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    Summary
    EudraCT Number:2014-003087-20
    Sponsor's Protocol Code Number:800_OPBG_2014
    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:2014-08-26
    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 number2014-003087-20
    A.3Full title of the trial
    Determination of Plasmatic and CSF Levels Of High Doses Of Micafungin in Neonates Suffering from Systemic Candidiasis and/or Candida meningitis.
    Determinazione dei livelli, nel sangue e nel liquido cefalorachidiano, di dosi elevate di micafungina in neonati affetti da Candida sistemica e/o meningite da Candida.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Determination of Levels of Micafungin in Neonates Suffering from Systemic Candidiasis and/or Candida meningitis.
    Determinazione dei livelli del farmaco micafungina in neonati che mostrano un infezione da Candida sistemica e/o meningite da Candida.
    A.4.1Sponsor's protocol code number800_OPBG_2014
    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
    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 supportAstellas Pharma S.p.A.
    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 Gesù
    B.5.2Functional name of contact pointCinzia Auriti
    B.5.3 Address:
    B.5.3.1Street AddressPiazza Sant’Onofrio 4
    B.5.3.2Town/ cityRome
    B.5.3.3Post code00165
    B.5.3.4CountryItaly
    B.5.4Telephone number+390668592347
    B.5.5Fax number+390668593916
    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 Mycamine
    D.2.1.1.2Name of the Marketing Authorisation holderAstellas Pharma Europe B.V.
    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.3.4Pharmaceutical form Powder 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 INNMICAFUNGIN
    D.3.9.1CAS number 235114-32-6
    D.3.9.4EV Substance CodeSUB16444MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number8
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Systemic Candidiasis and/or Candida Meningitis
    E.1.1.1Medical condition in easily understood language
    Infection by yeast called Candida that has spread into tissue and blood vessels and/or into the meninges
    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 17.0
    E.1.2Level LLT
    E.1.2Classification code 10027238
    E.1.2Term Meningitis fungal NOS
    E.1.2System Organ Class 100000004862
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level HLT
    E.1.2Classification code 10027237
    E.1.2Term Meningeal fungal infections
    E.1.2System Organ Class 100000004852
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10042941
    E.1.2Term Systemic fungal infection NOS
    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
    To study the pharmacokinetic profile of micafungin administered, at a dose of 8 mg/Kg/day, to neonates suffering from definite or suspected systemic Candidiasis.
    E.2.2Secondary objectives of the trial
    • To evaluate the proportion of success and of failure of the therapy with micafungin among treated neonates
    • To identify a conversion factor to relate plasma levels of micafungin into capillary and venous blood, measured through blood samples from the heel and from a peripheral vein, collected simultaneously
    • To evaluate the safety of Micafungin in neonates
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Infection by systemic Candidiasis, definite or suspected.
    a) Definite systemic Candidiasis is diagnosed in case of isolation of Candida from at least one sample collected from a normally sterile site (Blood, CSF, Urine, Peritoneal Fluid) and/or positivity to Candida through PCR (Septifast test), associated with at least one clinical symptom (fever or hypothermia, mottled skin, feeding difficulties, muscular hypotonia or hypertonia, apnoea crisis, bradycardia, tachycardia, hypotension, dyspnea, polypnea, desaturation) and one laboratory symptom (WBC ≤5000/mm3 or WBC ≥20.000/mm3, I/T ratio >2, Platelet count ≤100.000/mm3, C-reactive Protein >0,5 mg/dL, Standard Base Excess >-7 mmol/L, CSF pleocytosis-cells ≥ 6).
    b) Suspected systemic Candidiasis is diagnosed in case of worsening of clinical conditions while on therapy with antibiotics, isolation of Candida from at least two non contiguous sites (tracheal aspirate, gastric aspirate, faeces) associated with at least one clinical symptom (fever or hypothermia, mottled skin, feeding difficulties, muscular hypotonia or hypertonia, apnoea crisis, bradycardia, tachycardia, hypotension, dyspnea, polypnea, desaturation) and one laboratory symptom (WBC ≤5000/mm3 or WBC ≥20.000/mm3, I/T ratio >2, Platelet count ≤100.000/mm3, C-reactive Protein >0,5 mg/dL, Standard Base Excess >-7 mmol/L, CSF pleocytosis-cells ≥ 6) and positivity to test ELISA for the mannan antigen (≥250 pg/ml).
    2. Neonates affected by definite or suspected Candida meningitis and/or hydrocephalus due to Candida infection and/or bearing external ventricular derivation, until enrolment of 4 subjects with this characteristics.
    3. Parents of neonates, or their legal representative, able to consent and comply with protocol requirements.
    4. Survival expectation not inferior to 3 days.
    E.4Principal exclusion criteria
    1. Hepatic acute or chronic insufficiency with at least one of the following abnormal parameters:
    a. Ammonium > 85 µg/dL
    b. ALT/GPT > 40 UI/L
    c. AST/GOT > 40 UI/L
    d. Direct Bilirubin > 2mg/dL
    2. Baseline INR >5%
    3. Known allergy or hypersensitivity to echinocandins or any of the excipients present in the formulation of the IMP.
    4. Micafungin infusion already started.
    5. Neonates affected by definite or suspected Candida meningitis and/or hydrocephalus due to Candida infection and/or bearing external ventricular derivation, if already 4 subjects with this characteristics have been enrolled into the study.
    E.5 End points
    E.5.1Primary end point(s)
    • Quantification of micafungin levels into blood, through samples collected with capillary micro-method (draws from the heel). Blood draws are to be taken before infusion with the IMP and after 1, 2 and 8 hours from the end of the intravenous infusion, in one of the treatment days starting from the third and within the tenth.
    • Quantification of micafungin levels in the cerebrospinal fluid, through samples collected from 4 neonates with Candida meningitis, hydrocephalus due to Candida infection and/or with external ventricular derivation. CSF draws are to be taken before infusion with the IMP and after 1, 2 and 8 hours from the end of the intravenous infusion, in one of the treatment days starting from the third and within the tenth.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Between third and tenth day of therapy.
    E.5.2Secondary end point(s)
    1) To evaluate the percentage of success and failure of therapy with micafungin, among treated patients, on the basis of the following definitions:
    - In case of definite or suspected sepsis, the success of the therapy will be determined by survival associated with the negative results to Candida test of two consecutive blood cultures, performed starting from the beginning of treatment, or, only in case of suspected sepsis, determined by resolution of clinical and laboratory symptoms together with reduction of mannan antigen blood level (<250 pg/ml).
    - In case of definite or suspected sepsis, the failure of the therapy will be determined by death due to Candida sepsis, by confirmation of persistence of positive result to Candida test at 14th (± 2days) day from one blood culture performed in the previous days, by the need to add or switch to another antifungal agent and/or change of micafungin dosage for the resolution of infection at any time or, only in case of suspected sepsis, by the persistence of Candida colonization in the different indicated sites associated with the persistence of clinical and laboratory symptoms and with high blood level of the mannan antigen (≥250 pg/ml) at 14th day (± 2days) from the start of the therapy.
    - In case of Candida meningitis, the success of the therapy will be determined by survival associated with the negative results to Candida test of at least three consecutive CSF cultures, performed starting from the beginning of treatment, and resolution of clinical and laboratory symptoms.
    - In case of Candida meningitis, the failure of the therapy will be determined by death due to Candida meningitis, by persistence of Candida infection as resulted from confirmation of positive CSF culture after the 14th day of therapy (± 2days) or by the need to add or switch to another antifungal agent or dosage change of micafungin for the resolution of Candida infection at any time.
    - In case of neonates with hydrocephalus due to Candida infection and/or external ventricular derivation, the success of the therapy will be determined by survival associated with the negative results to Candida test of at least three consecutive CSF cultures, performed starting from the beginning of treatment.
    - In case of neonates with hydrocephalus due to Candida infection and/or external ventricular derivation, the failure of the therapy will be determined by death due to Candida infection, by need o add or switch to another antifungal agent or dosage change of micafungin for the resolution of Candida infection at any time or by the persistence of Candida infection as resulted from confirmation of positive CSF culture after from the 14th day of therapy (± 2days).
    2) For the pharmacokinetic assessment, in a sub-set of five neonates, blood draws will be collected simultaneously either via capillary micro-method (withdraw from the heel) and venous (withdraw from a peripheral vein) in order to compare the methods and identify a conversion factor between the levels of drug in capillary and venous blood, through simultaneous withdrawals.
    3) Evaluation of adverse events (serious and non serious) and adverse drug reactions throughout the duration of trial by assessment of physical examination, vital signs, 12-lead electrocardiogram. Serious and non serious adverse events will be collected from Day1 until 30 days after trial participation, where possible.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) 14 days to 21days
    2) Between third and tenth day of therapy.
    3) Throughout the treatment period of approximately 21 days.
    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 No
    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) No
    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) 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 concerned3
    E.8.5The trial involves multiple Member States No
    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 No
    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
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months30
    E.8.9.1In the Member State concerned days
    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: 30
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Yes
    F.1.1.2.1Number of subjects for this age range: 10
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 10
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 10
    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 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
    Neonates of various gestational age up to 90 days old
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state30
    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
    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 Decision2014-09-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-11-12
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-04-10
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