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:2014-004143-13
    Sponsor's Protocol Code Number:SHP-609-302
    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:2015-02-09
    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 number2014-004143-13
    A.3Full title of the trial
    An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction with Elaprase® in Patients with Hunter Syndrome and Cognitive Impairment
    Estudio de extensión abierto de HGT-HIT-094 para evaluar la seguridad y el resultado clínico a largo plazo de la idursulfasa intratecal (IT) administrada junto con Elaprase® en pacientes con síndrome de Hunter y deterioro cognitivo
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An extension peadiatric study of Idursulfase-IT with Elaprase® in patients with Hunter Syndrome and early cognitive impairment
    Estudio de extensión pediatrico de la idursulfasa-IT administrada junto con Elaprase® en pacientes con síndrome de Hunter y deterioro cognitivo
    A.4.1Sponsor's protocol code numberSHP-609-302
    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 HGT
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire HGT
    B.5.2Functional name of contact pointSenior Clinical Program Scientist
    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 number34914229372
    B.5.5Fax number0017814821819
    B.5.6E-mailInfoShireIberica@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/3/01/078
    D.3 Description of the IMP
    D.3.1Product nameIdursulfase-IT
    D.3.2Product code HGT-2310
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    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 INNIDURSULFASE
    D.3.9.1CAS number 50936-59-9
    D.3.9.2Current sponsor codeHGT-2310
    D.3.9.3Other descriptive nameidursulfase-IT
    D.3.9.4EV Substance CodeSUB22927
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 typeIdursulfase-IT, human enzyme produced from genetically engineered human cell line
    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
    Hunter syndrome and cognitive impairment
    Síndrome de Hunter y deterioro cognitivo
    E.1.1.1Medical condition in easily understood language
    Hunter syndrome-Iduronate-2-Sulfatase enzyme deficiency
    Síndrome de Hunter y deficiencia de la encima Iduronato-2-sulfatasa
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level PT
    E.1.2Classification code 10056889
    E.1.2Term Mucopolysaccharidosis II
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.0
    E.1.2Level LLT
    E.1.2Classification code 10056917
    E.1.2Term Hunter's syndrome
    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
    The primary objective of this study is to evaluate long-term safety in patients with Hunter syndrome and cognitive impairment who are receiving intrathecal idursulfase-IT and intravenous (IV) Elaprase® enzyme replacement therapy (ERT)
    El objetivo principal de este estudio consiste en evaluar la seguridad a largo plazo en pacientes con síndrome de Hunter y deterioro cognitivo tratados con idursulfasa-IT por vía intratecal y tratamiento de reposición enzimática (TRE) con Elaprase® por vía intravenosa (IV)
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are to evaluate long term clinical efficacy outcomes in patients with Hunter syndrome and cognitive impairment who are treated with idursulfase-IT in conjunction with Elaprase therapy with respect to the following:
    . Cognitive function as measured by General Conceptual Ability (GCA), the cluster areas and subtests of the Differential Abilities Scale, 2nd edition (DAS-II) or the British Abilities Scales, Second Edition (BAS-II), or domains of the Bayley Scales of Infant Development, Third Edition (BSID-III)
    . Adaptive behavior as measured by the Adaptive Behavior Composite (ABC) score and standard domain scores of the Vineland Adaptive Behavior Scales, Second Edition (VABS-II)
    . Brain structure volume as measured by magnetic resonance imaging (MRI)

