Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   42517   clinical trials with a EudraCT protocol, of which   7000   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .

    Clinical Trials marked as "Trial now transitioned" were transitioned to the Clinical Trial Regulation 536/2014 and can be further followed in the Clinical Trial Information System  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2013-003450-24
    Sponsor's Protocol Code Number:HGT-SAN-093
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-11-12
    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 number2013-003450-24
    A.3Full title of the trial
    A Randomized, Controlled, Open-label, Multicenter, Phase IIb Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Early Stage Mucopolysaccharidosis Type III A Disease
    Estudio de fase IIb aleatorizado, controlado, abierto, multicéntrico para evaluar la seguridad y la eficacia de la administración de HGT-1410 (heparán N sulfatasa humana recombinante) mediante un dispositivo de administración intratecal de medicación en pacientes pediátricos con mucopolisacaridosis de tipo IIIA incipiente
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy Study of HGT-1410 Administration in Pediatric Patients with Early Stage of Sanfilippo Syndrome Type A
    Estudio de seguridad y eficacia de la administración de HGT-1410 en pacientes pediátricos con síndrome de Sanfilippo de tipo A en estadío temprano.
    A.4.1Sponsor's protocol code numberHGT-SAN-093
    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 SponsorShire Human Genetic Therapies, Inc
    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 supportShire Human Genetic Therapies, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire Human Genetic Therapies Inc
    B.5.2Functional name of contact pointKenia Baez
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number0017814829620
    B.5.5Fax number0017814822954
    B.5.6E-mailkbaez@shire.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/08/582
    D.3 Description of the IMP
    D.3.1Product nameheparán N sulfatasa humana recombinante (rhHNS)
    D.3.2Product code HGT-1410
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntrathecal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNo disponible
    D.3.9.2Current sponsor codeHGT-1410
    D.3.9.3Other descriptive nameHeparán N sulfatasa humana recombinante
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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 Yes
    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 Yes
    D.3.11.13.1Other medicinal product typeHeparán N sulfatasa proteína humana producida en una línea celular humana mediante ingeniería genética.
    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
    Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Síndrome Sanfilippo Tipo A o mucopolisacaridosis (MPS IIIA)
    E.1.1.1Medical condition in easily understood language
    Sanfilippo Syndrome Type A or Mucopolysaccharidosis (MPS IIIA)
    Síndrome Sanfilippo Tipo A o mucopolisacaridosis (MPS IIIA)
    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 14.1
    E.1.2Level LLT
    E.1.2Classification code 10056918
    E.1.2Term Sanfilippo's syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10056890
    E.1.2Term Mucopolysaccharidosis III
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 potential clinical efficacy of HGT-1410 administered via a surgically implanted IDDD in patients with Sanfilippo Syndrome Type A (MPS IIIA). Efficacy will be measured as a meaningful amelioration in the progression of cognitive decline, and will be measured using the Bayley Scales of Infant Development, 3rd Edition (BSID III).
    Evaluar la eficacia clínica potencial de HGT-1410 administrado a través de un DAIM quirúrgicamente implantado en pacientes con síndrome Sanfilippo Tipo A (MPS IIIA). La eficacia se determinará como una mejoría significativa en el ritmo de deterioro cognitivo, y se evaluará aplicando las escalas de Bayley de desarrollo infantil, tercera edición(BSID III).
    E.2.2Secondary objectives of the trial
    To determine the following: the safety and tolerability of HGT-1410 IT; the effect of HGT-1410 administration on BSID-III age-equivalent and DQ scores; the effect of HGT-1410 IT on adaptive behavioral function, assessed by Vineland Adaptive Behavior Scales, Second Edition (VABS-II); the effect of HGT-1410 IT treatment on the total cortical grey matter volume, as assessed by volumetric MRI of the brain; the effect of HGT-1410 IT treatment on the concentration of GAG in CSF and urine; and the pharmacokinetics of HGT-1410 in cerebrospinal fluid (CSF) and serum.
    Se pretende determinar:
    La seguridad y tolerabilidad de HGT-1410 IT;
    El efecto de la administración de HGT-1410 sobre las puntuaciones del cociente de desarrollo (CD) y de edad equivalente de las escalas BSID III;
    El efecto de HGT-1410 IT sobre la función de la conducta adaptativa evaluada según las escalas de conducta adaptativa de Vineland, segunda edición (VABS II);
    El efecto del tratamiento con HGT-1410 IT sobre el volumen total de sustancia gris cortical, evaluado mediante RM volumétrica cerebral;
    El efecto del tratamiento con HGT-1410 IT sobre la concentración de glucosaminoglucanos (GAG) en LCR y en orina;
    La farmacocinética de HGT-1410 en el líquido cefalorraquídeo (LCR) y en el suero
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Patients must meet all of the following criteria to be considered eligible for enrollment:
    1. Documented MPS IIIA diagnosis:
    a) All patients must show a documented deficiency in sulfamidase enzyme activity of <= 10% of the lower limit of the normal range as measured in fibroblasts or leukocytes
    AND
    b) documented mutations in each SGSH allele OR there must be documentation of mutations in each SGSH allele in a sibling affected by MPSIIIA, provided parental consent is obtained to use this information.
    2. Age >= 12 months and <= 48 months
    3. The patient has a DQ score ?60%, assessed by cognitive evaluation at screening assessment using the BSID-III
    4. The patient is medically stable, in the opinion of the Investigator, and able to accommodate the protocol requirements, including travel, assessments, and IDDD surgery, without placing an undue burden on the patient/patient's family
    5. The patient's parent(s) or legal guardian must have voluntarily signed an Independent Ethics Committee-approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient's parent(s), or legal guardian. Consent of the patient's parent(s) or legally authorized guardian(s) must be obtained prior to the start of any study procedures.
    Los pacientes deberán cumplir todos los criterios siguientes para poder participar en el estudio:
    1. Diagnóstico de MPS IIIA documentado:
    a) Todos los pacientes deberán presentar una deficiencia documentada de actividad de la enzima sulfamidasa <=10 % del límite inferior del intervalo normal, determinada en fibroblastos o leucocitos
    Y
    b) Mutaciones documentadas en cada alelo SGSH o BIEN mutaciones documentadas en cada alelo SGSH en un hermano aquejado de MPS IIIA, previo consentimiento parental para el uso de tal información.
    2. Edad >= 12 meses y <= 48 meses
    3. Puntuación de cociente de desarrollo >= 60 %, determinada mediante evaluación cognitiva aplicando las escalas BSID III en la fase de selección.
    4. El paciente ha de estar médicamente estable, en opinión del investigador, y ser capaz de adaptarse a las exigencias del protocolo, tales como desplazamientos, evaluaciones y cirugía de implantación del DAIM, sin que ello suponga una carga indebida para el paciente o para su familia
    5. Los padres o representantes legales deberán haber firmado voluntariamente un documento de consentimiento informado, aprobado por el comité ético de investigación clínica, una vez les hayan sido explicados y comentados todos los aspectos pertinentes del estudio. Se obtendrá el consentimiento de los padres o representantes legales del paciente antes de empezar cualquier procedimiento del estudio.
    E.4Principal exclusion criteria
    Patients will be excluded from the study if any of the following criteria are met at screening:
    1. The presence of significant non-MPS IIIA related central nervous system (CNS) impairment or behavioral disturbances that would confound the scientific integrity or interpretation of study assessments, as determined by the Investigator.
    2. The presence of the S298P mutation in either or both SGSH alleles, associated with attenuated disease OR there is documentation of the S298P mutation in a sibling affected by MPS IIIA, provided parental consent is obtained to use this information.
    3. The presence of relatively attenuated MPS IIIA disease in an older sibling, defined as preservation of any comprehensible speech beyond the age of 10 years
    4. Visual or hearing impairment, in the clinical judgement of the investigator, sufficient to preclude cooperation with neurodevelopmental testing.
    5. In the opinion of the Investigator, the patient is assessed as having an unacceptably high risk for anesthesia due to airway compromise, drug hypersensitivity, or other conditions (such as neuroleptic malignant syndrome, malignant hyperthermia, or other anesthesia-related concerns).
    6. The patient has a history of poorly controlled seizure disorder.
    7. The patient is currently receiving psychotropic or other medications, which in the Investigator's opinion would be likely to substantially confound test results.
    8. The patient has a history of bleeding disorder or is unable to abstain from medications that, in the opinion of the investigator, place them at risk of bleeding following surgery or lumbar puncture..
    9. The patient participated in a clinical trial of another investigational medicinal product, within the 30 days prior to the study (or within 5 elimination half lives of the investigational product), or is currently enrolled in another study that involves an investigational drug or device. NOTE: Nutritional supplements, including genistein are permitted if they are taken or administered outside the context of a formal investigation.
    10. The patient has received a hematopoietic stem cell or bone marrow transplant, or gene therapy.
    11. The patient has a condition that is contraindicated as described in the SOPH-A-PORT Mini S IDDD Instructions for Use, including:
    a) The patient has had an allergic reaction to the materials of construction of the SOPH-A-PORT Mini S device
    b) The patient's body size is too small to support the size of the SOPH-A-PORT Mini S Access Port, as judged by the Investigator
    c) The patient has a known or suspected local or general infection
    d) The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
    e) The patient has a functioning CSF shunt device
    f) The patient has shown an intolerance to an implanted device
    12. The patient's parent(s) or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study, or do/does not agree to comply with the protocol defined schedule of assessments.
    13. The patient is unable to comply with the protocol (eg, has a clinically relevant medical condition making implementation of the protocol difficult, unstable social situation, or otherwise unlikely to complete the study) or is, in the opinion of the Investigator, otherwise unsuited for the study.
    14. The patient has any item (braces, tattoos, etc.) which would exclude the patient from being able to undergo MRI according to local Institutional Policy, or the patient has any other situation that would exclude the patient from undergoing any other procedure required in this study.
    Quedarán excluidos de este estudio los pacientes que cumplan alguno de los criterios siguientes en la fase de selección:
    1. Presencia de un deterioro significativo del sistema nervioso central (SNC) o de trastornos del comportamiento no relacionados con la MPS IIIA que, a criterio del investigador, pudieran perturbar la integridad científica o la interpretación de las evaluaciones del estudio.
    2. Presencia de la mutación S298P en uno o ambos alelos SGSH, asociada a una enfermedad atenuada, O BIEN mutación S298P documentada en un hermano aquejado de MPS IIIA, siempre que se obtenga el consentimiento parental para el uso de dicha información.
    3. Presencia de enfermedad MPS IIIA relativamente atenuada en un hermano de más edad, definida por la conservación de un habla comprensible después de los 10 años de edad.
    4. Insuficiencia visual o auditiva que a criterio clínico del investigador baste para impedir la colaboración durante las pruebas del desarrollo neurológico.
    5. Que la anestesia represente, a criterio del investigador, un riesgo elevado debido a compromiso de las vías aéreas, hipersensibilidad a los fármacos u otras patologías (como síndrome neuroléptico maligno, hipertermia maligna, u otros trastornos relacionados con la anestesia).
    6. Tener antecedentes de trastorno convulsivo mal controlado.
    7. Que el paciente esté recibiendo medicamentos psicótropos o de otro tipo que, a criterio del investigador, puedan alterar de manera sustancial los resultados de las pruebas.
    8. Que el paciente tenga antecedentes de trastornos hemorrágicos o no pueda prescindir de medicamentos que, a criterio del investigador, representen un riesgo hemorrágico después de la cirugía o la punción lumbar.
    9. Que el paciente haya participado en un ensayo clínico de otro fármaco experimental en los 30 días anteriores al estudio (o antes de que se cumplan 5 semividas de eliminación del fármaco experimental), o que esté participando actualmente en otra investigación de un fármaco o dispositivo experimental. NOTA: están permitidos los suplementos nutricionales, incluida la genisteína, siempre que se administren fuera del marco de una investigación formal.
    10. Que el paciente haya recibido un trasplante de médula ósea o de células madre hematopoyéticas, o terapia génica.
    11. Presentar alguna de las contraindicaciones que se indican en el manual de instrucciones del DAIM SOPH-A-PORT® Mini S, como por ejemplo
    a) Haber presentado una reacción alérgica a los materiales componentes del dispositivo SOPH-A-PORT® Mini S.
    b) Tener un tamaño corporal demasiado pequeño para soportar el tamaño del puerto de acceso SOPH-A-PORT® Mini S, según el criterio del investigador.
    c) Padecer una infección local o general confirmada o presunta.
    d) Tener una o más anomalías vertebrales que pudieran complicar la seguridad de la implantación o fijación.
    e) Tener un dispositivo de derivación del LCR en funcionamiento.
    f) Haber demostrado intolerancia a un dispositivo implantado.
    12. Que los padres o representantes legales del paciente no lleguen a comprender la naturaleza, el alcance y las posibles consecuencias del estudio, o rehúsen aceptar el programa de evaluaciones definido en el protocolo.
    13. Que el paciente no esté en condiciones de cumplir el protocolo (por ej., por padecer un trastorno médico clínicamente importante, o una situación social inestable o de otro tipo que haga difícil su realización) o que, a criterio del investigador, esté de algún modo incapacitado para el estudio.
    14. Que el paciente sea portador de algún objeto (ortesis, tatuajes, etc.) que haga desaconsejable la RM según las normas del centro, o se encuentre en cualquier otra situación que le impida someterse a algún procedimiento exigido en este estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Achievement of response, defined as a maximum decline in cognitive DQ of 10 points over 48 weeks, as assessed by the Bayley Scales of Infant Development, 3rd Edition.
    La obtención de respuesta, definida como un descenso máximo del CD cognitivo de 10 puntos a lo largo de 48 semanas, evaluado según las escalas de Bayley de desarrollo infantil, tercera edición.
    E.5.1.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 semanas
    E.5.2Secondary end point(s)
    - Safety endpoints including the assessment of adverse events (AEs), IDDD related issues, laboratory values, anti-rhHNS antibody development, vital signs, physical examination findings, and ECG results
    - The change from baseline to Week 48 in adaptive behavioral function, assessed by VABS II, using raw scores and age equivalent score
    - The change from baseline to Week 48 in the DQ assessed by neurocognitive testing, using the BSID-III age equivalent scores
    - The change from Baseline to Week 48 in total cortical grey matter volume, as assessed by MRI
    - The change from Baseline to Week 48 in concentrations of GAG in the CSF and urine
    - The concentration of HGT-1410 in CSF and serum
    - Los criterios de evaluación de la seguridad: evaluación de acontecimientos adversos (AA), aspectos relacionados con el DAIM, valores analíticos, desarrollo de anticuerpos anti-rhHNS, constantes vitales, resultados de la exploración física y resultados del ECG.
    - El cambio de las conductas adaptativas observado entre la situación basal y la semana 48, evaluado según las VABS II aplicando puntuaciones brutas y puntuaciones de edad equivalente.
    - El cambio del CD observado entre la situación basal y la semana 48, evaluado mediante pruebas neurocognitivas aplicando las puntuaciones de edad equivalente de las BSID-III
    - El cambio observado en el volumen de sustancia gris cortical entre la situación basal y la semana 48, evaluado mediante RM
    - La variación de las concentraciones de GAG en LCR y orina observada entre la situación basal y la semana 48
    - La concentración de HGT-1410 en LCR y suero
    E.5.2.1Timepoint(s) of evaluation of this end point
    48 weeks
    48 semanas
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other 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 Yes
    E.8.2.3.1Comparator description
    grupo control sin tratamiento
    no treatment control group
    E.8.2.4Number of treatment arms in the trial3
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Argentina
    France
    Germany
    Netherlands
    Spain
    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
    Last visit of the last patient in the clinical study
    Última visita del último paciente en el ensayo clínico
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    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: 18
    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: 8
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 10
    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
    Children between 12 and 48 months
    Niños con edades comprendidas entre los 12 y los 48 meses
    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 8
    F.4.2.2In the whole clinical trial 18
    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)
    It is anticipated that patients participating in this trial will be offered, upon completion of the study, enrollment in an extension trial, in which patients originally randomized to active treatment will continue to receive the same treatment regimen, while those originally randomized to the no-treatment control group will be re-randomized to one of the 2 active treatment regimens.
    Se prevé que los pacientes que participen en este ensayo se les ofrecerá, una vez completen el estudio, la inclusión en un ensayo de extensión, en el que los pacientes que en un principio se aleatorizaron para recibir tratamiento activo continuarán recibiendo el mismo tratamiento, mientras que aquellos que en un principio se asignaron al grupo de control sin tratamiento se volverán a aleatorizar a uno de los 2 tratamientos activos.
    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 Decision2014-01-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-11-27
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-06-01
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
    If you do not have an account, please visit the EMA Account management page page click on "Create an EMA account" and follow the instructions.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2022 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA