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 were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   43886   clinical trials with a EudraCT protocol, of which   7296   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 on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    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:2017-004087-35
    Sponsor's Protocol Code Number:AVXS-101-CL-304
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-04-20
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2017-004087-35
    A.3Full title of the trial
    A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2
    Estudio global de una única dosis y en una sola administración de AVXS-101 en lactantes con atrofia muscular espinal presintomática diagnosticada genéticamente y con múltiples copias del gen SMN2.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy
    Ensayo clinic de una sola dosis de terapia génica en lactantes con atrofia muscular espinal geneticamente diagnosticada y prseintomatica.
    A.3.2Name or abbreviated title of the trial where available
    SPR1NT
    A.4.1Sponsor's protocol code numberAVXS-101-CL-304
    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 SponsorAveXis, 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 supportAveXis, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAveXis, Inc.
    B.5.2Functional name of contact pointSr. Global Trial Operations Manager
    B.5.3 Address:
    B.5.3.1Street Address2275 Half Day Road, Suite 200
    B.5.3.2Town/ cityBannockburn IL
    B.5.3.3Post code60015
    B.5.3.4CountryUnited States
    B.5.4Telephone number12245002097
    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/3/15/1509
    D.3 Description of the IMP
    D.3.1Product nameAVXS-101 (previously known as scAAV9.CB.SMN)
    D.3.2Product code AVXS-101
    D.3.4Pharmaceutical form Infusion
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNonasemnogene abeparvovec
    D.3.9.1CAS number 1922968-73-7
    D.3.9.2Current sponsor codeAVXS-101
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number20000000000000 to 60000000000000
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    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
    Spinal Muscular Atrophy
    Atrofia Muscular Espinal
    E.1.1.1Medical condition in easily understood language
    Spinal Muscular Atrophy
    Atrofia Muscular Espinal
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Safety
    •Evaluate the safety of AVXS-101 through incidence of adverse events (AEs) and/or serious adverse events (SAEs)
    •Evaluate the safety of AVXS-101 based on the change from baseline in clinical laboratory parameters

    Efficacy objectives will be assessed independently for each cohort.
    Eff. for patients with bi-allelic SMN1 deletions and 2 copies of SMN2:
    •Assess the efficacy of AVXS-101 by demonstrating functional independent sitting for at least 30 seconds up to 18 months of age
    Efficacy for patients with 3 copies of SMN2:
    •Assess the efficacy of AVXS-101 based on the proportion of patients achieving the ability to stand without support for at least three seconds up to 24 months of age
    Efficacy for patients with 4 copies of SMN2:
    •Ass. the efficacy of AVXS-101 by demonstrating the ability to achieve a scaled score on Bayley V.3 Gross and Fine Motor Subtests within 1.5 standard deviations of chronological development reference standard as assessed at 36 months of age
    Seguridad:
    •Ev. seg. de AVXS-101 a través de la incidencia de acont. adv. (AA) y de aconte. adv. graves (AAG).
    •Ev. seg. de AVXS-101 basándose en el cambio con respecto al valor basal en los result. de los aná. clínics.
    Los objs. de la efic. se eva. de manera indepen. en cada cohorte.
    Efic. en pacien. con 2 copias del gen SMN2:
    •Ev. la efic. de AVXS-101 mostrando la cap. de sedestación indep. funcional durante un mín.: 30 seg - 18 meses de edad.
    Efic. en pacien. con 3 copias del gen SMN2:
    •Ev.la efic. de AVXS-101 basándose en la proporción de pacien. que consiguen quedarse de pie sin ayuda durante un mín. de: 3 segs. - 24 meses de edad.
    Efic. en pacien. con 4 copias del gen SMN2:
    •Ev. la efi. de AVXS-101 mostrando la capacidad para lograr una puntuación transformada en las subescalas de motricidad fina y general de las Esc. Bayley de desarrollo infantil-III dentro de 1,5 desviaciones estándar de un patrón de desarrollo cronológico de ref. evaluado a los 36 meses de edad.
    E.2.2Secondary objectives of the trial
    Efficacy for patients with 2 copies of SMN2:
    • Determine the efficacy of AVXS-101 based on survival, defined as avoidance of death or the requirement of permanent ventilation in the absence of acute illness or perioperatively as assessed at 14 months of age
    • Assess efficacy of AVXS-101 by demonstrating the ability to maintain weight at or above the third percentile without need for non-oral/mechanical feeding support at any visit up to 18 months of age

    Efficacy for patients with 3 copies of SMN2:
    • Assess the efficacy of AVXS-101 by demonstrating the ability to walk alone defined as the ability to take at least five steps independently displaying coordination and balance at any visit up to 24 months of age
    Eficacia en los pacientes con 2 copias del gen SMN2:

    • Determinar la eficacia de AVXS-101 basándose en la supervivencia, definida como la evitación de la muerte o la necesidad de ventilación mecánica permanente en ausencia de enfermedad aguda o de manera perioperatoria, evaluada a los 14 meses de edad.
    • Evaluar la eficacia de AVXS-101 mostrando la capacidad para mantener el peso corporal en el tercer percentil o por encima de este, sin necesidad de alimentación no oral/mecánica en cualquier visita hasta los 18 meses de edad.

    Eficacia en los pacientes con 3 copias del gen SMN2:

    • Evaluar la eficacia de AVXS-101 mostrando la capacidad para caminar solo, definida como la capacidad para dar un mínimo de cinco pasos de manera autónoma, exhibiendo coordinación y equilibrio, en cualquier visita hasta los 24 meses de edad.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    All patients:
    • Age ≤6 weeks (≤42 days) at time of dose
    • Ability to tolerate thin liquids as demonstrated through a formal bedside swallowing test
    • Compound muscle action potential (CMAP) ≥2 mV at Baseline
    • Gestational age of 35 to 42 weeks
    • Genetic diagnosis as described below, obtained from an acceptable newborn or pre-natal screening test method

    Patients with 2 copies of SMN2
    • Patients with pre-symptomatic SMA Type 1 as determined by the following features:
    • Bi-allelic deletion of SMN1
    • 2 copies of SMN2

    Patients with 3 copies of SMN2
    • Patients with pre-symptomatic SMA Type 2 as determined by the following features:
    • Bi-allelic deletion of SMN1
    • 3 copies of SMN2

    Patients with 4 copies of SMN2
    • Patients predominantly with pre-symptomatic SMA Type 3 as determined by the following features:
    • Bi-allelic deletion of SMN1
    • 4 copies of SMN2
    Todos los pacientes:
    • Edad ≤6 semanas (≤42 días) en el momento de la administración de la dosis.
    • Capacidad de tolerar líquidos diluidos, demostrada mediante una prueba de deglución formal de diagnóstico inmediato.
    • Potencial de acción muscular compuesto (PAMC) ≥2 mV en la visita basal; se llevará a cabo una revisión centralizada de los datos del PAMC
    • Edad gestacional de 35 a 42 semanas
    • Diagnóstico genético, tal y como se describe a continuación, obtenido a partir de un método aceptable de cribado prenatal o neonatal.

    Pacientes con 2 copias del genSMN2
    • Pacientes con AME tipo 1 presintomática determinada por las siguientes características:
    - 2 copias del gen SMN2
    - Delecion Bialelica de SMN1

    Pacientes con 3 copias del genSMN2
    • Pacientes con AME tipo 2 presintomática determinada por las siguientes características:
    - 3 copias del gen SMN2
    - Delecion Bialelica de SMN1

