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    Summary
    EudraCT Number:2017-002404-28
    Sponsor's Protocol Code Number:111-302
    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:2018-11-23
    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 number2017-002404-28
    A.3Full title of the trial
    A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia
    Estudio de extensión de fase III, abierto, para evaluar la seguridad y la eficacia a largo plazo de BMN 111 en pacientes pediátricos con acondroplasia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia
    Estudio de extensión de fase III, para evaluar la seguridad y la eficacia a largo plazo de BMN 111 en pacientes pediátricos con acondroplasia
    A.4.1Sponsor's protocol code number111-302
    A.5.4Other Identifiers
    Name:INDNumber:111299
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/379/2017
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBioMarin Pharmaceutical 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 supportBioMarin Pharmaceutical Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBioMarin Pharmaceutical Inc.
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street Address105 Digital Drive
    B.5.3.2Town/ cityNovato
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@bmrn.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/3/12/1094
    D.3 Description of the IMP
    D.3.1Product namemodified recombinant human C-type natriuretic peptide
    D.3.2Product code BMN 111
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNvosoritide
    D.3.9.1CAS number 1480724-61-5
    D.3.9.2Current sponsor codeBMN 111
    D.3.9.3Other descriptive nameMODIFIED RHCNP
    D.3.9.4EV Substance CodeSUB120857
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.8
    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 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 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 No
    D.IMP: 2
    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/3/12/1094
    D.3 Description of the IMP
    D.3.1Product namemodified recombinant human C-type natriuretic peptide
    D.3.2Product code BMN 111
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNvosoritide
    D.3.9.1CAS number 1480724-61-5
    D.3.9.2Current sponsor codeBMN 111
    D.3.9.3Other descriptive nameMODIFIED RHCNP
    D.3.9.4EV Substance CodeSUB120857
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin 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 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 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 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
    Achondroplasia
    Acondroplasia
    E.1.1.1Medical condition in easily understood language
    Dwarfism
    Enanismo
    E.1.1.2Therapeutic area Body processes [G] - Bones and nerves physological processes [G11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10000452
    E.1.2Term Achondroplasia
    E.1.2System Organ Class 100000004850
    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 extension study is to:
    - Evaluate the long-term safety, tolerability, and efficacy for growth in children with ACH treated with BMN 111
    El objetivo principal de este estudio de extensión es:
    - Evaluar la seguridad, tolerabilidad y eficacia a largo plazo para el crecimiento en niños con ACH tratados con BMN 111
    E.2.2Secondary objectives of the trial
    The secondary objectives of the study are to:
    - Evaluate the pharmacokinetics of BMN 111
    - Evaluate changes in health-related quality of life as measured by quality of life questionnaires
    - Evaluate changes in daily activity performance as measured by activity of daily living (ADL) questionnaires
    - Evaluate change from baseline in bone metabolism biomarkers
    Los objetivos secundarios del estudio son:
    • Evaluar la farmacocinética (FC) de BMN 111
    • Evaluar los cambios en la calidad de vida relacionada con la salud, medidos a través de cuestionarios de calidad de vida
    • Evaluar los cambios en el desempeño de las actividades cotidianas, medidos a través de cuestionarios de hábitos normales de vida (HNV)
    • Evaluar el cambio respecto al periodo basal en los biomarcadores de metabolismo óseo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Must have completed Study 111-301 (eligible to enroll in 302 within 3 months after completing 111-301 at the discretion of the principal investigator and medical monitor).
    2. Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, subjects under the age of majority are willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure. Subjects who reach the age of majority in their country while the study is ongoing will be asked to provide their own written consent again upon reaching the legal age of majority.
    3. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline Visit and be willing to have additional pregnancy tests during the study
    4. If sexually active, are willing to use contraception as specified in section 9.3.3 of the protocol,
    5. Are willing and able to perform all study procedures as physically possible
    1. Haber completado el estudio 111 301 (aptos para participar en 302 en un plazo de 3 meses después de completar 111-301 a discreción del investigador principal y del supervisor médico).
    2. Los progenitores o tutores deben estar dispuestos y ser capaces de proporcionar y firmar un consentimiento informado por escrito después de explicarles la naturaleza del estudio y antes de llevar a cabo cualquier
    procedimiento relacionado con la investigación. Además, los pacientes menores de edad deben estar dispuestos y ser capaces de proporcionar un asentimiento por escrito (si es necesario según la normativa local o el
    comité de ética/CEIC) después de explicarles la naturaleza del estudio y antes de llevar a cabo cualquier procedimiento relacionado con la investigación. A los sujetos que vayan a llegar a la mayoría de edad en
    su país durante su participación en el estudio, se les pedirá un nuevo consentimiento por escrito una vez que alcancen la mayoría de edad legal.
    3. Las mujeres ≥ 10 años de edad o que hayan tenido la primera menstruación deben tener un resultado negativo en la prueba de embarazo en la visita inicial y deben estar dispuestas a realizarse más
    pruebas de embarazo durante el estudio
    4. Si son sexualmente activas, deben estar dispuestas a usar métodos anticonceptivos conforme a lo especificado en la Sección 9.3.3.
    5. Dispuestos y capaces de realizar todos los procedimientos del estudio según sus posibilidades físicas
    E.4Principal exclusion criteria
    1. Permanently discontinued BMN 111 or placebo prior to completion of Study 111-301
    2. Have a clinically significant finding or arrhythmia on Baseline electrocardiogram (ECG) that indicates abnormal cardiac function or conduction or QTc-F > 450 msec
    3. Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months or of growth plate closure (proximal tibia, distal femur) through bilateral lower extremity X-rays including both AP and lateral views
    4. Require any investigational agent prior to completion of study period
    5. Current therapy with antihypertensive medications, angiotensin-converting enzyme (ACE) inhibitors, angiotensin II receptor blockers, diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides, systemic anticholinergic agents, GnRH agonists, any medication that may impair or enhance compensatory tachycardia, diuretics, or other drugs known to alter renal or tubular function
    6. Planned or expected to have limb-lengthening surgery during the study period.
    7. Planned or expected bone-related surgery (ie. surgery involving disruption of bone cortex (excluding tooth extraction), during the study period.
    8. Have had a fracture of the long bones or spine within 6 months prior to 111-302 baseline visit
    9. Pregnant or breastfeeding at the Baseline Visit or planning to become pregnant (self or partner) at any time during the study
    10. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason
    11. Have a condition or circumstance that, in the view of the investigator, places the subject at high risk for poor treatment compliance or for not completing the study
    1. Suspensión permanente de BMN 111 o placebo antes de la conclusion del estudio 111-301
    2. Presentar un resultado o arritmia clínicamente significativos en el elelectrocardiograma (ECG) de la selección que indique una function cardíaca o conducción anómalas o un QTc-F > 450 ms
    3. Evidencia de velocidad de crecimiento reducida (AGV < 1,5 cm/año) valorada a lo largo de un periodo de al menos 6 meses o de cierre de la placa de crecimiento (tibia proximal, fémur distal) detectado por medio
    de una radiografía bilateral de extremidades inferiores con vistas anteroposterior y lateral
    4. Necesidad de algún fármaco en fase de investigación antes de finalizar el periodo de estudio
    5. Tratamiento actual con medicamentos antihipertensivos, inhibidores de la enzima convertidora de angiotensina (ECA), bloqueantes del receptor de la angiotensina II, diuréticos, betabloqueantes, bloqueantes
    de los canales de calcio, glucósidos cardíacos, agentes anticolinérgicos sistémicos, agonistas de GnRH, cualquier medicamento que pueda impedir o potenciar la taquicardia compensatoria, diuréticos u otros fármacos que se sepa que alteran la función renal o tubular (Tabla 9.3.2.1)
    6. Cirugía de alargamiento de las extremidades programada o prevista durante el periodo del estudio.
    7. Cirugía relacionada con los huesos programada o prevista (es decir, cirugía que implique la alteración de la corteza ósea [except extracciones dentales]) durante el periodo del estudio.
    8. Haber sufrido una fractura de huesos largos o columna en los 6 meses anteriores a la visita inicial de 111-302
    9. Estar embarazada o en periodo de lactancia en la visita inicial o tener previsto quedarse embarazada (la paciente o la pareja del paciente) en cualquier momento durante el estudio
    10. Enfermedad o afección concurrente que, en opinión del investigador, podría interferir con la participación en el estudio o las evaluaciones de seguridad, por cualquier motivo
    11. Presentar una afección o encontrarse en una circunstancia que, en opinión del investigador, sitúe al paciente en una situación de riesgo elevado de un mal cumplimiento terapéutico o de no completar el estudio
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the change from baseline in annualized growth velocity (AGV).

    Safety will be evaluated by the incidence of AEs, SAEs, and clinically significant changes in vital signs, physical examination, Tanner stage, ECG, echocardiogram, bone morphology/quality assessed by X-rays/DXA, and laboratory test results (urinalysis, chemistry, hematology). Additionally, imaging, hip monitoring, immunogenicity, salivary cortisol, serum prolactin, and FSH/LH levels (FSH/LH will be monitored for all subjects >8 years of age, and for subjects at Tanner stage 2, whichever is earlier); and anxiety, motor and cognitive assessment with the Childhood Behavioral Checklist (CBCL) and WeeFIM will be utilized for safety-related reviews and analysis. Additional assessments will be conducted to evaluate changes from baseline in bone metabolism and BMN 111 activity biomarkers.
    El criterio de valoración de la eficacia principal es el cambio de la velocidad de crecimiento anualizada (AGV) desde el periodo inicial. Se evaluará la seguridad en función de la incidencia de AA, AAG y cambios clínicamente significativos en constantes vitales, exploración física, etapa de Tanner, ECG, ecocardiograma, calidad/morfología ósea evaluada por radiografía/DXA y resultados de pruebas de laboratorio (bioquímica clínica, hematología y análisis de orina). Además, se realizarán pruebas de diagnóstico por imagen, supervisión de cadera, inmunogenicidad, cortisol salival, prolactina en suero y niveles de FSH/HL (FSH/HL se supervisará para todos los sujetos >8 años y para los sujetos en la etapa de Tanner 2, lo que sea anterior), así como evaluaciones de ansiedad, motoras y cognitivas con Listas de verificación de la conducta infantil (CBCL) y se utilizará WeeFIM para las revisiones y análisis relacionados con la seguridad. También se llevarán a cabo evaluaciones de los cambios respecto al periodo basal en el metabolismo óseo y los biomarcadores de actividad de BMN 111.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Anthropometric measurements: 111-301 Week52/302 BL, Weeks 13, 26,39, 52, 65, 78, 91, 104, every 26 weeks up to 260

