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    Summary
    EudraCT Number:2009-016590-15
    Sponsor's Protocol Code Number:CTBM100C2304
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-10-11
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2009-016590-15
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Crossover Multi-Center Study to Assess the Efficacy
    and Safety of Inhaled Tobramycin Nebuliser Solution (TOBI®) for the Treatment of Early Infections of P.
    aeruginosa in Cystic Fibrosis Subjects Aged from 3 Months to less than 7 years.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to assess the treatment with nebulised tobramycin in terms of safety and ability to kill Pseudomonas bacteria in the lungs of cystic fibrosis patients aged 3 months to 6 years included
    A.4.1Sponsor's protocol code numberCTBM100C2304
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/146/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNovartis Pharma Services AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma Services AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationClinical Trial Information Desk
    B.5.2Functional name of contact pointNovartis Pharma Services AG
    B.5.3 Address:
    B.5.3.1Street AddressForum 1, Novartis Campus
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4056
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+41 61 3241 111
    B.5.5Fax number+41 613248 001
    B.5.6E-mailclinicaltrial.enquiries@novartis.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 TOBI 300 mg / 5 mL nebuliser solution
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Pharmaceuticals UK Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 Nebuliser solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPInhalation use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTobramycin
    D.3.9.1CAS number 32986-56-4
    D.3.9.2Current sponsor codeTBM100
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    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
    D.8.3Pharmaceutical form of the placeboNebuliser solution
    D.8.4Route of administration of the placeboInhalation use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Lung colonisation with Pseudomonas aeruginosa in cystic fibrosis patients
    E.1.1.1Medical condition in easily understood language
    Presence of Pseudomonas bacteria in the lung of patients 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 14.1
    E.1.2Level PT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10068292
    E.1.2Term Pseudomonas colonization
    E.1.2System Organ Class 100000004862
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10068297
    E.1.2Term Pseudomonas colonisation
    E.1.2System Organ Class 100000004862
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To estimate the proportion of subjects free from any strain of P. aeruginosa assessed by sputum / swab culture at Day 29, i.e. after completion of a 28-day treatment period with either TOBI or placebo solution inhaled twice daily.
    E.2.2Secondary objectives of the trial
    • To assess the safety profile of TOBI inhaled twice daily or placebo throughout the treatment period in subjects in this age group.
    • To estimate the proportion of subjects free from any strain of P. aeruginosa assessed by sputum / swab culture 28 days after termination of the 2nd treatment cycle (day 91) with either TOBI inhaled twice daily for 28 days or placebo.
    • To estimate the proportion of subjects free from any strain of P. aeruginosa assessed by sputum / swab culture 28 days after termination of treatment with either TOBI inhaled twice daily for 28 days or placebo.
    • To assess the pharmacokinetics of TOBI in this age group
    • To assess lung function in the subset of subjects able to reliably perform spirometry
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent given by the parent/legal guardian on behalf of the subject.

    2. Diagnosis of CF by one or more clinical features of CF:
    • a documented sweat chloride test of > 60 mEq/L by quantitative pilocarpine iontophoresis
    • genotype with two identifiable CF-causing mutations
    • a positive newborn screening for CF

    3. Male and female subjects aged 3 months to less than 7 years of age at the time of screening.

    4. Early lower respiratory tract infection with P. aeruginosa, defined by either of the following:
    a. The first time P. aeruginosa isolated
    b. P. aeruginosa isolated after at least 1 year of negative cultures (documented with at least two negative cultures during the latter 1-year period, with no positive culture during that period)

    5. Positive P. aeruginosa culture isolated from sputum, deep throat swab or nasopharyngeal aspiration specimens collected and tested from routine clinical culture performed at local site laboratory within 1 months prior to randomization

    6. Able to comply with all protocol requirements (except spirometry where not applicable)
    Clinically stable in the opinion of the investigator
    E.4Principal exclusion criteria
    1. Any oral or iv anti-pseudomonal antibiotic treatment within 1 year prior to randomization

    2. Serum creatinine above the upper limit of the normal range for age documented from at least one analysis performed at site laboratory within 6 month prior to randomization

    3. Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics.

    4. Signs and symptoms of acute pulmonary disease, e.g., pneumonia, pneumothorax.

    5. Administration of any investigational drug within 30 days or 5 half-lives prior to enrollment, whichever is longer.

    6. Administration of loop diuretics within 7 days prior to study drug administration.

    7. Personal/family history of abnormal hearing

    8. Current (continuing, present at screening) or persisting abnormal result from audiology testing (defined as either a unilateral pure-tone audiometry test showing a threshold elevation > 20 dB at any frequency across the frequency range 0.25 kHz to 8 kHz or the absence of emission at the evoked otoacoustic emission test).

    9. History of sputum culture, throat swab, or lower respiratory specimen culture yielding Burkholderia cepacia (B. cepacia) within 2 years prior to screening and/or sputum culture yielding B. cepacia at screening.

    10. Hemoptysis which is clinically significant based on the subjects age and clinical status within 30 days prior to study drug administration.

    11. History of malignancy of any organ system treated or untreated, regardless of whether there is evidence of local recurrence or metastases,

    12. Subjects with clinically significant laboratory abnormalities including congenital diseases other than CF (not associated with the study indication) documented from at least one laboratory chemistry and haematology analysis performed within 1 year prior to randomization

    13. Subjects with other clinically significant conditions (not associated with the study indication) at screening which might interfere with the assessment of this study

    14. Subjects or caregivers with a history of noncompliance to medical regimens and subjects or caregivers who are considered potentially unreliable.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of patient P aeruginosa free at day 29.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Day 29
    E.5.2Secondary end point(s)
    • Proportion of subjects with P. aeruginosa free 28 days after last study medication,

    • Proportion of subjects with P. aeruginosa free at day 91 (end of treatment phase)

    • Relative change in FEV1, FVC and FEF 25-75 (all values as % predicted) from baseline to final visit (end of treatment phase), at selected sites in subjects able to perform PFT.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Stated directly in endpoints description above. Please also refer to assessment schedule in full protocol.
    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) No
    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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Blinded cross-over only if negative Pa culture at day 29. Open label active if Pa positive culture
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    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 EEA30
    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
    France
    Germany
    Greece
    Hungary
    Italy
    Poland
    Romania
    Russian Federation
    Switzerland
    United States
    Egypt
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months9
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial 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: 50
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 45
    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
    Children aged 3 months to less than 7 years
    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 34
    F.4.2.2In the whole clinical trial 50
    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)
    A 12-month follow up period is included in the protocol after the 3-month randomised period. During this followup
    patients are tested quarterly for Pa culture receive 28-day TOBI treatment upon recurrence of Pa colonisation.
    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 Decision2013-12-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-10-17
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-06-24
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