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    Summary
    EudraCT Number:2016-000301-37
    Sponsor's Protocol Code Number:EFC13738
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-06-20
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2016-000301-37
    A.3Full title of the trial
    Open label, Two Cohort (with and without Imiglucerase), Multicenter Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Eliglustat in Pediatric Patients with Gaucher Disease Type 1 and Type 3
    “Studio in aperto, multicentrico, con due coorti di trattamento, volto a valutare la farmacocinetica, la sicurezza e l’efficacia di eliglustat (con e senza imiglucerasi) in pazienti pediatrici affetti da malattia di Gaucher di tipo 1 e tipo 3”
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy of Eliglustat with or without Imiglucerase in Pediatric Patients with Gaucher Disease (GD) Type 1 and Type 3
    “Sicurezza e Efficacia di Eliglustat con e senza Imiglucerasi in pazienti pediatrici affetti da malattia di Gaucher (GD) di tipo 1 e tipo 3”
    A.3.2Name or abbreviated title of the trial where available
    ELIKIDS
    ELIKIDS
    A.4.1Sponsor's protocol code numberEFC13738
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1172-2950
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/191/2015
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGenzyme Corporation
    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 supportGenzyme Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationContact-Point
    B.5.2Functional name of contact pointContact-Point
    B.5.3 Address:
    B.5.3.1Street AddressVIALE BODIO, 37/B
    B.5.3.2Town/ cityMILANO
    B.5.3.3Post code20158
    B.5.3.4CountryItaly
    B.5.4Telephone number800226343
    B.5.5Fax number00390239394168
    B.5.6E-mailinformazioni.medicoscientifiche@sanofi.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Cerdelga
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme Europe B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/07/514
    D.3 Description of the IMP
    D.3.1Product nameEliglustat
    D.3.2Product code GZ385660
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEliglustat
    D.3.9.2Current sponsor codeGZ385660
    D.3.9.3Other descriptive nameELIGLUSTAT TARTRATE
    D.3.9.4EV Substance CodeSUB180210
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number84
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product Yes
    D.3.11.13.1Other medicinal product typeEliglustat tartrate is a synthetic small molecule with 2 chiral centers possessing chemical and chiral stability. The drug substance is the 1R,2R isomer.
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Cerezyme
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme Europe B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameImiglucerase
    D.3.4Pharmaceutical form Powder for concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    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 INNIMIGLUCERASE
    D.3.9.1CAS number 154248-97-2
    D.3.9.3Other descriptive nameCerezyme
    D.3.9.4EV Substance CodeSUB08148MIG
    D.3.10 Strength
    D.3.10.1Concentration unit U unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number400
    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 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 Yes
    D.3.11.13.1Other medicinal product typeRecombinant human derived macrophage-targeted beta-Glucocerebrosidase
    D.IMP: 3
    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/3/07/514
    D.3 Description of the IMP
    D.3.1Product nameEliglustat
    D.3.2Product code GZ385660
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEliglustat
    D.3.9.2Current sponsor codeGZ385660
    D.3.9.3Other descriptive nameELIGLUSTAT TARTRATE
    D.3.9.4EV Substance CodeSUB180210
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number21
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product Yes
    D.3.11.13.1Other medicinal product typeEliglustat tartrate is a synthetic small molecule with 2 chiral centers possessing chemical and chiral stability. The drug substance is the 1R,2R isomer.
    D.IMP: 4
    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/3/07/514
    D.3 Description of the IMP
    D.3.1Product nameEliglustat
    D.3.2Product code GZ385660
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNEliglustat
    D.3.9.2Current sponsor codeGZ385660
    D.3.9.3Other descriptive nameELIGLUSTAT TARTRATE
    D.3.9.4EV Substance CodeSUB180210
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number42
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product Yes
    D.3.11.13.1Other medicinal product typeEliglustat tartrate is a synthetic small molecule with 2 chiral centers possessing chemical and chiral stability. The drug substance is the 1R,2R isomer.
