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   36111   clinical trials with a EudraCT protocol, of which   5936   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).
     
    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:2016-002066-32
    Sponsor's Protocol Code Number:BEB-13
    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:2017-03-10
    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 number2016-002066-32
    A.3Full title of the trial
    Double-blind, Randomised, Vehicle-controlled, Phase III, Efficacy and Safety Study with 24-month Open-label Follow up of Oleogel-S10 in Patients with Inherited Epidermolysis Bullosa
    Estudio de fase III, doble ciego, aleatorizado, controlado con vehículo y con un seguimiento abierto de 24 meses sobre la eficacia y la seguridad de Oleogel-S10 en pacientes con epidermólisis ampollosa hereditaria.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Clinical Trial which compare the safety and efficacy of a wound gel, the study treatment, or a sunflower oil-based vehicle gel in patients with Inherited Epidermolysis Bullosa (EB)
    Un ensayo clínico que compara la seguridad y la eficacia de un gel para las lesiones, el tratamiento del estudio, o un vehículo que será un gel a base de aceite de girasol en pacientes con epidermólisis ampollosa (EA) hereditaria.
    A.3.2Name or abbreviated title of the trial where available
    EASE study
    Estudio EASE
    A.4.1Sponsor's protocol code numberBEB-13
    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 SponsorAmryt Research Limited
    B.1.3.4CountryIreland
    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 supportAmryt Research Limited
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmryt Research Limited
    B.5.2Functional name of contact pointTobias Zahn
    B.5.3 Address:
    B.5.3.1Street AddressFitzwilliam Hall, Fitzwilliam Place
    B.5.3.2Town/ cityDublin 2
    B.5.3.3Post codeNA
    B.5.3.4CountryIreland
    B.5.4Telephone number0034916307447
    B.5.6E-mailtobias.zahn@amrytpharma.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 Episalvan gel
    D.2.1.1.2Name of the Marketing Authorisation holderBirken AG
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameOleogel-S10
    D.3.4Pharmaceutical form Gel
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPCutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBirch bark extract
    D.3.9.1CAS number 1640971-03-4
    D.3.9.2Current sponsor codeDry extract from betulae cortex
    D.3.9.3Other descriptive nameBIRCH BARK EXTRACT
    D.3.9.4EV Substance CodeSUB180085
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    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 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 Yes
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboGel
    D.8.4Route of administration of the placeboCutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Inherited Epidermolysis Bullosa
    Epidermólisis ampollosa hereditaria
    E.1.1.1Medical condition in easily understood language
    Illness called Epidermolysis bullosa (EB) that makes skin blister and have open sores and wounds
    Enfermedad denominada Epidermólisis ampollosa (EA) que produce ampollas en la piel y tiene llagas y lesiones abiertas.
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.1
    E.1.2Level PT
    E.1.2Classification code 10014989
    E.1.2Term Epidermolysis bullosa
    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 the double-blind phase is to compare the efficacy of Oleogel-S10 (treatment arm A) with vehicle (treatment arm B) in the promotion of healing of EB partial thickness wounds. This will be assessed as evidenced by the incidence of the first complete closure of the EB target wound (defined as EB partial thickness wound of 10 cm2 to 50 cm2 in size aged >=21 days in any subtype of inherited EB) within 45+-7 days of treatment).
    El objetivo principal de la fase con doble ciego es comparar la eficacia de Oleogel-S10 (grupo de tratamiento A) con la del vehículo (grupo de tratamiento B) en el estímulo de la cicatrización de lesiones ampollosas de espesor parcial. Esto se evaluará en función de la incidencia del primer cierre completo de la lesión ampollosa diana (definida como lesión ampollosa de espesor parcial de 10 cm2 a 50 cm2 de tamaño y una antigüedad >=21 días en cualquier subtipo de epidermólisis ampollosa hereditaria) en el plazo de 45+-7 días de tratamiento.
    E.2.2Secondary objectives of the trial
    1/ Compare the efficacy of IMP with placebo as evidenced by several criteria described in the protocol
    2/ Compare the safety of IMP with placebo as evidenced by the incidence, severity, and relatedness of AEs and based on laboratory assessments
    3/ Compare the tolerability of IMP with placebo
    4/ Assess betulin exposure
    1/ Comparar la eficacia del producto en investigación con placebo en función de varios criterios descritos en el protocolo.
    2/ Comparar la seguridad del producto en investigación con placebo en función de la incidencia, la gravedad y el grado de relación de los acontecimientos adversos (AA), basándose en evaluaciones de laboratorio.
    3/ Comparar la tolerabilidad del producto en investigación con placebo
    4/Evaluar la exposición a la betulina.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A patient will be eligible for study participation only if all of the following criteria apply:
    1. Male and female patients with any subtype of inherited EB including EBS, JEB, DEB, and Kindler syndrome age >=4 years [Note: Children <4 years of age may be included only after confirmation by the Independent Data Monitoring Committee (IDMC) upon review of the safety and bioanalytical data at the interim safety review stage].
    2. Patients with an EB target wound (i.e., EB partial thickness wound of 10 cm2 to 50 cm2 in size aged >=21 days)
    3. Patient and/or his/her legal representative has/have been informed, has/have read and understood the patient information/informed consent form, and has/have given written informed consent
    4. Patient and/or his/her legal representative must be able and willing to follow study procedures and instructions
    Un/a paciente podrá participar en este estudio si cumple todos los criterios siguientes:
    1. Hombres y mujeres con cualquier subtipo de EA hereditaria, como EAS, EAU, EAD y síndrome de Kindler >=4 años [nótese que los niños y las niñas <4 años podrán incluirse únicamente con posterioridad a la confirmación por parte del Comité Independiente de Supervisión de los Datos (CISD), tras la revisión de los datos bioanalíticos y sobre la seguridad en la etapa de revisión intermedia de la seguridad];
    2. Pacientes con una lesión ampollosa diana (es decir, una lesión ampollosa de espesor parcial de 10 cm2 a 50 cm2 de tamaño y una antigüedad >=21 días);
    3. El/la paciente (y/o su representante legal) ha sido informado, ha leído y comprendido la hoja de información para pacientes y el documento de consentimiento informado, y ha otorgado su consentimiento informado por escrito;
    4. El/la paciente (y/o su representante legal) deberá ser capaz a seguir las instrucciones y los procedimientos del estudio y estar dispuesto a ello.
    E.4Principal exclusion criteria
    A patient will not be eligible to participate in this study if any of the following criteria apply:
    1. EB target wound with clinical signs of local infection
    2. Use of systemic antibiotics for wound-related infections within 7 days prior to enrolment
    3. Administration of systemic or topical steroids (except for inhaled, ophthalmic or topical applications, such as budesonide suspension for oesophageal strictures [e.g., Pulmicort respules® 0.25 mg/2 mL or 0.5 mg/2 mL]) within 30 days before enrolment
    4. Immunosuppressive therapy or cytotoxic chemotherapy within 60 days prior to enrolment
    5. Patient has undergone stem cell transplant or gene therapy for the treatment of inherited EB
    6. Current and/or former malignancy including basal cell carcinomas and squamous cell carcinomas
    7. Enrolment in any interventional study or treated with any investigational drug for any disease within 4 weeks prior to study entry
    8. Factors present in the patient and/or his/her legal representative that could interfere with study compliance such as inability to attend scheduled study visits or compliance with home dressing changes
    9. Pregnant or nursing women and women of childbearing potential including postmenarchal female adolescents not willing to use an effective form of birth control with failure rates <1% per year (e.g., implant, injectable, combined oral contraceptive, intrauterine contraceptive device, sexual abstinence, vasectomised partner) during participation in the study (and at least 3 months thereafter)
    10.Patient is a member of the investigational team or his/her immediate family
    11.Patient lives in the same household as a study participant.
    Un/a paciente no podrá participar en este estudio si cumple alguno de los siguientes criterios:
    1. la lesión ampollosa diana presenta indicios clínicos de infección local;
    2. uso de antibióticos sistémicos para tratar las infecciones relacionadas con las lesiones dentro de los 7 días previos a la inclusión;
    3. administración de corticoesteroides tópicos o sistémicos (excepto inhalados, oftálmicos o tópicos, como la suspensión de budesonida para la estenosis esofágica [p. ej., Pulmicort respules® 0,25 mg/2 ml o 0,5 mg/2 ml]) dentro de los 30 días previos a la inclusión;
    4. tratamiento inmunodepresor o quimioterapia citotóxica dentro de los 60 días previos a la inclusión;
    5. el/la paciente ha sido sometido/a a un trasplante de células madre o a genoterapia para tratar la EA hereditaria;
    6. neoplasia maligna actual o anterior, incluidos los carcinomas basocelulares y espinocelulares;
    7. inclusión en cualquier estudio de intervención o tratamiento con cualquier medicamento en fase de investigación para cualquier enfermedad dentro de las 4 semanas previas al comienzo de este estudio;
    8. el/la paciente (y/o su representante legal) presenta factores que podrían interferir con el cumplimiento de sus obligaciones en el marco del estudio tales como la incapacidad para asistir a las visitas del estudio programadas o el cumplimiento de los cambios de apósito en el domicilio;