    For PK/PD, health economics and outcome research and device objectives see protocol section 2.3 - 2.5.
    Los objetivos secundarios de este estudio consisten en evaluar resultados de eficacia clínica a largo plazo en pacientes con síndrome de Hunter y deterioro cognitivo tratados con idursulfasa-IT junto con Elaprase en lo que respecta a:
    . Función cognitiva, determinada mediante la capacidad conceptual general (GCA), las áreas agrupadas y subpruebas de la escala DAS-II (Escala de habilidades diferenciales, 2ª edición), o la escala BAS-II (Escala británica de aptitudes, 2ª edición) o dominios de la escala BSID-III (Escalas de desarrollo infantil de Bayley, 3ª edición).
    . Conducta adaptativa, determinada mediante la puntuación ABC (Combinado de conducta adaptativa) y las puntuaciones de dominios normalizadas de la escala VABS-II (Escalas de conducta adaptativa de Vineland, 2ª ed).
    . Volumen de las estructuras cerebrales, medido mediante resonancia magnética (RM).
    Para FC/FD, economía sanitaria e investigación de resultados y objetivo del dispositivo, ver sección del protocolo 2.3-2.5.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients must have completed Visit Week 52 assessments in Study HGT-HIT-094
    2. The patient´s parent(s) or legally authorized guardian(s) must have voluntarily signed an Institutional Review Board (IRB)/Independent Ethics Committee (IEC) approved informed consent form after all relevant aspects of the study have been explained and discussed. Consent of the patient´s parent(s) or legally authorized guardian(s) and the patient´s assent, as relevant, must be obtained
    3. The patient has continued to receive Elaprase on a regular basis in Study HGT-HIT-094
    1. El paciente ha completado las evaluaciones de la visita de la semana 52 del estudio HGT-HIT-094.
    2. El progenitor o tutor legal del paciente ha firmado voluntariamente un documento de consentimiento informado aprobado por el comité ético de investigación clínica (CEIC), una vez explicados y comentados todos los aspectos pertinentes del estudio. Deberá obtenerse el consentimiento del progenitor o tutor legal del paciente y el asentimiento del paciente, según proceda.
    3. El paciente ha seguido recibiendo Elaprase de forma regular en el estudio HGT-HIT-094.
    E.4Principal exclusion criteria
    1. The patient has experienced, in the opinion of the Investigator, a safety or medical issue that contraindicates treatment with idursulfase-IT, including, but not limited to, uncontrolled seizure disorder, bleeding disorder, and clinically relevant hypertension
    2. The patient has a known hypersensitivity to any of the components of idursulfase-IT
    3. The patient has clinically relevant intracranial hypertension
    4. The patient is enrolled in another clinical study, other than HGT-HIT-094, that involves clinical investigations or use of any investigational product (drug or [intrathecal/spinal] device) within 30 days prior to study enrollment or at any time during the study
    5. The patient has any known or suspected hypersensitivity to anesthesia or is thought to be at an unacceptably high risk for anesthesia due to compromised airways or other conditions.
    6. 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, or may have, 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´s drug therapy requires substances known to be incompatible with the materials of construction
    d. The patient has a known or suspected local or general infection
    e. The patient is at risk of abnormal bleeding due to a medical condition or therapy
    f. The patient has one or more spinal abnormalities that could complicate safe implantation or fixation
    g. The patient has a functioning CSF shunt device
    h. The patient has shown an intolerance to an implanted device
    1. El paciente ha experimentado, en opinión del investigador, un problema de seguridad o médico que contraindica el tratamiento con idursulfasa-IT, entre otros, trastorno convulsivo no controlado, trastorno hemorrágico o hipertensión clínicamente relevante.
    2. El paciente presenta hipersensibilidad confirmada a alguno de los componentes de la idursulfasa-IT.
    3. El paciente presenta hipertensión intracraneal clínicamente relevante.
    4. El paciente ha participado en otro ensayo clínico, distinto del HGT-HIT-094, que suponga la investigación o el uso clínico de cualquier producto en investigación (fármaco o dispositivo intratecal/vertebral) en los 30 días previos a la inclusión en el estudio o lo hace en algún momento durante el mismo.
    5. El paciente presenta hipersensibilidad confirmada o presunta a anestésicos o una afectación de las vías respiratorias u otras enfermedades que conlleven un riesgo inaceptablemente alto de la anestesia.
    6. El paciente presenta alguna de las contraindicaciones que se indican en el manual de instrucciones del SAMI SOPH-A-PORT Mini S, como por ejemplo:
    a. Haber presentado o poder presentar una reacción alérgica a los materiales de construcción del sistema SOPH-A-PORT Mini S.
    b. Tener una constitución corporal demasiado pequeña para soportar el tamaño del puerto de acceso SOPH-A-PORT Mini S, según el criterio del investigador.
    c. Necesitar tratamiento farmacológico con sustancias incompatibles con los materiales de construcción.
    d. Padecer una infección local o general confirmada o presunta.
    e. Presentar riesgo de hemorragia anormal debida a una enfermedad o tratamiento.
    f. Tener una o más anomalías vertebrales que pudieran complicar la seguridad de la implantación o fijación.
    g. Tener un dispositivo de derivación del LCR en funcionamiento.
    h. Haber mostrado intolerancia a un dispositivo implantado.
    E.5 End points
    E.5.1Primary end point(s)
    Safety will be assessed during the study by the following:
    . adverse events (AEs) (by type, severity, and relationship to treatment [idursulfase-IT, the IDDD, device surgical procedure, or IT administration process] and IV Elaprase infusion)
    . changes in clinical laboratory testing (serum chemistry, hematology, urinalysis)
    . vital signs
    . 12-lead ECG recordings
    . CSF laboratory parameters (chemistries, cell counts)
    . anti-idursulfase antibodies in CSF and serum, including determination of anti-idursulfase antibodies having enzyme neutralizing activity
    La seguridad será valorada durante el estudio por los siguientes:
    . Acontecimientos adversos (AA; según su tipo, intensidad y relación con el tratamiento [idursulfasa-IT, sistema de administración de medicación por vía intratecal (SAMI), procedimiento quirúrgico vinculado al dispositivo o proceso de administración intratecal] y la infusión de Elaprase IV).
    . Variaciones de los análisis clínicos (bioquímica sérica, hematología, análisis de orina).
    . Constantes vitales.
    . ECG de 12 derivaciones.
    . Parámetros analíticos en LCR (bioquímica, recuentos celulares).
    . Anticuerpos antiidursulfasa en LCR y suero, incluida la determinación de anticuerpos antiidursulfasa con actividad neutralizante de la enzima.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Adverse events (AEs) will be assessed during all the study. CSF assessments (other than standard chemistry) and clinical laboratory tests of blood and urine will be performed every 12 weeks. 12-lead ECG will be performed every 48 weeks.

    For all other endpoints please refer to Appendix 1 to 7 of the protocol.
    Los Acontecimientos adversos (AA) serán valorados durante todo el estudio. Valoraciones del LCR (distinto al estándar de química) y analisis de laboratorio clínico de sangre y orina se realizarán cada 12 semanas. ECG de 12 derivaciones se realizará cada 48 semanas.

    Para todas las demás pruebas consultar el Apéndice 1 a 7 del protocolo.
    E.5.2Secondary end point(s)
    . Change from baseline in standard scores in cluster areas of the DAS-II/BAS-II: GCA, Verbal, Nonverbal, Spatial, and Special Nonverbal Composite (SNC), or the age equivalents and Development Quotient (DQ) from the BSID-III domains: Cognitive and Language
    . Change from baseline in standard scores of the VABS-II domains: ABC, Communication, Daily Living Skills, Socialization, and Motor Skills
    . Change from baseline in age equivalents, developmental quotients, and T-scores for the subtests of the DAS-II: Verbal Comprehension, Picture Similarities, Naming Vocabulary,
    Pattern Construction, Matrices, and Copying for the DAS-II/Early Years and Recall of Designs, Word Definitions, Pattern Construction, Matrices, Verbal Similarities, and Sequential and Quantitative Reasoning for the DAS-II/BAS-II/School School Years
    . Change from baseline in age equivalents, developmental quotients, and V-scale scores of the VABS-II subdomains: Communication (Receptive, Expressive, Written), Daily Living Skills
    (Personal, Domestic, Community), Socialization (Interpersonal Relationships, Play and Leisure Time, Coping Skills), Motor Skills (Gross, Fine)
    . Change from baseline in the V-scale scores and observed maladaptive levels of the VABS-II Maladaptive Behavior Index and its subscales (Internalizing, Externalizing)
    . Change from baseline in Brain Total Intracranial Volume, Brain Total Tissue Volume, Brain Total White Matter, Brain Total Gray Matter, and Total CSF Volume

    Health Economics and Outcomes Research Endpoints:
    . Health status dimensions as obtained by the EQ-5D questionnaire
    . Health care resource utilization will be assessed using the Healthcare Resource Utilization and Impact Questionnaire
    . Functional status as obtained by the HS-FOCUS form

    Pharmacokinetic and Pharmacodynamic Endpoints:
    . Serum concentration of idursulfase and serum PK parameters after IT administration
    . CSF concentration of idursulfase
    . Change from baseline in the concentration of GAG in CSF
    . Change from baseline in the concentration of GAG in urine

    SOPH-A-PORT Mini S Assessments:
    The SOPH-A-PORT Mini S device will be evaluated using assessments of device implantation, device function, device longevity, and AEs associated with the implant surgery or device. These data will be collected on the patient?s eCRF from the time of initial implantation.
    . Variación con respecto al momento basal de las puntuaciones normalizadas en las áreas agrupadas de la escala DAS-II/BAS-II: GCA, combinado verbal, no verbal, espacial y no verbal especial (SNC) o equivalentes de edad y cociente de desarrollo (CD) a partir de los dominios de la escala BSID-III: cognitivo y lenguaje.
    . Variación con respecto al momento basal de las puntuaciones normalizadas en los dominios VABS-II: ABC, comunicación, destrezas para la vida cotidiana, socialización y habilidades motoras.
    . Variación con respecto al momento basal de los equivalentes de edad, cociente de desarrollo y puntuaciones T en las subpruebas de la escala DAS-II: comprensión verbal, semejanzas de imágenes, vocabulario denominativo, construcción de modelos, matrices y copia en relación con la escala DAS-II/primeros años y recuerdo de diseños, definición de palabras, construcción de modelos, matrices, semejanzas verbales y razonamiento secuencial y cuantitativo en relación con la escala DAS-II/edad escolar.
    . Variación con respecto al momento basal de los equivalentes de edad, cociente de desarrollo y puntuaciones en la escala V de los subdominios VABS-II: comunicación (receptiva, expresiva, por escrito), destrezas para la vida cotidiana (personal, doméstica, comunitaria), socialización (relaciones interpersonales, tiempo de juego y ocio, capacidad de adaptación) y habilidades motoras (groseras, finas).
    . Variación con respecto al momento basal de las puntuaciones en la escala V y el grado de inadaptación observado del índice de conducta inadaptada VABS-II y sus subescalas (interiorización, exteriorización).
    . Variación con respecto al momento basal del volumen cerebral intracraneal total, volumen total de tejido cerebral, sustancia blanca cerebral total, sustancia gris cerebral total y volumen total de LCR.

    Criterios de valoración de economía sanitaria e investigación de resultados:
    . Dimensiones del estado de salud obtenidas mediante el cuestionario EQ-5D.
    . Utilización de recursos sanitarios evaluada mediante el Cuestionario de utilización de recursos sanitarios y repercusión.
    . Estado funcional obtenido mediante el cuestionario HS-FOCUS.

    Criterios de valoración farmacocinéticos y farmacodinámicos:
    . Concentración sérica de idursulfasa y parámetros FC en suero tras la administración intratecal.
    . Concentración de idursulfasa en LCR.
    . Variación de la concentración de glucosaminoglucanos (GAG) en LCR con respecto al momento basal.
    . Variación de la concentración de GAG en orina con respecto al momento basal.

    Evaluaciones del dispositivo SOPH-A-PORT Mini S:
    El dispositivo SOPH-A-PORT Mini S se valorará mediante evaluaciones de la implantación del dispositivo, el funcionamiento del dispositivo, la longevidad del dispositivo y los AA asociados a la intervención de implante o el dispositivo. Estos datos se recopilarán en el CRDe del paciente desde el momento de la implantación inicial.
    E.5.2.1Timepoint(s) of evaluation of this end point
    *Initial Treatment Phase: Patients not treated in Study HGT-HIT-094 undergo neurodevelopmental assessment at week 16, 28, 40; EQ-5D at week 4, 16, 28 and 40; Health Resource Utilization and Impact Questionnaire and HS-FOCUS at baseline, week 4, 16, 28 and 40.
    *Extended Treatment Phase:
    - Patients not treated in Study HGT-HIT-094 undergo all assessment above at week 52 and thereafter every 24 weeks.
    - Patients treated in Study HGT-HIT-094 do not undergo neurodevelopmental assessment and EQ-5D at week 52 but thereafter every 24 weeks. Health Resource Utilization and Impact Questionnaire and HS-FOCUS will be performed at week 52 and thereafter every 24 weeks.

    For all other endpoints please refer to Appendix 1 to 7 of the protocol.
    *Tratamiento Inicial: Pacientes no tratados en el estudio HGT-HIT-094 sometidos a evaluaciones del desarrollo neurológico en la sem. 16, 28, 40; EQ-5D en la sem 4, 16, 28 y 40; Cuestionario de utilización de recursos sanitarios y repercusión y HS-FOCUS basal, sem 4, 16, 28 y 40.
    * Tratamiento Ampliado:
    - Pacientes no tratados en el estudio HGT-HIT-094 sometidos a todas las evaluaciones por encima de la sem 52 y luego cada 24 semanas.
    - Pacientes tratados en el estudio HGT-HIT-094 no sometidos a evaluaciones del desarrollo neurológico y EQ-5D en la sem 52 pero luego cada 24 semanas. Cuestionario de utilización de recursos sanitarios y repercusión y HS-FOCUS serán realizados en la sem 52 y luego cada 24 semanas.

    Para todas las demás pruebas consultar el Apéndice 1 a 7 del protocolo.
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Quality of Life and Health Economics
    Calidad de Vida y valoración de Economía sanitaria
    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) 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 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 EEA2
    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
    Canada
    Colombia
    Mexico
    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
    The study will conclude after the last patient has completed his last visit.
    El estudio finalizará después de la última visita del último paciente.
    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: 42
    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: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 36
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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
    minors
    menores
    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 8
    F.4.2.2In the whole clinical trial 42
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    Ninguno
    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 Decision2015-04-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-02-25
    P. End of Trial
    P.End of Trial StatusOngoing
    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