    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
    Congenital, hereditary and neonatal diseases
    MALATTIA NEONATALE
    CONGENITA ED EREDITARIA
    E.1.1.1Medical condition in easily understood language
    Congenital, hereditary and neonatal diseases
    MALATTIA NEONATALE CONGENITA ED EREDITARIA
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10075697
    E.1.2Term Gaucher's disease type I
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10075699
    E.1.2Term Gaucher's disease type III
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (≥6 to <18 years old).
    Valutare la sicurezza e la farmacocinetica di eliglustat in pazienti pediatrici (fra ≥6 e <18 anni di età).
    E.2.2Secondary objectives of the trial
    Evaluate the efficacy of eliglustat and quality of life in pediatric patients (≥6 to <18 years old).
    Valutare l’efficacia di eliglustat e la qualità della vita in pazienti pediatrici (fra ≥6 e <18 anni di età)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -The patient is 6 to <18 years old at the time of informed consent.
    -Male and female patients with a clinical diagnosis of Gaucher disease (GD) type 1 or type 3 with documented deficiency of acid beta-glucosidase activity by enzyme assay and glucocerebrosidase (GBA) genotype.
    -Postmenarchal female patients must have a documented negative pregnancy test prior to enrollment and throughout the study. Patients must be willing to practice true abstinence in line with their preferred and usual lifestyle, or use a medically accepted form of contraception throughout the study.

    Cohort 1 (Eliglustat monotherapy):
    -Patients must have been receiving an enzyme replacement therapy (ERT) for a minimum of 24 months at a monthly dose equivalent to 30 U/kg to 130 U/kg of Cerezyme® (imiglucerase) with treatment ongoing at the time of enrollment. Patients must be at pre-specified treatment goals, as defined by:
    -Hemoglobin level for ages 6 to <12 years: ≥11.0 g/dL; for ages 12 to <18 years: ≥11.0 g/dL for females and ≥12.0 g/dL for males;
    -Platelet count ≥100,000/mm3;
    -Spleen volume <10.0 multiples of normal (MN);
    -Liver volume <1.5 MN;
    -Absence of GD related pulmonary disease, and severe bone disease, as defined below for Cohort 2.

    Cohort 2 (Eliglustat plus imiglucerase):
    -Patients must have been receiving an ERT for a minimum of 36 months at a dose equivalent to at least 60 U/kg of imiglucerase every 2 weeks at the time of enrollment with treatment ongoing at the time of enrollment and the dose stable for at least the 6 months preceding enrollment. Patients must have severe clinical manifestations of GD, as defined by the presence of at least of the
    following:
    -GD related pulmonary disease such as interstitial lung disease (ILD). The diagnosis of ILD must be confirmed by the presence of reticulonodular densities on chest X-ray.
    AND/OR
    -Symptomatic bone disease characterized by pathological fracture, osteonecrosis,
    osteopenia/osteoporosis, or bone crisis occurring in the 12 months prior to enrollment.
    AND/OR
    -Persistent thrombocytopenia (<80,000/mm3) related to GD.
    • Il paziente è di età compresa fra 6 e <18 anni al momento del consenso informato
    • Pazienti di sesso maschile e femminile con diagnosi clinica di malattia di Gaucher (GD) di tipo 1 e 3 con deficit dell’attività dell’enzima beta­glucosidasi acida (GBA) documentata da dosaggio enzimatico e genotipo GBA
    • Le pazienti di sesso femminile post-menarca devono avere un test di gravidanza negativo documentato prima dell’arruolamento e per tutta la durata dello studio. I pazienti devono essere disposti a praticare l’astinenza totale, in linea con il loro stile di vita preferito e usuale, o fare uso di una forma di contraccezione accettata dal punto di vista medico per tutta la durata dello studio
    • Per la Coorte 1 (Eliglustat in Monoterapia):
    I pazienti devono essere in trattamento con una terapia sostitutiva enzimatica (ERT) da un minimo di 24 mesi a una dose mensile equivalente a 30-130 U/kg di Cerezyme ® (imiglucerase) con trattamento in corso al momento dell’arruolamento. I pazienti devono aver raggiunto gli obiettivi terapeutici prespecificati , come definito da:
    -livello di emoglobina per le età comprese fra 6 e <12 anni: ≥11,0 g/dl; per le età comprese fra 12 e <18 anni: ≥11,0 g/dl per le femmine e ≥12,0 g/dl per i maschi;
    -conta piastrinica ≥100.000/mm3;
    -volume della milza <10,0 volte il valore normale (multiples of normal, MN);
    -volume del fegato <1,5 MN;
    -assenza di malattia polmonare correlata alla GD e di malattia ossea grave, secondo la definizione che segue per la Coorte 2.
    • Per la Coorte 2 (Eliglustat più Imiglucerase):
    I pazienti devono essere in trattamento con una terapia sostitutiva enzimatica (ERT) da un minimo di 36 mesi a una dose equivalente di almeno 60 U/kg di imiglucerasi ogni 2 settimane al momento dell’arruolamento, con il trattamento in corso al momento dell’arruolamento e a dosaggio stabile da almeno 6 mesi prima dell’arruolamento. I pazienti devono presentare manifestazioni cliniche gravi della GD, come definito dalla presenza di almeno una delle condizioni seguenti:
    1) Malattia polmonare correlata alla GD come la patologia polmonare interstiziale ( Interstitial Lung Disease ILD). La diagnosi di ILD deve essere confermata dalla presenza di densità reticolonodulari alla radiografia del torace.
    E/O
    2) Malattia ossea sintomatica caratterizzata da fratture patologiche, osteonecrosi, osteopenia/osteoporosi oppure crisi ossea avvenuta nei 12 mesi precedenti all’arruolamento.
    E/O
    3) Trombocitopenia persistente (<80.000/mm3) correlata alla GD
    E.4Principal exclusion criteria
    -Substrate reduction therapy for GD within 6 months prior to enrollment
    -Partial or total splenectomy if performed within 2 years prior to enrollment
    -The patient is transfusion dependent, a history of esophageal varices or liver infarction, elevated liver enzymes, significant congenital cardiac defect, coronary artery disease or left sided heart failure; clinically significant arrhythmias or conduction defect such as Type 2 second degree or third degree atrioventricular (AV) block, complete bundle branch block, prolonged QTc interval, or sustained ventricular tachycardia (VT).
    -The patient has any clinically significant disease other than GD.
    -The patient has neurological symptoms other than oculomotor apraxia at study entry.
    -The patient has received an investigational product within 30 days prior to enrollment.
    -The patient is unable to receive treatment with imiglucerase due to a known hypersensitivity or is unwilling to receive imiglucerase treatment every 2 weeks.
    -The patient has a known hereditary galactose intolerance, Lapp lactase deficiency or glucose galactose malabsorption, or is a CYP2D6 ultra-rapid metabolizer or indeterminate metabolizer.
    • Terapia di riduzione del substrato per la GD nei 6 mesi precedenti all’arruolamento
    • Splenectomia parziale o totale se effettuata entro i 2 anni precedenti all’arruolamento.
    • Il paziente è dipendente dalle trasfusioni, presenta anamnesi di varici esofagee o infarto epatico, valori di Enzimi epatici elevati, difetto cardiaco congenito clinicamente significativo, coronaropatia o insufficienza cardiaca sinistra; aritmie clinicamente significative o disturbi della conduzione, come blocco atrioventricolare (AV) di 2° o 3° grado, blocco completo di branca, prolungamento dell’intervallo QTc o tachicardia ventricolare (TV) sostenuta
    • Il paziente presenta qualsiasi malattia clinicamente significativa, diversa dalla GD
    • Il paziente soffre di sintomi neurologici eccetto aprassia oculomotoria all’inizio dello studio
    • Il paziente è stato trattato con un prodotto sperimentale nei 30 giorni precedenti all’arruolamento
    • Il paziente non è in grado di essere trattato con imiglucerasi a causa di una ipersensibilità nota o non è disposto a ricevere il trattamento con imiglucerasi ogni 2 settimane.
    • Il paziente presenta nota intolleranza ereditaria al galattosio, deficienza di Lapp lattasi o malassorbimento di glucosio-galattosio o è un metabolizzatore indeterminato o ultra­rapido del CYP2D6.
    E.5 End points
    E.5.1Primary end point(s)
    1- Maximum concentration (Cmax) of eliglustat in plasma.
    2- Area under the plasma eliglustat concentration-time curve (AUC)
    3- Number of adverse events in pediatric patients
    1) Concentrazione massima (Cmax) di Eliglustat nel plasma
    2) Area sotto la curva (AUC) riferita ad Eliglustat nel plasma in rapporto concentrazione –tempo
    3) Numero di eventi avversi in pazienti pediatrici
    E.5.1.1Timepoint(s) of evaluation of this end point
    1- Maximum concentration (Cmax) of eliglustat in plasma : Weeks 2, 13, 26 and 52
    2- Area under the plasma eliglustat concentration-time curve (AUC) : Weeks 2 and 52
    3- Number of adverse events in pediatric patients : Up to Week 104
    1) Concentrazione massima ( Cmax) di Eliglustat nel plasma: settimana 2, 13, 26 e 52
    2) Area sotto la curva (AUC) riferita ad Eliglustat nel plasma in rapporto concentrazione –tempo: settimana 2 e 52
    3) numero di eventi avversi in pazienti pediatrici: fino alla settimana 104
    E.5.2Secondary end point(s)
    1- Absolute change from baseline for hemoglobin (g/dL) (Cohort 1 patients)
    2- Percent change from baseline for platelet count (Cohort 1 patients)
    3- Percent change from baseline for liver volume (Cohort 1 patients)
    4- Percent change from baseline for spleen volume (Cohort 1 patients)
    5- Proportion of patients with improvement in pulmonary disease (Cohort 2 patients)
    6- Proportion of patients with improvement in bone disease (Cohort 2 patients)
    7- Proportion of patients with improvement in thrombocytopenia (Cohort 2 patients)
    8- Health-related quality of life will be measured by the Pediatric Quality of Life InventoryTM (PedsQLTM) questionnaires.
    1) Variazioni assoluta dal basale per emoglobina (g/dL) (Per i pazienti della Coorte 1)
    2) Variazione percentuale dal basale per la conta piastrinica (Per i pazienti della Coorte 1)
    3) Variazione percentuale dal basale del volume epatico (Per i pazienti della Coorte 1)
    4) Variazione percentuale dal basale del volume della milza (Per i pazienti della Coorte 1)
    5) Percentuale di pazienti che presentano un miglioramento della malattia polmonare (Per i pazienti della Coorte 2)
    6) Percentuale di pazienti che presentano un miglioramento della malattia ossea (Per i pazienti della Coorte 2)
    7) Percentuale di pazienti che presentano un miglioramento della trombocitopenia (Per i pazienti della Coorte 2)
    8) Lo stato della qualità della vita correlata alla salute sarà valutato utilizzando i questionari sulla qualità della vita in età pediatrica (Pediatric Quality of Life Inventory TM, PedsQLTM).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline and Week 52
    Dal basale alla settimana n 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 Yes
    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 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.8.2.4Number 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 EEA11
    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
    Argentina
    Canada
    Egypt
    France
    Italy
    Netherlands
    Russian Federation
    Spain
    Sweden
    Turkey
    United Kingdom
    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 years4
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months11
    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: 120
    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: 60
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 60
    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 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
    Pediatric population
    Popolazione pediatrica
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 48
    F.4.2.2In the whole clinical trial 120
    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
    NESSUNO
    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 Decision2018-07-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-06-18
    P. End of Trial
    P.End of Trial StatusOngoing
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