    Pacientes con 4 copias del genSMN2:
    • Pacientes con AME tipo 3 presintomática determinada por las siguientes características:
    - 4 copias del gen SMN2
    - Delecion Bialelica de SMN1
    E.4Principal exclusion criteria
    • Weight at screening visit <2 kg
    • Hypoxemia (oxygen saturation <96% awake or asleep without any supplemental oxygen or respiratory support) at the screening visit
    • Any clinical signs or symptoms at screening or immediately prior to dosing that are, in the opinion of the Investigator, strongly suggestive of SMA (e.g., tongue fasciculation, hypotonia, areflexia)
    • Tracheostomy or current prophylactic use or requirement of non invasive ventilatory support at any time and for any duration prior to screening or during the screening period
    • Patients with signs of aspiration/inability to tolerate non thickened liquids based on a formal swallowing test performed as part of screening or patients receiving any non-oral feeding method
    • Treatment with an investigational or commercial product, including nusinersen, given for the treatment of SMA. This includes any history of gene therapy, prior antisense oligonucleotide treatment, or cell transplantation.
    • Patients whose weight-for-age is below the third percentile based on World Health Organization (WHO) Child Growth Standards
    • Serious non respiratory tract illness requiring systemic treatment and/or hospitalization within 2 weeks prior to screening
    • Upper or lower respiratory infection requiring medical attention, medical intervention, or increase in supportive care of any manner within 4 weeks prior to dosing
    • Previous, planned or expected major surgical procedure including scoliosis repair surgery/procedure during the study assessment period
    • Concomitant use of any of the following: drugs for treatment of myopathy or neuropathy, agents used to treat diabetes mellitus, or ongoing immunosuppressive therapy, plasmapheresis, immunomodulators such as adalimumab, immunosuppressive therapy within 4 weeks prior to gene replacement therapy (e.g., corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, IV immunoglobulin, rituximab)
    • Anti AAV9 antibody titer >1:50 as determined by Enzyme linked Immunosorbent Assay (ELISA) binding immunoassay
    • Peso corporal en la visita de selección <2 kg.
    • Hipoxemia (saturación de oxígeno <96 % durante el sueño o la vigilia sin oxígeno suplementario ni ventilación mecánica) en la visita de selección.
    • Cualquier signo o síntoma clínico en la selección o inmediatamente antes de la administración que, en opinión del investigador, sea muy sugestivo de AME (p. ej., fasciculaciones linguales, hipotonía, arreflexia)
    • Traqueotomía o uso profiláctico en curso o necesidad de ventilación mecánica no invasiva en cualquier momento y de cualquier duración antes de la selección o durante el período de selección.
    • Pacientes con signos de aspiración/incapacidad para tolerar líquidos no espesos, sobre la base de una prueba de deglución formal realizada como parte de la selección, o pacientes que estén recibiendo alimentación no oral por cualquier método.
    • Tratamiento para la AME con un producto en investigación o comercializado, por ejemplo, nusinersén. Entre los tratamientos para la AME se incluyen los antecedentes de terapia génica, tratamiento previo con oligonucleótidos antisentido o trasplante de células.
    • Pacientes cuyo peso para la edad esté por debajo del tercer percentil según los patrones de crecimiento infantil de la Organización Mundial de la Salud (OMS)
    • Enfermedad grave no respiratoria que requiera tratamiento sistémico u hospitalización en las 2 semanas previas a la selección.
    • Infección de las vías respiratorias altas o bajas que precise atención médica, intervención médica o aumento del tratamiento complementario de cualquier manera en las 4 semanas anteriores a la administración.
    • Intervención quirúrgica mayor previa, programada o prevista, incluida la corrección quirúrgica de la escoliosis, durante el período de evaluación del studio.
    • Uso concomitante de cualquiera de los siguientes: fármacos para el tratamiento de miopatías o neuropatías, fármacos para tratar la diabetes mellitus o tratamiento inmunodepresor en curso, plasmaféresis, inmunomoduladores como adalimumab, tratamiento inmunodepresor en las 4 semanas previas a la terapia génica (p. ej., corticoesteroides, ciclosporina, tacrolimús, metotrexato, ciclofosfamida, inmunoglobulina i.v., rituximab).
    • Título de anticuerpos anti-AAV9 >1:50 determinado por enzimoinmunoanálisis de adsorción (ELISA).
    E.5 End points
    E.5.1Primary end point(s)
    Criteria for Evaluation:
    Safety:
    Primary:
    • Incidence of adverse events (AEs) and/or serious adverse events (SAEs)
    • Change from baseline in clinical laboratory parameters

    Efficacy objectives will be assessed independently for each cohort.

    Efficacy for patients with 2 copies of SMN2:
    Primary:
    • Proportion of patients achieving the development milestone of functional independent sitting at any visit up to 18 months of age

    Efficacy for patients with 3 copies of SMN2:
    Primary:
    • Proportion of patients achieving the ability to stand without support for at least three seconds at any visit up to 24 months of age

    Efficacy for patients with 4 copies of SMN2:
    Primary:
    • Proportion of patients demonstrating the ability to achieve a scaled score on Bayley V.3 Gross and Fine Motor Subtests within 1.5 standard deviations of chronological development reference standard as assessed at 36 months of age
    Criterios de evaluación:
    Seguridad:
    Principal:
    • Incidencia de acontecimientos adversos (AA) y acontecimientos adversos graves (AAG).
    • Cambio con respecto al valor basal en los resultados de los análisis clínicos.
    Los objetivos de la eficacia se evaluarán de manera independiente en cada cohort

    Eficacia en los pacientes con 2 copias del gen SMN2:
    Principal:
    • Proporción de pacientes que alcanzan el hito de desarrollo de sedestación independiente funcional en cualquier visita hasta los 18 meses de edad.

