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    Summary
    EudraCT Number:2019-004511-31
    Sponsor's Protocol Code Number:cASPerCF_2007_OPBG_2019
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2020-10-06
    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 ConcernedUK - MHRA
    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.
    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 dosingregimen for children and adolescents with cystic fibrosis and Aspergillus infection.
    A.3.2Name or abbreviated title of the trial where available
    cASPerCF
    A.4.1Sponsor's protocol code numbercASPerCF_2007_OPBG_2019
    A.5.4Other Identifiers
    Name:EUDRACTNumber:2019-004511-31
    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 SponsorBambino Gesù Children's Hospital
    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.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBambino Gesù Children's Hospital
    B.5.2Functional name of contact pointA.R. INFETTIVOLOGIA E SVILUPPO DI F
    B.5.3 Address:
    B.5.3.1Street Addresspiazza S.Onofrio, 4
    B.5.3.2Town/ cityRome
    B.5.3.3Post code00165
    B.5.3.4CountryItaly
    B.5.4Telephone number0039 06 68594053
    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 - oral suspension 40 mg/ml
    D.2.1.1.2Name of the Marketing Authorisation holderMerck Sharp & Dohme B.V
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 - oral suspension 40 mg/ml
    D.3.4Pharmaceutical form Oral suspension
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 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 100 mg
    D.2.1.1.2Name of the Marketing Authorisation holderMerck Sharp & Dohme B.V
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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 100 mg
    D.3.4Pharmaceutical form Gastro-resistant tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    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
    E.1.1.1Medical condition in easily understood language
    Aspergillus infection in people with cystic fibrosis
    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
    PK - To validate a paediatric posaconazole dosing regimen for children and adolescents with CF and Aspergillus infection. Clinical efficacy

    - To examine the efficacy of posaconazole versus usual care in children and adolescents with CF and Aspergillus infection in terms of clearance of Aspergillus from the airways after three months.
    E.2.2Secondary objectives of the trial
    PK
    - To define in silico the optimal dose for patients with CF aged 8 - 17 years.
    - To define the detailed pharmacokinetics in a limited number of children and adolescents with CF and Aspergillus infection aged 8-17 yrs through an intensive PK sampling study

    Epidemiology, clinical efficacy and safety
    - To determine the prevalence of Aspergillus infection in children and adolescents aged 8 to 17 years.
    - To assess the safety and tolerability of posaconazole in children and adolescents with CF and Aspergillus infection.
    - To assess the clinical efficacy of posaconazole versus usual care in terms of (1) serological responses to Aspergillus; (2) airway inflammation, and (3) clinical outcomes (as measured by pulmonary exacerbation rate, days on antibiotics and corticosteroids, hospital admissions, quality of life, change in FEV1, change in BMI, CT- chest abnormalities)
    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/or abnormal sweat test and clinical phenotype of lung disease)
    2. Age ≥ 8 yrs and < 18 yrs
    3. Body weight ≥ 20 kg
    4. Able to produce a sputum sample (spontaneous or induced sputum)
    5. 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. Body weight ≥ 20 kg
    4. Presence of Aspergillus infection as defined for this study
    5. Clinically stable condition without a significant change in lung function (FEV1 +/- 10%) or significant worsening of respiratory symptoms over the previous month
    6. Able to perform lung function test (FEV1%)
    7. Able to produce a sputum sample (spontaneous or induced sputum)
    8. Informed Consent given
    9. If female and of childbearing age must be using highly effective contraception (and must agree to continue for 7 days after the last dose of investigational medicinal product [IMP]) [1[

    Footnote: [1] Highly effective contraception is defined as one of the following: combined (estrogen and progestogen containing) hormonal
    contraception associated with inhibition of ovulation (oral, injectable or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable); intrauterine device (IUD); intrauterine hormone-releasing system ( IUS); bilateral tubal occlusion; vasectomised partner; sexual abstinence (defined as refraining from heterosexual intercourse when it is in line with the preferred and usual lifestyle of the subject)


    E.4Principal exclusion criteria
    Exclusion criteria for the screening phase:
    1. Non-CF lung disorder
    2. Age < 8 yrs of age or ≥ 18 yrs of age
    3. Body weight < 20 kg
    4. Not able to provide sputum sample
    5. Informed Consent not given

    Exclusion criteria for the intervention phase:
    1. Non-CF lung disorder
    2. Age < 8 yrs or ≥ 18 yrs
    3. Body weight < 20 kg
    4. Not able to perform lung function test (FEV1%)
    5. Unable to produce a sputum sample (spontaneous or induced sputum)
    6. Clinically unstable condition with significant change in lung function or significant worsening of respiratory symptoms
    7. Unable to tolerate oral medication
    8. Known hypersensitivity to itraconazole or posaconazole, or it’s excipients.
    9. On active transplant list or transplant recipient
    10. Azole resistant Aspergillus sp. cultured
    11. Patients receiving terfenadine, ergot alkaloids, astemizole, cisapride, pimozide, halofantrine, quinidine, or HMG-CoA reductase inhibitors metabolised through CYP3A4 (eg. simvastatin, lovastatin, and atorvastatin)
    12. Patients receiving omalizumab
    13. Received systemic mould-active antifungals in the last month
    14. Shortened or elongated QT interval
    15. Cardiac failure
    16. ALT ≥ 200 U/L
    17. AST ≥ 225 U/L
    18. Alkaline phosphatase ≥ 460 U/L
    19. Bilirubin ≥ 50 umol/L
    20. eGFR < 20 ml/min/1.73 m2 (calculated with the Schwartz formula)
    21. Patients with known glucose-galactose malabsorption problems
    22. Pregnancy [2] or breastfeeding
    23. Females of childbearing age who do not intend to use contraception measures.
    24. Informed Consent not given


    Footnote: [2] For females of child bearing age this must be confirmed by a pregnancy test within 10 days prior to randomizatio
    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
    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
    E.5.2Secondary end point(s)
    PK
    -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, EOT) based on the adult reference concentrations for treatment of susceptible pathogens.

    Epidemiology, clinical efficacy and safety
    - The proportion of children and adolescents aged 8 to 17 years with CF who screened positive for Aspergillus infection - The proportion of participants experiencing AEs and SAEs
    - Dose-response relationships 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) 3, 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, CT-chest abnormalities) 3, 6 and 12 months post randomisation to evaluate if posaconazole has a beneficial effect.
    - Patient reported outcomes 3, 6 and 12 months post randomisation to evaluate if posaconazole has a beneficial effect (validated CFQR).
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Day 21-35
    - 3, 6 and 12 months post randonisation
    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
    pharmacokinetics and dose finding study
    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
    There is no comparator - two formulations of the same IMP are being used but they are not compared
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA35
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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
    Final Data Lock. This will allow for data cleaning.
    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.1.1Number of subjects for this age range: 135
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    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.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state38
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 135
    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)
    This treatment Posaconazole (brand name Noxafil®) is available in the NHS however, is not currently indicted for use in children and their clinician will decide whether it is appropriate to prescribe after the end of the study.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-11-03
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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