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    Summary
    EudraCT Number:2015-003836-11
    Sponsor's Protocol Code Number:111-301
    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: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 number2015-003836-11
    A.3Full title of the trial
    A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia.
    Estudio en fase III, multicéntrico, aleatorizado, doble ciego y controlado con placebo para evaluar la eficacia y la seguridad de BMN 111 en niños con acondroplasia.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia
    Estudio en fase III para evaluar la eficacia y la seguridad de BMN 111 en niños con acondroplasia.
    A.4.1Sponsor's protocol code number111-301
    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 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 INNvorsoritide
    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 typerange
    D.3.10.3Concentration number0.2 to 10
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboLyophilisate and solvent for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    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 19.1
    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
    Evaluate change from baseline in mean annualized growth velocity at 52 weeks in subjects treated with BMN 111 compared with control subjects in the placebo group.
    Evaluar el cambio respecto al inicio en la velocidad de crecimiento anualizada a las 52 semanas en pacientes tratados con BMN 111 en comparación con pacientes control en el grupo de placebo.
    E.2.2Secondary objectives of the trial
    • Evaluate change from baseline in mean height Z-score in subjects treated with BMN 111 compared with control subjects in the placebo group at 52 weeks
    • Evaluate change from baseline in mean upper:lower segment body ratio in subjects treated with BMN 111 compared with control subjects in the placebo group at 52 weeks
    • Evaluate safety and tolerability of BMN 111 in children with ACH
    • Evaluate the pharmacokinetics of BMN 111
    •Evaluar el cambio respecto al inicio en la media de puntuación Z de la estatura en pacientes tratados con BMN 111 en comparación con pacientes control del grupo de placebo a las 52 semanas.
    •Evaluar el cambio respecto al inicio en la media de la relación segmento superior:inferior del cuerpo en pacientes tratados con BMN 111 en comparación con pacientes control del grupo de placebo a las 52 semanas.
    •Evaluar la seguridad y tolerabilidad de BMN 111 en niños con AC.
    •Evaluar la farmacocinética de BMN 111.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. 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 18 are willing and able to provide written assent (if required by local regulations or the IRB/EC) after the nature of the study has been explained and prior to performance of any research-related procedure.
    2. 5 to < 15 years old at study entry
    3. Have ACH, documented by clinical grounds and confirmed by genetic testing
    4. Have at least a 6-month period of pretreatment growth assessment in Study 111-901 immediately before study entry, and has one documented standing height at least 6 months (+/- 10 days) prior to the screening visit for Study 111-301
    5. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study
    6. If sexually active, willing to use a highly effective method of contraception while participating in the study
    7. Are ambulatory and able to stand without assistance
    8. Are willing and able to perform all study procedures as physically possible
    9. Caregivers are willing to administer daily injections to the subjects and complete the required training
    1.El/Los progenitor(es) o tutor(es) 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 18 años deben estar dispuestos y ser capaces de proporcionar un asentimiento por escrito (si es necesario según la normativa local o el comité de ética) después de explicarles la naturaleza del estudio y antes de llevar a cabo cualquier procedimiento relacionado con la investigación.
    2.De 5 a <15 años de edad en el momento de incorporarse al estudio.
    3.Tener AC documentada según criterios clínicos y confirmada mediante análisis genético.
    4.Tener un periodo mínimo de 6 meses de evaluación del crecimiento previa al tratamiento en el estudio 111-901, inmediatamente antes de la inclusión en este estudio, y tener una estatura en bipedestación documentada de al menos 6 meses (+/- 10 días) antes de la visita de selección del estudio 111-301.
    5.Las mujeres de ≥10 años de edad o que han tenido la primera menstruación deben tener un resultado negativo en la prueba de embarazo en la visita de selección y deben estar dispuestas a realizarse más pruebas de embarazo durante el estudio.
    6.Si son sexualmente activas, deben estar dispuestas a usar un método anticonceptivo muy eficaz mientras participen en el estudio.
    7.Ser capaces de deambular y de mantenerse en pie sin ayuda.
    8.Estar dispuestos y ser capaces de realizar todos los procedimientos del estudio según sus posibilidades físicas.
    9.Los cuidadores estarán dispuestos a administrar las inyecciones diarias a los pacientes y realizar la formación necesaria.
    E.4Principal exclusion criteria
    1. Have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia)
    2. Have any of the following: Hypothyroidism or hyperthyroidism; Insulin-requiring diabetes mellitus; Autoimmune inflammatory disease (including celiac disease, lupus (SLE), juvenile dermatomyositis, scleroderma, and others); Inflammatory bowel disease; Autonomic neuropathy
    3. Have a history of any of the following: Renal insufficiency defined as serum creatinine > 2 mg/dl; Chronic anemia; Baseline systolic blood pressure (BP) < 70 millimeters of mercury (mm Hg) or recurrent symptomatic hypotension (defined as episodes of low BP generally accompanied by symptoms i.e., dizziness, fainting) or recurrent symptomatic orthostatic hypotension; Cardiac or vascular disease (including Cardiac dysfunction (abnormal echocardiogram) at Screening Visit; Hypertrophic cardiomyopathy; Pulmonary hypertension; Congenital heart disease; Cerebrovascular disease; Aortic insufficiency or other clinically significant valvular dysfunction; Clinically significant atrial or ventricular arrhythmias)
    4. Have a clinically significant finding or arrhythmia on screening electrocardiogram (ECG) that indicates abnormal cardiac function or conduction or QTc-F > 450 msec
    5. Have an unstable condition likely to require surgical intervention during the study (including progressive cervical medullary compression or severe untreated sleep apnea)
    6. Evidence of growth plate closure (proximal tibia, distal femur) based on bilateral lower extremity X-rays (including both anterior-posterior [AP] and lateral views) obtained at the Screening visit
    7. Documented Vitamin D deficiency (concentration of blood 25-hydroxy-vitamin D <12 ng/mL or <30 nmol/L)
    8. Require any investigational agent prior to completion of study period9. Have received another investigational product or investigational medical device within 6 months before the Screening Visit
    10. Have used any investigational product or investigational medical device for the treatment of ACH or short stature at any time, including BMN 111
    11. 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 (Table 9.3.2.1)
    12. Have been treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or treatment greater than 6 months at any time
    13. Have had > 1 month treatment with oral corticosteroids (low-dose ongoing inhaled steroid for asthma, or intranasal steroids, are acceptable) in the previous 12 months
    14. Planned or expected to have limb-lengthening surgery during the study period. Subjects with previous limb-lengthening surgery may enroll if surgery occurred at least 18 months prior to the study and healing is complete without sequelae.
    15. Planned or expected bone-related surgery (ie. surgery involving disruption of bone cortex), during the study period. Subjects with previous bone-related surgery may enroll if surgery occurred at least 12 months prior to the study and healing is complete without sequelae.
    16. Have had a fracture of the long bones or spine within 6 months prior to screening
    17. Pregnant or breastfeeding at the Screening Visit or planning to become pregnant (self or partner) at any time during the study
    18. Have aspartate aminotransferase (AST) or alanine aminotransferase (ALT) at least 3x upper limit of normal (ULN) or total bilirubin at least 2x ULN (except for subjects with known history of Gilbert’s disease)
    19. History of severe untreated sleep apnea
    20. Have had new initiation of sleep apnea treatment (e.g. CPAP or sleep apnea-mitigating surgery) in the previous 2 months prior to screening
    21. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy
    22. Have known hypersensitivity to BMN 111 or its excipients
    23. Have a history of hip surgery or hip dysplasia atypical for achondroplasia subjects
    24. Have a history of clinically significant hip injury in the 30 days prior to screening
    25. History of slipped capital femoral epiphysis or avascular necrosis of the femoral head
    26. Are unable to lie flat when in prone position (needed for hip exam)
    27. Have abnormal findings on baseline clinical hip exam or imaging assessments that are determined to be clinically significant as determined by the investigator
    28. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason
    29. 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.Presentar hipocondroplasia o estatura baja por motivos distintos a la AC.
    2.Tener algo de lo siguiente-: Hipotiroidismo o hipertiroidismo, Diabetes mellitus que requiera tto con insulina, Enfermedad inflamatoria autoinmunitaria, Enfermedad intestinal inflamatoria, Neuropatía autónoma.
    3.Presentar antecedentes de cualquiera de los siguientes: Insuficiencia renal definida como una creatinina sérica >2 mg/dl, Anemia crónica, Tensión arterial (TA) sistólica al inicio <70 milímetros de mercurio (mm Hg) o hipotensión recurrente sintomática o hipotensión ortostática recurrente sintomática, Enfermedad cardíaca o vascular, incluidas las siguientes: Disfunción cardíaca en la visita de selección, Miocardiopatía hipertrófica, Hipertensión pulmonar, Cardiopatía congénita, Enfermedad cerebrovascular, Insuficiencia aórtica u otra disfunción valvular clínicamente significativa, Arritmias ventriculares o auriculares clínicamente significativas.
    4.Presentar un hallazgo o arritmia clínicamente significativos ECG de la selección que indique una función cardíaca o conducción anómalas o un QTc-F >450 ms.
    5.Tener una enfermedad inestable que probablemente requiera una intervención quirúrgica durante el studio.
    6.Indicios de cierre del cartílago de crecimiento en radiografías bilaterales de la extremidad inferior obtenidas en la visita de selección.
    7.Deficiencia documentada de vitamina D.
    8.Necesidad de algún agente en investigación antes de finalizar el periodo del estudio.
    9.Haber recibido otro producto en investigación o producto sanitario en investigación en los 6 meses previos a la visita de selección.
    10.Haber usado cualquier producto en investigación o producto sanitario en investigación destinado al tratamiento de la AC o la estatura baja en cualquier momento, incluido BMN 111.
    11.Tratamiento actual con medicamentos antihipertensivos, inhibidores de la enzima convertidora de angiotensina (ECA), antagonistas 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 u otros fármacos que se sepa que alteran la función renal o tubular (Tabla 9.3.2.1)
    12.Haber recibido tratamiento con hormona del crecimiento, factor de crecimiento análogo a la insulina 1 (IGF-1) o esteroides anabólicos en los 6 meses anteriores o como tratamiento de larga duración (>6 meses) en cualquier momento.
    13.Haber recibido tratamiento durante >1 mes con corticoesteroides orales en los 12 meses previos.
    14.Cirugía de alargamiento de las extremidades programada o prevista durante el periodo del estudio. Los pacientes con cirugía previa de alargamiento de las extremidades pueden participar si dicha cirugía ha tenido lugar por lo menos 18 meses antes del estudio y la curación es completa y sin secuelas.
    15.Cirugía relacionada con los huesos programada o prevista durante el periodo del estudio. Los pacientes con cirugía previa relacionada con los huesos pueden participar si dicha cirugía ha tenido lugar por lo menos 12 meses antes del estudio y la curación es completa y sin secuelas.
    16.Haber tenido una fractura de huesos largos o columna en los 6 meses anteriores a la selección.
    17.Estar embarazada o en periodo de lactancia en la visita de selección o tener previsto quedarse embarazada en cualquier momento durante el estudio.
    18.Tener un nivel de aspartato aminotransferasa (AST) o alanina aminotransferasa (ALT) de al menos 3 veces (x) el límite superior de la normalidad (LSN) o de bilirrubina total al menos 2 x LSN (excepto pacientes con antecedentes conocidos de enfermedad de Gilbert).
    19.Antecedentes de apnea del sueño grave no tratada.
    20.Haber comenzado un tratamiento nuevo para la apnea del sueño.
    21.Presencia actual de una neoplasia maligna, antecedentes de neoplasia maligna o realización de pruebas por una presunta neoplasia maligna en ese momento.
    22.Hipersensibilidad conocida a BMN 111 o sus excipientes.
    23.Antecedentes de cirugía de cadera o displasia de cadera atípica en pacientes con acondroplasia.
    24.Antecedentes de cirugía de cadera clínicamente significativa en los 30 días previos a la selección.
    25.Antecedentes de epifisiólisis de la cabeza femoral o necrosis avascular de la cabeza femoral.
    26.Incapacidad para tumbarse en decúbito prono.
    27.Presentar hallazgos anómalos en la exploración clínica de cadera o en las exploraciones por imagen en el inicio que el investigador determine como clínicamente significativas.
    28.Enfermedad o afección simultánea que, en opinión del investigador, podría interferir con la participación en el estudio o las evaluaciones de seguridad, por cualquier motivo.
    29.Presentar cualquier trastorno 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) at Week 52 (12- month).
    Evaluar el cambio respecto al inicio en la velocidad de crecimiento anualizada (VCA) a las 52 semanas (12meses)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Anthropometric measurements: Screen, Day 1, Week 13, Week 26, Week 29, Week 52
    Medidas antropometricas: Screen, Dia 1, Semana 13, semana26, semana 29, semana 52
    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.
    Los criterios de valoración secundarios de la eficacia incluyen el cambio respecto al inicio en la puntuación Z de la estatura y el cambio respecto al inicio en la relación segmento superior:inferior del cuerpo.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Anthropometric measurements: Screen, Day 1, Week 13, Week 26, Week 29, Week 52
    Meidas antropometricas: screen, dia 1, semana 13, semana 26, semana 29, semana 52
    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 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 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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA10
    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
    France
    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
    LVLS
    UVUP: Ultima visita Ultimo paciente
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months52
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months52
    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: 110
    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: 88
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 22
    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
    Subjects are children and adolescents 5 to less than 15 years of age.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    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)
    Following completion of 52 weeks subjects in both treatment groups may be eligible to receive BMN 111 in an open-label extension study, to assess safety and efficacy of BMN 111 over a longer term.
    Después de que hayan finalizado el periodo de tratamiento del estudio, los pacientes de ambos grupos, tratamiento y control, podrán ser aptos para recibir BMN 111 en un grupo aparte, con el fin de evaluar la seguridad y la eficacia de BMN 111 durante un periodo de tiempo más prolongado.
    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 Decision2017-04-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-04-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-10-30
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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