    Clinical labs; Vital signs/AEs; Physical exam; ECG; Anti-BMN 111 immunogenicity; bone metabolism biomarkers: 111-301 Week 52/302 BL, Day 1, Weeks 13, 26, 39, 52, 65, 78, 91, 104, every 26 weeks up to 260

    X-Ray/DXA: 111-301 Week 52/302 BL, Weeks 52, 104, 156, 208, 260

    Clinical hip assessment: 111-301 Week 52/302 BL, Weeks 26, 52, 78, 104, every 26 weeks up to 260

    Salivary cortisol; serum prolactin; FSH/LH: 111-301 Week 52/302 BL, 26, 52, 78, 26 weeks up to week 260

    CBCL and WeeFIM: 111-301 Week 52/302 BL, 26, 52, 78, 104, every 26 weeks up to 260

    BMN 111 activity biomarkers: 111-301 Week 52/302 BL, Day1, Weeks 26, 52, 78, 104, every 26 weeks up to 260
    Mediciones antropométricas: 111-301 S. 52/302 BL, S. 13, 26, 39, 52, 65, 78, 91, 104, cada 26 s. hasta la 260. Análisis de laboratorio clínicos; const. vitales/efect. adversos; examen físico; ECG; inmunogenicidad anti-BMN 111; biomarcadores de metabolismo óseo: 111-301 S. 52/302 BL, D1, S. 13, 26, 39, 52, 65, 78, 91, 104, cada 26 s. hasta la 260. Radiografía/DXA: 111-301 S. 52/302 BL, S. 52, 104, 156, 208, 260. Eval. clínica de la cadera: 111-301 S. 52/302 BL, S. 26, 52, 78, 104, cada 26 s. hasta la 260. Cortisol en saliva; prolactina en suero; FSH/HL: 111-301 S. 52/302 BL, 26, 52, 78, 26 s. hasta la s. 260. CBCL y WeeFIM: 111-301 S. 52/302 BL, 26, 52, 78, 104, cada 26 s. hasta 260. Biomarcadores de actividad de BMN 111: 111-301 S. 52/302 BL, D1, S. 26, 52, 78, 104, cada 26 s. hasta la 260
    E.5.2Secondary end point(s)
    The secondary efficacy endpoints include the change from baseline in height Z-score and the change from baseline in upper:lower body segment ratio.

    PK sampling will be carried out over the study period.

    Health-related Quality of Life (HRQoL) and ADL questionnaires will be administered to assess quality of life and daily activities performance of study subjects.
    Los criterios de valoración de la eficacia secundarios incluyen el cambio desde el periodo inicial de la puntuación Z de estatura, así como el cambio desde el periodo inicial de la relación segmento superior:inferior
    del cuerpo. Se tomarán muestras para FC a lo largo del periodo del estudio. Se administrarán los cuestionarios de Calidad de vida relacionada con la salud (CdVRS) y HNV para evaluar la calidad de vida y el desempeño de
    las actividades cotidianas de los sujetos del estudio.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Anthropometric measurements: 111-301 Week 52/302 BL, Weeks 13, 26, 39, 52, 65, 78, 91, 104, every 26 weeks up to Week 260

    PK: 301 Week 52/302 BL, Day 1, Week 26, Week 52, Week 78, Week 104, every 26 weeks up to Week 260

    Health-related Quality of Life (HRQoL) and ADL questionnaires: 111-301 Week 52/302 BL, Week 26, Week 52, Week 78, Week 104, every 26 weeks up to 260
    Mediciones antropométricas: 111-301 Semana 52/302 BL, Semanas 13, 26, 39, 52, 65, 78, 91, 104, cada 26 semanas hasta la 260
    FC: 301 Semana 52/302 BL, Día 1, Semana 26, Semana 52, Semana 78, Semana 104, cada 26 semanas hasta la semana 260
    Cuestionarios de calidad de vida relacionada con la salud (CdVRS) y HNV: 111-301 Semana 52/302 BL, Semana 26, Semana 52, Semana 78, Semana 104, cada 26 semanas hasta la 260
    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 No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 EEA7
    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
    Germany
    Japan
    Spain
    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
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    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: 108
    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: 70
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 38
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    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 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
    Paediatric subjects who are below age of majority will sign assents and have parents or guardians sign consents
    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 39
    F.4.2.2In the whole clinical trial 110
    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 Decision2018-11-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-09-20
    P. End of Trial
    P.End of Trial StatusOngoing
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