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    Summary
    EudraCT Number:2017-004087-35
    Sponsor's Protocol Code Number:AVXS-101-CL-304
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2018-04-23
    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 ConcernedItaly - Italian Medicines Agency
    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
    Studio internazionale di un’unica dose singola di AVXS-101 somministrata a neonati affetti da atrofia muscolare spinale con copie multiple di SMN2 geneticamente diagnosticata e presintomatica
    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
    Sperimentazione clinica sulla terapia di sostituzione genica a dose singola per neonati affetti da atrofia muscolare spinale geneticamente diagnosticata e presintomatica
    A.3.2Name or abbreviated title of the trial where available
    SPR1NT
    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 number1224500 2097
    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) Yes
    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 muscolare spinale
    E.1.1.1Medical condition in easily understood language
    Spinal Muscular Atrophy
    atrofia muscolare spinale
    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.
    Efficacy 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:
    • Assess 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

    Sicurezza
    •Valutare la sicurezza di AVXS-101 attraverso l’incidenza di eventi avversi (AE) e/o eventi avversi seri (SAE)
    •Valutare la sicurezza di AVXS-101 in base alla variazione rispetto al basale in parametri clinici di laboratorio
    La valutazione degli obiettivi di efficacia avverrà in modo indipendente per ciascuna coorte.
    Efficacia per i pazienti con delezioni bialleliche di SMN1 e 2 copie di SMN2:
    •Valutare l’efficacia di AVXS-101 dimostrando l’autonomia funzionale nel mantenimento della posizione seduta per almeno 30 secondi fino a 18 mesi d’età Efficacia per i pazienti con 3 copie di SMN2:
    •Valutare l’efficacia di AVXS-101 in base alla percentuale di pazienti che acquisiscono la capacità di mantenere la stazione eretta senza sostegno per almeno tre secondi fino a 24 mesi d’età
    Efficacia per i pazienti con 4 copie di SMN2:
    •Valutare l’efficacia di AVXS-101 dimostrando la capacità di ottenere un punteggio pesato sulle
    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

    Efficacia per i pazienti con 2 copie di SMN2:
    •Determinare l’efficacia di AVXS-101 in base alla sopravvivenza, definita come nessun decesso o necessità di ventilazione permanente salvo in caso di malattia acuta o in fase perioperatoria, come valutata a 14 mesi d’età
    •Valutare l’efficacia di AVXS-101 dimostrando la capacità di mantenere il peso pari o superiore al terzo percentile senza necessità di supporto nutrizionale non orale/meccanico in corrispondenza di qualsiasi visita fino a 18 mesi d’età
    Efficacia per i pazienti con 3 copie di SMN2:
    •Valutare l’efficacia di AVXS-101 dimostrando la capacità di deambulare autonomamente, definita come capacità di compiere autonomamente almeno cinque passi mostrando coordinazione ed equilibrio, in corrispondenza di qualsiasi visita fino a 24 mesi d’età
    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



    Tutti i pazienti:
    •Età ≤6 settimane (≤42 giorni) alla data di somministrazione della dose
    •Capacità di tollerare liquidi sottili, come dimostrata mediante un test di deglutizione formale eseguito al letto del paziente
    •Potenziale d’azione muscolare composto (CMAP) ≥2 mV al basale
    •Età gestazionale di 35-42 settimane
    •Diagnosi genetica come descritta sotto, ottenuta da un test di screening neonatale o prenatale metodologicamente accettabile
    Pazienti con 2 copie di SMN2
    •Pazienti con SMA presintomatica di tipo 1, come determinata in base alle seguenti caratteristiche:
    •Delezione biallelica di SMN1
    •2 copie di SMN2
    Pazienti con 3 copie di SMN2
    •Pazienti con SMA presintomatica di tipo 2, come determinata in base alle seguenti caratteristiche:
    •Delezione biallelica di SMN1
    •3 copie di SMN2
    Pazienti con 4 copie di SMN2
    •Pazienti con quadro predominante di SMA presintomatica di tipo 3, come determinata in base alle seguenti caratteristiche:
    •Delezione biallelica di SMN1
    •4 copie di SMN2
    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 alla visita di screening <2 kg
    •Ipossiemia (saturazione di ossigeno <96% in stato di veglia o durante il sonno in assenza di qualsiasi supplementazione di ossigeno o supporto respiratorio) alla visita di screening
    •Presenza allo screening o subito prima della somministrazione di segni o sintomi clinici che, a giudizio dello sperimentatore, appaiono fortemente suggestivi di SMA (per es., fascicolazione della lingua, ipotonia, areflessia)
    •Tracheostomia o attuale uso profilattico o necessità di supporto ventilatorio non invasivo in qualsiasi momento e per qualunque durata prima dello screening o nel corso del periodo di screening
    •Pazienti con segni di aspirazione/incapacità di tollerare liquidi non addensati in base a un test di deglutizione formale eseguito nell’ambito dello screening o pazienti sottoposti a qualsiasi forma di nutrizione non orale
    •Trattamento con un prodotto sperimentale o commerciale, compreso nusinersen, somministrato per il trattamento della SMA. È inclusa qualsiasi anamnesi di terapia genica, precedente trattamento con oligonucleotidi antisenso o trapianto cellulare
    •Pazienti il cui peso per l’età è al di sotto del terzo percentile in base agli Standard di crescita del bambino dell’Organizzazione mondiale della sanità OMS)
    •Malattia grave non a carico del tratto respiratorio che richieda un trattamento sistemico e/o il ricovero nelle 2 settimane precedenti lo screening
    •Infezione del tratto respiratorio superiore o inferiore che richieda attenzione medica, intervento medico o potenziamento di qualsiasi tipo di trattamento di supporto nelle 4 settimane precedenti la somministrazione
    •Procedura chirurgica maggiore, incluso l’intervento chirurgico/la procedura di correzione della scoliosi, precedentemente eseguita, programmata o prevista durante il periodo di valutazione dello studio
    •Uso concomitante di qualsiasi dei seguenti: farmaci per il trattamento della miopatia o neuropatia, agenti utilizzati per il trattamento del diabete mellito o terapia immunosoppressiva in corso, plasmaferesi, immunomodulatori come adalimumab, terapia immunosoppressiva nelle 4 settimane precedenti la terapia di sostituzione genica (per es., corticosteroidi, ciclosporina, tacrolimus, metotrexato, ciclofosfamide, immunoglobulina EV, rituximab)
    •Titolo anticorpale anti-AAV9 >1:50, come determinato mediante test di legame eseguito con la metodica dell’immunoassorbimento enzimatico (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
    Criteri di valutazione:
    Sicurezza:
    Primari:
    •Incidenza di eventi avversi (AE) e/o eventi avversi seri (SAE)
    •Variazione rispetto al basale in parametri clinici di laboratorio
    La valutazione degli obiettivi di efficacia avverrà in modo indipendente per ciascuna coorte.
    Efficacia per i pazienti con 2 copie di SMN2:
    Primario:
    •Proporzione di pazienti che raggiungono la tappa fondamentale di sviluppo dell’autonomia funzionale nel mantenimento della posizione seduta in corrispondenza di qualsiasi visita fino a 18 mesi d’età
    Efficacia per i pazienti con 3 copie di SMN2:
    Primario:
    •Proporzione di pazienti che acquisiscono la capacità di mantenere la stazione eretta senza sostegno per almeno tre secondi in corrispondenza di qualsiasi visita fino a 24 mesi d’età
    Efficacia per i pazienti con 4 copie di SMN2:
    Primario:
    •Proporzione di pazienti che dimostrano la capacità di ottenere un punteggio pesato sulle scale Bayley V.3, sottotest della motricità grossolana e fine, entro 1,5 deviazioni standard rispetto a uno standard di riferimento per lo sviluppo cronologico, come valutata a 36 mesi d’età
    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



    Pazienti con 2 copie di SMN2:
    18 mesi d’età
    Pazienti con 3 copie di SMN2:
    24 mesi d’età
    Pazienti con 4 copie di SMN2:
    36 mesi d’età
    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

    Pazienti con 2 copie di SMN2:
    •Proporzione di pazienti che sono sopravvissuti e non hanno richiesto di essere sottoposti a ventilazione permanente salvo in caso di malattia acuta o in fase perioperatoria, valutata a 14 mesi d’età
    •Proporzione di pazienti che hanno acquisito la capacità di mantenere il peso pari o superiore al terzo percentile senza necessità di supporto nutrizionale non orale/meccanico in corrispondenza di qualsiasi visita fino a 18 mesi d’età
    Pazienti con 3 copie di SMN2:
    •Proporzione di pazienti che dimostrano la capacità di deambulare autonomamente, definita come capacità di compiere autonomamente almeno cinque passi mostrando coordinazione ed equilibrio, in corrispondenza di qualsiasi visita fino a 24 mesi d’età
    Pazienti con 4 copie di SMN2:
    Non applicabile
    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
    Pazienti con 2 copie di SMN2:
    •Proporzione di pazienti che sono sopravvissuti e non hanno richiesto di essere sottoposti a ventilazione permanente, valutata a 14 mesi d’età
    •Proporzione di pazienti che hanno acquisito la capacità di mantenere il peso pari o superiore al terzo percentile senza necessità di supporto nutrizionale non orale/meccanico in corrispondenza di qualsiasi visita fino a 18 mesi d’età
    Pazienti con 3 copie di SMN2:
    •24 mesi d’età
    Pazienti con 4 copie di SMN2:
    Non applicabile
    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 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 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
    LVLS
    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.
    Dopo la visita di fine studio, ai pazienti eleggibili verrà chiesto di entrare in uno studio di follow up a lungo termine.
    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 Decision2018-12-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-21
    P. End of Trial
    P.End of Trial StatusCompleted
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    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.

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