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   43801   clinical trials with a EudraCT protocol, of which   7264   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:2019-004511-31
    Sponsor's Protocol Code Number:cASPerCF_2007_OPBG_2019
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2020-10-07
    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 number2019-004511-31
    A.3Full title of the trial
    Prospective validation and clinical evaluation of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection.
    Validazione prospettica e valutazione clinica di un nuovo dosaggio di posaconazolo per bambini e adolescenti con fibrosi cistica e con infezione da Aspergillus.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Prospective validation and clinical evaluation of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection.
    Validazione prospettica e valutazione clinica di un nuovo dosaggio di posaconazolo per bambini e adolescenti con fibrosi cistica e con infezione da Aspergillus.
    A.3.2Name or abbreviated title of the trial where available
    cASPerCF
    cASPerCF
    A.4.1Sponsor's protocol code numbercASPerCF_2007_OPBG_2019
    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 SponsorIRCCS, OSPEDALE PEDIATRICO BAMBINO GESÙ DI ROMA
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportInnovative Medicines Initiative 2
    B.4.2CountryItaly
    B.4.1Name of organisation providing supportEuropean Union’s Horizon 2020
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIRCCS OSPEDALE PEDIATRICO BAMBINO GESU'
    B.5.2Functional name of contact pointA.R. INFETTIVOLOGIA E SVILUPPO DI F
    B.5.3 Address:
    B.5.3.1Street AddressP.zza S.Onofrio, 4
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00165
    B.5.3.4CountryItaly
    B.5.6E-mailbetty.polikar@opbg.net
    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 NOXAFIL - 40 MG/ML SOSPENSIONE ORALE USO ORALE FLACONE (VETRO) 105 ML 1 FLACONE
    D.2.1.1.2Name of the Marketing Authorisation holderMERCK SHARP e DOHME LIMITED
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameNoxafil
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Powder for oral suspension
    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 INNPosaconazolo
    D.3.9.2Current sponsor codeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Noxafil
    D.2.1.1.2Name of the Marketing Authorisation holderMerck Sharp & Dohme Ltd
    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 nameNoxafil
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Powder for oral suspension
    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 INNPOSACONAZOLO
    D.3.9.2Current sponsor codena
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 No
    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 Yes
    D.2.1.1.1Trade name Noxafil
    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 nameNoxafil
    D.3.2Product code [NA]
    D.3.4Pharmaceutical form Tablet
    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 INNPOSACONAZOLO
    D.3.9.2Current sponsor codena
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg/h milligram(s)/kilogram/hour
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 No
    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 Yes
    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 nameNoxafil
    D.3.2Product code [na]
    D.3.4Pharmaceutical form Tablet
    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 INNPOSACONAZOLO
    D.3.9.2Current sponsor codeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 No
    D.IMP: 5
    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 NOXAFIL - 40 MG/ML SOSPENSIONE ORALE USO ORALE FLACONE (VETRO) 105 ML 1 FLACONE
    D.2.1.1.2Name of the Marketing Authorisation holderMERCK SHARP e DOHME LIMITED
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameNoxafil
    D.3.2Product code [na]
    D.3.4Pharmaceutical form Powder for oral suspension
    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 INNPOSACONAZOLO
    D.3.9.2Current sponsor codeNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 No
    D.IMP: 6
    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 Noxafil
    D.2.1.1.2Name of the Marketing Authorisation holderMERCK SHARP e DOHME LIMITED
    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 nameNoxafil
    D.3.2Product code [na]
    D.3.4Pharmaceutical form Tablet
    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 INNPosaconazolo
    D.3.9.2Current sponsor codena
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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 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
    Cystic Fibrosis and Aspergillus infection.
    Fibrosi cistica e con infezione da Aspergillus.
    E.1.1.1Medical condition in easily understood language
    Cystic Fibrosis and Aspergillus infection.
    Fibrosi cistica e con infezione da Aspergillus.
    E.1.1.2Therapeutic area Diseases [C] - Bacterial Infections and Mycoses [C01]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10003488
    E.1.2Term Aspergillosis
    E.1.2System Organ Class 100000004862
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - In silico definition of the most optimal posaconazole dose for children and adolescents with CF aged 8 to 17 years.
    - Assess the prevalence of Aspergillus infection in children and adolescents with CF aged 8 -17 years.
    - An intensive sampling pharmacokinetic study of posaconazole in a limited number of children and adolescents with CF and Aspergillus infection aged 8 to 17 years.
    - Prospective clinical evaluation of the defined posaconazole dosing regimen in children and adolescents with CF and Aspergillus infection aged 8 to 17 year.
    - Assess the tolerability and safety of posaconazole in children and adolescents with CF and Aspergillus infection aged 8 to 17 years.
    - Assess the clinical efficacy of posaconazole in terms of (1) clearance of Aspergillus from the airways; (2) dampening airway inflammation, and (3) clinical outcomes (as measured by pulmonary exacerbation rate, days on antibiotics and corticosteroids, hospital admissions, change in FEV1, change in BMI, CT-ches
    Definire con modello in silico la dose di posaconazolo per bambini ed adolescenti con FC di età tra 8 e 17 anni.
    Valutare la prevalenza dell'infezione da Aspergillus in bambini ed adolescenti con FC di età tra 8 e 17 anni.
    Effettuare uno studio farmacocinetico di campionamento intensivo di posaconazolo in un numero limitato di bambini ed adolescenti con infezione da FC e Aspergillus da 8 a 17 anni .
    Effettuare la convalida clinica prospettica del regime di trattamento con posaconazolo definito in bambini ed adolescenti affetti da infezione da FC e Aspergillus da 8 a 17 anni.
    Valutare la tollerabilità e la sicurezza di posaconazolo in bambini ed adolescenti con infezione da FC e Aspergillus da 8 a 17 anni.
    Valutare l'efficacia clinica del posaconazolo in termini di (1) clearance di Aspergillus dalle vie aeree; (2) riduzione dell'infiammazione delle vie aeree e (3) risultati clinici (incluso FEV1, esacerbazioni polmonari, giorni di trattamento con antibiotici e steroidi)
    E.2.2Secondary objectives of the trial
    na
    na
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion criteria for the screening phase:
    1. Diagnosed with cystic fibrosis (genetic diagnosis and clinical phenotype of lung disease)
    2. Age = 8 yrs and < 18 yrs
    3. Able to produce a sputum sample (spontaneous or induced sputum)
    4. Informed Consent given
    Inclusion criteria for the intervention phase:
    1. Diagnosed with cystic fibrosis (genetic diagnosis and/or abnormal sweat test and clinical phenotype of lung disease)
    2. Age = 8 yrs and < 18 yrs
    3. Presence of Aspergillus infection as defined for this study
    4. Clinically stable condition without a significant change in lung function (FEV1 +/- 10%) or significant worsening of respiratory symptoms over the previous month
    5. Able to perform lung function test (FEV1)
    6. Able to produce a sputum sample (spontaneous or induced sputum)
    7. Informed Consent given
    Criteri di inclusione per la fase di screening dello studio (n=1500)
    I soggetti devono soddisfare i seguenti criteri per essere ammessi alla partecipazione alla fase di screening:
    1. Diagnosi di FC (diagnosi genetica e fenotipo clinico della malattia polmonare)
    2. Età =8 anni e <18 anni
    3. Capacità di produrre un campione di espettorato (espettorato spontaneo o indotto)
    4. Firma del Consenso Informato
    Criteri di inclusione per la fase con posaconazolo (n=135)
    1. Diagnosi di FC (diagnosi genetica e fenotipo clinico della malattia polmonare).
    2. Età =8 anni e <18 anni.
    3. Segni di infezione da Aspergillus come definito per questo studio (vedere sopra).
    4. Condizione clinicamente stabile senza un cambiamento significativo nella funzionalità polmonare (FEV1 +/- 10%) o un peggioramento significativo dei sintomi respiratori nell'ultimo mese.
    5. Capacità di eseguire test di funzionalità polmonare (FEV1%).
    6. Capacità di produrre un campione di espettorato (espettorato spontaneo o indotto)
    7. Firma del Consenso informato.
    E.4Principal exclusion criteria
    Exclusion criteria for the trial:
    1. Age < 8 yrs or = 18 yrs
    2. Unable to perform lung function test (FEV1)
    3. Unable to produce a sputum sample (spontaneous or induced sputum)
    4. Clinically unstable condition with significant change in lung function or significant worsening of respiratory symptoms
    5. Unable to tolerate oral medication
    6. Known hypersensitivity to the drug/excipients
    7. On active transplant list or transplant recipient
    8. Azole resistant Aspergillus sp. cultured
    9. Patients receiving terfenadine, ergot alkaloids, astemizole, cisapride, pimozide, halofantrine, quinidine, or HMG-CoA reductase inhibitors metabolised through CYP3A4
    10. Patients receiving omalizumab
    11. Received systemic mould-active antifungals in the last month
    12. Shortened or elongated QT interval
    13. Cardiac failure
    14. ALT = 200 U/L
    15. AST = 225 U/L
    16. Alkaline phosphatase = 460 U/L
    17. Bilirubin = 50 umol/L
    18. Pregnancy
    19. Informed Consent not given
    Criteri di esclusione per la fase di screening dello studio (n=1500)
    1. Nessun disturbo polmonare associato alla fibrosi cistica
    2. Età < 8 anni e =18 anni
    3. Non in grado di fornire l’espettorato
    4. Mancato rilascio del Consenso informato

    Criteri di esclusione per la fase con posaconazolo (n=135)
    1. Età <8 anni e =18 anni.
    2. Impossibilità ad eseguire il test di funzionalità polmonare (FEV1%).
    3. Impossibilità a produrre un campione di espettorato.
    4. Condizione clinicamente instabile con cambiamenti significativi nella funzionalità polmonare o peggioramento significativo dei sintomi respiratori.
    5. Impossibilità di tollerare farmaci per via orale.
    6. Coltura di Aspergillus resistente all'azolo.
    7. Ipersensibilità nota a itraconazolo/posaconazolo/eccipienti
    8. Pazienti che ricevono inibitori della terfenadina, alcaloidi dell'ergot, astemizolo, cisapride, pimozide, alofantrina, chinidina o HMG-CoA reduttasi metabolizzati dal CYP3A4.
    9. Pazienti che ricevono Omalizumab.
    10. Trattamento con antifungini nell’ultimo mese
    11. Intervallo QT ridotto o allungato.
    12. Insufficienza cardiaca.
    13. ALT = 200 U / L
    14. AST = 225 U / L
    15. Fosfatasi alcalina = 460 U / L
    16. Bilirubina = 50 umol / L
    17. Gravidanza
    18. Inserimento nella lista dei trapianti o come destinatario di trapianti.
    19. Mancato rilascio del Consenso informato.
    E.5 End points
    E.5.1Primary end point(s)
    - For the dosing algorithm: The number of children and adolescents with CF and Aspergillus infection aged 8 to 17 years reaching the pre-defined area under the concentration time curve (AUC) of posaconazole at the first assessment (between day 5 and 10) based on the adult reference concentrations for treatment of susceptible pathogens.
    - For clinical efficacy: The number of children with negative sputum sample for Aspergillus infection at 3 months.
    ¿ Per l’algoritmo di dosaggio: la percentuale di bambini e adolescenti con CF e infezione da Aspergillus di età compresa tra gli 8 e i 17 anni che raggiungono l’area predefinita sotto la curva di concentrazione/tempo (AUC) di posaconazolo alla prima valutazione (tra il giorno 5 e il giorno 10), basato sulla concentrazione di riferimento per gli adulti per il trattamento di agenti patogeni sensibili.
    ¿ Per l’efficacia clinica: il numero di bambini con campione di espettorato negativo per infezione da Aspergillus a 3 mesi.
    E.5.1.1Timepoint(s) of evaluation of this end point
    - For the dosing algorithm: between day 5 and 10
    - For clinical efficacy: 3 months.
    Per l’algoritmo di dosaggio:Tra il giorno 5 e il giorno 10

    Per l’efficacia clinica: 3 mesi
    E.5.2Secondary end point(s)
    -The number of children and adolescents with CF and Aspergillus infection aged 8 to 17 years reaching the pre-defined area under the concentration time curve (AUC) of posaconazole at the second and third assessment (day 21-35 and EOT) based on the adult reference concentrations for treatment of susceptible pathogens.
    - To assess the safety and tolerability of posaconazole.
    - To assess any relation between posaconazole exposure and toxicity.
    - Forced expiratory volume (FEV1) at 3, 6 and 12 months post randomisation to evaluate if posaconazole is beneficial for CF-related Aspergillus infection.
    - Aspergillus in the airways (positive sputum culture) 6 and 12 months post randomisation to evaluate if posaconazole is able to clear CF-related Aspergillus infection.
    - Levels of Aspergillus specific IgG and IgE 3, 6 and 12 months post randomisation to evaluate if posaconazole is able to normalize those levels.
    - Airway inflammation as measured by proteome profiling of sputum at baseline and 3 months post randomisation to evaluate if posaconazole is able to dampen inflammation in CF-related Aspergillus infection.
    - Clinical disease severity (as measured by pulmonary exacerbation rate, days on antibiotics and corticosteroids, hospital admissions, change in FEV1, change in BMI) 3, 6 and 15 months post randomisation to evaluate if posaconazole has a beneficial clinical effect.
    - Patient reported outcomes 3, 6 and 12 months post randomisation to evaluate if posaconazole has a beneficial effect on people’s life.
    ¿ Il numero di bambini ed adolescenti con infezione da FC e Aspergillus da 8 a 17 anni che raggiungono l'area predefinita sotto la curva di concentrazione/tempo (AUC) di posaconazolo alla seconda e terza valutazione (giorno 21-35, EOT) in base alle concentrazioni di riferimento per adulti per il trattamento di agenti patogeni sensibili.
    ¿ Valutare la sicurezza e la tollerabilità del posaconazolo.
    ¿ Valutare qualsiasi relazione tra l’esposizione al posaconazolo e tossicità.
    ¿ Misurazione del volume espiratorio forzato (FEV1) a 3, 6 e 12 mesi dopo la randomizzazione per valutare se il posaconazolo è efficace per il trattamento dell'infezione da Aspergillus correlata alla FC.
    ¿ Valutare la presenza di Aspergillus nelle vie aeree (cultura dell'espettorato positivo) a 3, 6 e 12 mesi dopo la randomizzazione per valutare se il posaconazolo è in grado di eliminare l'infezione da Aspergillus correlata alla FC.
    ¿ Misurare i livelli di IgG e IgE specifiche di Aspergillus a 3, 6 e 12 mesi dopo la randomizzazione per valutare se il posaconazolo è in grado di normalizzare tali livelli.
    ¿ Valutare l’infiammazione delle vie aeree misurata mediante profilazione del proteoma dell'espettorato al basale e 3 mesi dopo la randomizzazione, per valutare se il posaconazolo è in grado di attenuare l'infiammazione nell'infezione da Aspergillus correlata alla FC.
    ¿ Valutare la gravità della malattia clinica (misurata in base al tasso di esacerbazione polmonare, giorni di trattamento con antibiotici ecorticosteroidi, ricoveri ospedalieri, variazione del FEV1, cambiamenti del BMI, anomalie della TAC toracica) a 3, 6 e 12 mesi dopo la randomizzazione per valutare se il posaconazolo ha un effetto benefico.
    ¿ Valutare i risultati del questionario sulla qualità della vita compilato dai pazienti a 3, 6 e 12 mesi dopo la randomizzazione per valutare se il posaconazolo ha un effetto benefico (CFQR convalidato).
    E.5.2.1Timepoint(s) of evaluation of this end point
    -Day 21-35
    -3, 6 and 12 months post randomisation
    -Giorno 21-35
    -3, 6 e 12 mesi dopo la randomizzazione
    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 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) Yes
    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 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) No
    E.8.2.2Placebo No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    na
    na
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA29
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Czech Republic
    France
    Germany
    Greece
    Ireland
    Italy
    Netherlands
    Portugal
    Spain
    Sweden
    Switzerland
    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 years3
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months4
    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.1.1.1In 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: 67
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 68
    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
    Children
    Bambini
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state32
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 115
    F.4.2.2In the whole clinical trial 135
    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)
    NA
    NA
    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 Decision2020-10-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-10-21
    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