    9. mujeres embarazadas o en estado de lactancia y mujeres fértiles, incluidas las adolescentes posmenárquicas, que no estén dispuestas a usar un anticonceptivo eficaz con tasas de fracaso <1 % al año (por ejemplo, anticonceptivos implantables, inyectables, orales combinados, dispositivo intrauterino, abstinencia sexual o pareja vasectomizada) durante su participación en el estudio (y al menos durante 3 meses después de su finalización);
    10. el paciente es un familiar cercano o un miembro del equipo investigador;
    11. el paciente cohabita con un participante del estudio.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patients with first complete closure of the EB target wound (defined as EB partial thickness wound of 10 cm2 to 50 cm2 in size aged >=21 days in any subtype of inherited EB) within 45±7 days of treatment with Oleogel-S10 compared to vehicle based on clinical assessment by the investigator (the wound will be rated as “closed” at first appearance of complete reepithelialisation without drainage confirmed by a second observation within the following week)
    Proporción de pacientes que presente un primer cierre completo de la lesión ampollosa diana (definida como una lesión ampollosa de espesor parcial de 10 cm2 a 50 cm2 de tamaño y una antigüedad >=21 días en cualquier subtipo de epidermólisis ampollosa hereditaria) en un plazo de 45±7 días de tratamiento con Oleogel-S10 en comparación con el vehículo, basada en la evaluación clínica por parte del investigador (la lesión se clasificará como «cerrada» cuando se observe por primera vez una completa reepitelización sin supuración confirmada por una segunda observación en el plazo de la semana siguiente).
    E.5.1.1Timepoint(s) of evaluation of this end point
    See section E.5.1
    Ver sección E.5.1
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints
    • Time to first complete closure of the EB target wound as evidenced by clinical assessment until D90+-7.
    • Proportion of patients with first complete closure of the EB target wound within D14+-5, D30+-7, D60+-7, and D90+-7 based on clinical assessment by the investigator
    • Proportion of patients with first complete closure of the EB target wound within at D7+-5, D14+-5, D30+-7, D45+-7, D60+-7, and D90+-7 based on patient assessment
    • Proportion of patients with first complete closure of the EB target wound at D7+-5, D14+-5, D30+-7, D45+-7, D60+-7, and D90+-7 based on blinded evaluation of photographs
    • Percentage change from baseline (D0) in EB target wound size as evidenced by blinded evaluation of photographs taken at D7+-5, D14+-5, D30+-7, D45+-7, D60+-7, and D90+-7
    • Change from baseline (D0) in total body wound burden as evidenced by clinical assessment using Section I (assessment of the skin except for the anogenital region) of the ‘EB Disease Activity and Scarring Index’ (EBDASI) at D30+-7, D60+-7, and D90+-7
    • Change from baseline (D0) in body surface area percentage (BSAP) of TBSA affected by EB partial thickness wounds as evidenced by clinical assessment based on the ‘Lund and Browder’ chart at D30+-7, D60+-7, and D90+-7
    • The incidence and maximum severity of wound infection between baseline (D0) and D90+-7 as evidenced by AEs and/or use of topical and/or systemic antibiotics (related to wound infection)
    • Change from baseline (D0) in ‘background’ pain using the ‘Face, Legs, Activity, Cry, Consolability’ (FLACC) scale in patients <4 years of age and the ‘Wong-Baker FACES® Pain Rating Scale’ in patients >=4 years of age before wound dressing changes at D7+-5, D14+-5, D30+-7, D45+-7, D60+-7, and D90+-7
    • Change from baseline (D0) in ‘procedural’ pain using the FLACC scale in patients <4 years of age and the ‘Wong-Baker FACES® Pain Rating Scale’ in patients >=4 years of age after wound dressing changes at D7+-5, D14+-5, D30+-7, D45+-7, D60+-7, and D90+-7
    • Change from baseline (D0) in itching using the ‘Itch Man Scale’ in patients >=4 years and up to 13 years of age and the ‘Leuven Itch Scale’ in patients >=14 years of age before wound dressing changes at D7+-5, D30+-7, D60+-7, and D90+-7
    • Change from baseline in impact of wounds on sleep (in patients >=14 years of age) as measured by differences in 11-point Likert scales at D7+-5, D30+-7, D60+-7, and D90+-7
    • The number of days missed from school or from work due to EB as reported by patients at D0, D14+-5, D30+-7, D45+-7, D60+-7, and D90+-7
    • Evaluation of the treatment response (in patients >=14 years of age) using the Treatment Satisfaction Questionnaire for Medication (TSQM), Version 9 at D7+-5, D30+-7, D60+-7, and D90+-7
    Safety endpoints
    • Incidence, severity, and relatedness of AEs
    • Local tolerability as judged by the investigator
    • Safety laboratory data
    • Systemic exposure to betulin, if consent provided
    Criterios de valoración secundarios de la eficacia
    • Tiempo transcurrido hasta el cierre completo de la lesión ampollosa diana, en función de la evaluación clínica hasta el D90+-7.
    • Proporción de pacientes que presente un primer cierre completo de la lesión ampollosa diana los días D14+-5, D30+-7, D60+-7 y D90+-7, basada en la evaluación clínica realizada por el investigador.
    • Proporción de pacientes que presente un primer cierre completo de la lesión ampollosa diana los días D7+-5, D14+-5, D30+-7, D45+-7, D60+-7 y D90+-7, basada en la evaluación del paciente.
    • Proporción de pacientes que presente un primer cierre completo de la lesión ampollosa diana los días D7+-5, D14+-5, D30+-7, D45+-7, D60+-7 y D90+-7, basada en la evaluación de fotografías enmascaradas.
    • Porcentaje de cambio respecto del valor basal (D0) en el tamaño de la lesión ampollosa diana, en función de la evaluación enmascarada de fotografías tomadas los días D7+-5, D14+-5, D30+-7, D45+-7, D60+-7 y D90+-7.
    • Cambio respecto del valor basal (D0) en la carga corporal total de lesiones, en función de la evaluación clínica empleando el apartado I (evaluación cutánea, excluyendo la región anogenital) del Índice de actividad y cicatrización de la epidermólisis ampollosa (EB Disease Activity and Scarring Index, EBDASI) los días D30+-7, D60+-7 y D90+-7.
    • Cambio respecto del valor basal (D0) en el porcentaje del área de superficie corporal (PASC) del ASCT afectada por lesiones ampollosas de espesor parcial, en función de la evaluación clínica basada en el gráfico de Lund y Browder los días D30+-7, D60+-7 y D90+-7.
    • Incidencia y gravedad máximas de la infección de la lesión entre el momento basal (D0) y el D90+-7, en función de los AA y/o del uso de antibióticos tópicos y/o sistémicos (relacionado con la infección de la lesión).
    • Cambio respecto del valor basal (D0) en el dolor de fondo utilizando la escala FLACC (Face, Legs, Activity, Cry, Consolability) en pacientes <4 años de edad y la escala de valoración del dolor Wong Baker FACES® en pacientes >=4 años antes de los cambios de apósito los días D7+-5, D14+-5, D30+-7, D45+-7, D60+-7 y D90+-7.
    • Cambio respecto del valor basal (D0) en el dolor procedimental utilizando la escala FLACC en pacientes <4 años y la escala de valoración del dolor Wong-Baker FACES® en pacientes >=4 años después de los cambios de apósito los días D7+-5, D14+-5, D30+-7, D45+-7, D60+-7 y D90+-7.
    • Cambio respecto del valor basal (D0) en la picazón usando la Itch Man Scale en pacientes>=4 años y de hasta 13 años de edad y la Leuven Itch Scale en pacientes >=14 años de edad antes de los cambios de apósito los días D7+-5, D30+-7, D60+-7 y D90+-7.
    • Cambio respecto del valor basal del efecto de las lesiones en el sueño (en pacientes >=14 años de edad), en función de las diferencias determinadas en las escalas de Likert de 11 puntos los días D7+-5, D30+-7, D60+-7 y D90+-7.
    • El número de días que no se ha asistido a la escuela o al trabajo debido a la epidermólisis ampollosa según lo comunicado por los pacientes los días D0, D14+-5, D30+-7, D45+-7, D60+-7 y D90+-7
    • Evaluación de la respuesta al tratamiento (en pacientes >=14 años de edad) usando el cuestionario TSQM (Treatment Satisfaction Questionnaire for Medication), versión 9, los días D7+-5, D30+-7, D60+-7 y D90+-7.
    Criterios de valoración de la seguridad
    • Incidencia, gravedad y grado de relación de los AA.
    • Tolerabilidad local determinada por el investigador.
    • Datos de laboratorio de seguridad.
    • Exposición general a la betulina (si se ha otorgado el consentimiento).
    E.5.2.1Timepoint(s) of evaluation of this end point
    See section E.5.2
    Ver sección E.5.2
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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) No
    E.8.2.2Placebo Yes
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Australia
    Austria
    Croatia
    France
    Germany
    Greece
    Ireland
    Israel
    Italy
    Mexico
    Spain
    Switzerland
    Turkey
    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
    LVLS estimated in June 2020
    Última visita de ultimo paciente estimada en junio 2020
    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 months2
    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 months2
    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: 82
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 41
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 41
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 82
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 110
    F.4.2.2In the whole clinical trial 164
    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
    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 Decision2017-03-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-03-10
    P. End of Trial
    P.End of Trial StatusOngoing
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2019 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA