuerpo se deshace de los residuos de exceso de proteína en la dieta
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10013373
    E.1.2Term Disorders of urea cycle metabolism
    E.1.2System Organ Class 200000003094
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety, tolerability, pharmacokinetics (PK) and ammonia control of RAVICTI and NaPBA in UCD subjects not currently treated with phenylacetic acid (also referred to as phenylacetate; PAA) prodrugs.
    Evaluar la seguridad, la tolerabilidad, la farmacocinética (FC) y el control del amonio con RAVICTI y con NaPBA en sujetos con TCU que actualmente no reciben tratamiento con profármacos de ácido fenilacético (también conocido como fenilacetato (phenylacetate, PAA).
    E.2.2Secondary objectives of the trial
    Not applicable
    No se aplica
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Signed informed consent given by the subject or the subject?s parent/legal guardian for those under 18 years of age or the age of consent by local regulation
    -Male and female subjects with a suspected or confirmed UCD diagnosis of any subtype, except NAGS deficiency
    Suspected diagnosis is defined as having experienced a HAC or a documented high ammonia of ≥ 100 µmol/L
    Confirmed diagnosis is determined via enzymatic, biochemical, or genetic testing
    - Requires nitrogen-binding agents according to the judgment of the Investigator
    ≥2 months of age and older
    -All females of childbearing potential and all sexually active males must agree to use an acceptable method of contraception from signing the informed consent throughout the duration of the study and for 30 days after the last dose of study drug. Appropriate contraceptive methods include hormonal contraceptives (oral, injected, implanted, or transdermal), tubal ligation, intrauterine device, hysterectomy
    vasectomy, or double barrier methods. Abstinence is an acceptable form
    of birth control, though appropriate contraception must be used if the
    subject becomes sexually active.
    Criterios de inclusión:
    - Consentimiento informado firmado por el sujeto o los padres/tutores legales del sujeto para los menores de 18 años o de la edad estipulada de consentimiento por la normativa local.
    -Hombres y mujeres con una sospecha o confirmación de diagnóstico de cualquier subtipo de TCU excepto deficiencia de N-acetilglutamato sintetasa
    La sospecha del diagnóstico se define como haber experimentado una crisis de hiperamonemia o un nivel alto de amoniaco ≥ 100 μmol/l
    El diagnóstico confirmado se determina mediante análisis enzimáticos, bioquímicos o genéticos
    -Requiere agentes aglutinantes de nitrógeno a criterio del investigador
    2 meses de edad o más
    - Todas las mujeres en edad fértil y todos los hombres que tengan relaciones sexuales deben aceptar usar un método anticonceptivo aceptable durante todo el estudio. Entre los métodos anticonceptivos adecuados se incluyen anticonceptivos hormonales (orales, inyectados, implantados o transdérmicos), ligadura de trompas, dispositivos intrauterinos, histerectomías, vasectomías o métodos de doble barrera. La abstinencia es una forma aceptable de anticoncepción, aunque debe usarse un método anticonceptivo adecuado si el sujeto empieza a tener relaciones sexuales
    E.4Principal exclusion criteria
    - Subject has received chronic treatment with an oral phenylbutyrate (RAVICTI, NaPBA, Pheburane, or other) longer than 14 days consecutive within one year prior to enrollment
    - Temporary use of oral PBA for acute management of a hyperammonemic crisis in the past is acceptable.
    - Any concomitant illness (e.g., malabsorption or clinically significant liver or bowel disease) which would preclude the subject's safe participation, as judged by the Investigator
    - Have undergone liver transplantation, including hepatocellular transplant
    -Subjects on NaBz at Baseline will be excluded if they are viewed by the Investigator as being unable to undergo NaBz transition to a PAA
    prodrug during the Initial Treatment Period
    -Known hypersensitivity to PBA or any excipients of the NaPBA/PBA formulations
    - Pregnant or breast-feeding patients. Women of childbearing potential must have a pregnancy test performed at the Baseline Visit prior to the start of study drug.
    Criterios de exclusión:
    - El sujeto ha recibido tratamiento crónico con fenilbutirato oral (RAVICTI, NaPBA, Pheburano u otro)durante más de 14 días dentro del año anterior a la inscripción
    -Es aceptable el uso temporal de NaPBA para la gestión aguda de crisis de hiperamonemia en el pasado
    - Cualquier enfermedad concomitante (p. ej., hipoabsorción o afección intestinal o hepática clínicamente significativa) que pudiera impedir la participación segura del sujeto, a criterio del investigador
    - Haberse sometido a un trasplante de hígado, incluido un trasplante hepatocelular
    - Los sujetos en tratamiento con NaBz en el inicio se excluirán si el investigador considera que no pueden someterse a la transición de NaBz a un profármaco de PAA durante el periodo de tratamiento inicial.
    ? Pacientes embarazadas o en período de lactancia. Debe realizarse una prueba de embarazo a las mujeres que puedan tener hijos en la visita inicial antes de comenzar el medicamento del estudio
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of this study is the Rate of Treatment Success: Efficacy
    El objetivo primario de este estudio es la tasa de éxito del tratamiento: Eficacia
    E.5.1.1Timepoint(s) of evaluation of this end point
    The efficacy endpoint will be evaluated during the Initial Treatment Period
    Los criterios de valoración de la eficacia se evaluarán durante Período inicial de tratamiento
    E.5.2Secondary end point(s)
    1) Efficacy
    a) Initial Treatment Period Endpoints (RAVICTI vs NaPBA)
    • Drug discontinuation due to any reason
    b) Transition, Maintenance, and Safety Extension Periods (RAVICTI)
    • Control of plasma ammonia
    • Annualized rate of HAC
    2) Safety and Tolerability
    • Assessment of AEs
    • Standard clinical laboratory tests
    • Amino acid panel
    • Rate of drug discontinuation due to AEs
    3) Clinical Outcome Assessments
    • Rate of treatment success in the Initial Treatment Period
    • Subject preference for study drug (Arm 2 after exposure to both
    RAVICTI and NaPBA)
    • Palatability of study drug (Hedonic Scale)
    • Changes in CGI scales (Investigator)
    • Neuropsychological assessments: CBCL or ABCL or ASR
    • EQ-5D-5L health status quality of life assessment
    1) Eficacia
    a) Valoracion del Período inicial de tratamiento (RAVICTI vs NaPBA)
    • Suspensión del fármaco debido a cualquier razón
    b) Períodos de Extensión, Transición, Mantenimiento y Seguridad (RAVICTI)
    • Control del amoníaco plasmático
    • Índice anual de CHA
    2) Criterios de valoración de seguridad y tolerabilidad
    •Evaluación de AA
    •Análisis clínicos habituales
    •Perfil de aminoácidos
    •Índice de suspensión del fármaco debido a AA
    3)Evaluaciones de Resultados Clínicos
    •Índice de éxito del tratamiento en el período de tratamiento inicial
    •Preferencia por un fármaco (Rama 2 después de la exposición a ambos RAVICTI y NaPBA)
    •Sabor agradable del medicamento del estudio (escala hedónica)
    •Cambios en las escalas de impresión clínica global (Clinical Global Impression, CGI) (investigador)
    •Evaluaciones neurofisiológicas: Lista de comprobación de comportamiento infantil (Child Behavior Checklist, CBCL) o Lista de comprobación de comportamiento del adulto (Adult Behavior Checklist, ABCL)/Autoinforme del adulto (Adult Self-Report, ASR)
    •Evaluación de calidad de vida y estado de salud EQ-5D-5L
    E.5.2.1Timepoint(s) of evaluation of this end point
    a) Initial Treatment Period (RAVICTI vs NaPBA)
    • Drug discontinuation due to any reason
    • Amino acid panel
    • Standard clinical laboratory tests
    • Assessment of AEs
    • Rate of Treatment Success
    • EQ-5D-5L health status quality of life assessment
    b) Transition, Maintenance, and Safety Extension Periods (RAVICTI)
    • Control of plasma ammonia
    • Annualized rate of HAC
    • Amino acid panel
    • Standard clinical laboratory tests
    • Neuropsychological assessments: CBCL or ABCL or ASR
    • EQ-5D-5L health status quality of life assessment
    • Subject preference for study drug (Arm 2 after exposure to both RAVICTI and NaPBA)
    • Assessment of AEs
    • Changes in CGI scales (Investigator)
    • Palatability of study drug (Hedonic Scale)
    • Rate of drug discontinuation due to AEs
    a) Período inicial de tratamiento
    • Suspensión del fármaco debido a cualquier razón
    •Perfil de aminoácidos
    •Análisis clínicos habituales
    •Evaluación de AA
    •Índice de éxito del tratamiento
    •Evaluación de calidad de vida y estado de salud EQ-5D-5L
    b) Períodos de Extensión, Transición, Mantenimiento y Seguridad (RAVICTI)
    • Control del amoníaco plasmático
    • Índice anual de CHA
    •Perfil de aminoácidos
    •Análisis clínicos habituales
    •Evaluaciones neurofisiológicas:CBCL o ABCL o ASR
    •Evaluación de calidad de vida y estado de salud EQ-5D-5L
    •Preferencia por un fármaco (Rama 2 después de la exposición a ambos RAVICTI y NaPBA)
    •Evaluación de AA
    •Cambios en las escalas de ICG (investigador)
    Sabor agradable del medicamento (escala hedónica)
    Índice de suspensión del fármaco debido a AA
    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 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 Yes
    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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    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
    Austria
    Belgium
    Italy
    Spain
    Switzerland
    United Kingdom
    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
    LVLS
    LVLS - Ultima visita del ultimo 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 months4
    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 months4
    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: 23
    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: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 7
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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
    Subjects with diminished capacity may be enrolled in the study.
    Consent will be sought from parents/ guardians as appropriate
    Los sujetos con capacidad disminuida pueden participar en el estudio.
    Se solicitará el consentimiento de los padres / tutores legales según corresponda
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    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)
    On completion of study participation subjects will be assigned to treatment at the discretion of the investigator
    Al término de la participación en el estudio, los sujetos serán asignados al tratamiento a discreción del investigador
    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 Decision2016-02-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-11-06
    P. End of Trial
    P.End of Trial StatusOngoing
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