    Eficacia en los pacientes con 3 copias del gen SMN2:
    Principal:
    • Proporción de pacientes que consiguen quedarse de pie sin ayuda durante un mínimo de tres segundos en cualquier visita hasta los 24 meses de edad

    Eficacia en los pacientes con 4 copias del gen SMN2:
    Principal:
    • Proporción de pacientes con la capacidad para lograr una puntuación transformada en las subescalas de motricidad fina y general de las Escalas Bayley de desarrollo infantil-III dentro de 1,5 desviaciones estándar de un patrón de desarrollo cronológico de referencia evaluado a los 36 meses de edad.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Patients with 2 copies of SMN2:
    18 months of age
    Patients with 3 copies of SMN2:
    24 months of age
    Patients with 4 copies of SMN2:
    36 months of age
    Pacientes con 2 copias de SMN2:
    18 meses de edad
    Pacientes con 3 copias de SMN2:
    24 meses de edad
    Pacientes con 4 copias de SMN2:
    36 meses de edad
    E.5.2Secondary end point(s)
    Patients with 2 copies of SMN2:
    • Proportion of patients that have survived and have not required permanent ventilation in the absence of acute illness or perioperatively assessed at 14 months of age
    • Proportion of patients that have achieved the ability to maintain weight at or above the third percentile without need for non-oral/mechanical feeding support at any visit up to 18 months of age


    Patients with 3 copies of SMN2:
    • Proportion of patients demonstrating the ability to walk alone defined as the ability to take at least five steps independently displaying coordination and balance at any visit up to 24 months of age

    Patients with 4 copies of SMN2:
    Not Applicable
    Pacientes con 2 copias de SMN2:
    • Proporción de pacientes que han sobrevivido y no han necesitado ventilación mecánica permanente en ausencia de enfermedad aguda o de forma perioperatoria a los 14 meses de edad.
    • Proporción de pacientes que logran mantener el peso corporal en el tercer percentil o por encima de este, sin necesidad de alimentación no oral/mecánica en cualquier visita hasta los 18 meses de edad

    Pacientes con 3 copias de SMN2:
    • Proporción de pacientes que logran la capacidad de caminar solos, definida como la capacidad para dar un mínimo de cinco pasos de manera autónoma, exhibiendo coordinación y equilibrio, en cualquier visita hasta los 24 meses de edad.

    Pacientes con 4 copias de SMN2:

    No aplica.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Patients with 2 copies of SMN2:
    • Proportion of patients that have survived and have not required permanent ventilation assessed at 14 months of age
    • Proportion of patients that have achieved the ability to maintain weight at or above the third percentile without need for non-oral/mechanical feeding support at any visit up to 18 months of age

    Patients with 3 copies of SMN2:
    • 24 months of age

    Patients with 4 copies of SMN2:
    Not Applicable
    Pacientes con 2 copias de SMN2:
    • Proporción de pacientes que han sobrevivido y no han necesitado ventilación mecánica permanente en ausencia de enfermedad aguda o de forma perioperatoria a los 14 meses de edad.
    • Proporción de pacientes que logran mantener el peso corporal en el tercer percentil o por encima de este, sin necesidad de alimentación no oral/mecánica en cualquier visita hasta los 18 meses de edad.

    Pacientes con 3 copias de SMN2:
    • 24 meses de edad.

    Pacientes con 4 copias de SMN2:

    No aplica.
    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 No
    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 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 Yes
    E.8.1.7.1Other trial design description
    Open-label, single-arm, single-dose
    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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Belgium
    Canada
    Germany
    Israel
    Italy
    Japan
    Korea, Democratic People's Republic of
    Netherlands
    Spain
    Taiwan
    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
    Ultima visita ultimo paciente
    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 days0
    E.8.9.2In all countries concerned by the trial years5
    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: 44
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 22
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 22
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 44
    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)
    After the End of Trial Visit, eligible patients will be asked to rollover into the long-term follow up trial.
    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 Decision2018-12-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-12
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA