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    Summary
    EudraCT Number:2016-004318-82
    Sponsor's Protocol Code Number:CTBM100CUS03
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2017-05-15
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2016-004318-82
    A.3Full title of the trial
    Tobramycin Inhalation Powder (TIP) Administered Once Daily Continuously Versus TIP Administered BID in 28 Day on / 28 Day Off Cycles
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study comparing Tobramycin Inhalation Powder (TIP) administered once daily continuously versus TIP administered BID in 28 day on / 28 day off cycles for the treatment of pulmonary Pseudomonas aeruginosa in patients with cystic fibrosis
    A.4.1Sponsor's protocol code numberCTBM100CUS03
    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 SponsorNovartis Pharmaceuticals 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 supportNovartis Pharmaceuticals Corporation
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma AG
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressForum 1 Novartis AG
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4056
    B.5.3.4CountrySwitzerland
    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 Podhaler
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Pharmaceuticals Corporation
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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/3/03/140
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Capsule
    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.3.9.3Other descriptive nameTOBRAMYCIN
    D.3.9.4EV Substance CodeSUB11134MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number112
    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 Yes
    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
    E.1.1.1Medical condition in easily understood language
    Cystic Fibrosis
    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 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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10021860
    E.1.2Term Infection Pseudomonas aeruginosa
    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 provide efficacy and safety data comparing two dosing schedules of Tobramycin Inhalation Powder (TIP) for the treatment of pulmonary Pseudomonas aeruginosa in patients with cystic fibrosis.
    E.2.2Secondary objectives of the trial
    To provide efficacy and safety data comparing two dosing schedules of Tobramycin Inhalation Powder (TIP) for the treatment of pulmonary Pseudomonas aeruginosa in patients with cystic fibrosis.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Provide written informed consent, HIPPA (Health Insurance Portability and Accountability Act) authorization (where applicable), and assent (as appropriate) prior to the performance of any study-related procedure.
    Confirmed diagnosis of CF
    3.FEV1 at screening (Visit 1) ≥25% and ≤ 80% of normal predicted values for age, sex, and height
    4.P. aeruginosa must be present within 6 months prior to screening and at screening
    5.Able to comply with all protocol requirements
    6.Clinically stable in the opinion of the investigator
    E.4Principal exclusion criteria
    History of Burkholderia cenocepacia (Bcc) complex within 2 years prior to screening and/or Bcc complex at screening
    2.Hemoptysis more than 60 cc at any time within 30 days prior to study drug administration
    3.History of hearing loss or chronic tinnitus deemed clinically significant by the investigator
    4.Serum creatinine 2 mg/dL or greater, BUN 40 mg/dL or greater, or an abnormal urinalysis defined as 2+ or greater proteinuria at screening
    5.Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics
    6.Patients who are unable to discontinue previously received inhaled antibiotic regimen(s) (inhaled antibiotics are not allowed other than study drug)
    7.Use of inhaled aminoglycosides within 28 days prior to study drug administration (Visit 2)
    8.Use of systemic anti-pseudomonal antibiotics within 28 days prior to study drug administration
    9.Use of loop diuretics within 7 days prior to study drug administration
    10.Administration of any investigational drug within 30 days prior to enrollment or 5 half-lives, whichever is longer
    11.Signs and symptoms of acute pulmonary disease, e.g , pneumonia, pneumothorax
    12.Hospitalization during the baseline visit
    13.History of malignancy
    14.Patients with clinically significant laboratory abnormalities (not associated with the study indication) at screening
    15.Patients with other clinically significant conditions (not associated with the study indication) which might interfere with the assessment of this study
    16.Patients or caregivers with a history of noncompliance to medical regimens and patients or caregivers who are considered potentially unreliable
    17.Pregnant or nursing (lactating) women
    18.Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle, or sexual orientation precludes intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, UNLESS they are using two birth control methods.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in Forced Expiratory Volume in 1 second ( FEV1) percent predicted [ Time Frame: Baseline and Day 168 ]
    The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Time Frame: Baseline and Day 168
    E.5.2Secondary end point(s)
    Percent change from baseline in Forced Expiratory Volume in 1 second (FEV1) percent predicted [ Time Frame: Baseline and Day 168 ]
    The Forced Expiratory Volume in 1 second (FEV1) percent predicted expresses FEV1 as a percentage of the "predicted values" for participants of similar characteristics (height, age, sex, and sometimes race and weight). A positive change from baseline in FEV1 percent predicted indicates improvement in lung function.

    •Percent change from baseline in Forced Vital Capacity (FVC) percent predicted [ Time Frame: Baseline and Day 168 ]
    Forced Vital Capacity (FVC) is the amount of air which can be forcibly exhaled from the lungs after taking the deepest breath possible. FVC will be assessed via spirometry. A positive change from baseline in FVC indicates improvement in lung function.

    •Percent change from baseline in forced expiratory flow (FEF) 25%-75% predicted [ Time Frame: Baseline and day 168 ]
    The Forced Expiratory Flow (FEF) 25%-75% measurement describes the amount of air expelled from the lungs during the middle half (25% - 75%) of the forced vital capacity test and is measured using spirometry. A positive change from baseline in FEF indicates improvement in lung function. The predicted percent will be assessed.

    •Change from baseline in Pseudomonas aeruginosa sputum density [ Time Frame: Baseline and day 168 ]
    Change from baseline in Pseudomonas aeruginosa sputum density will be measured by log10 colony forming units per gram of sputum.

    •Time to first hospitalization due to respiratory-related events [ Time Frame: Day 1 to day 168 ]
    Time to the first hospitalization due to respiratory-related events (number of days) per patient.

    •Percentage of patients with hospitalizations due to respiratory-related events [ Time Frame: Day 1 to day 168 ]
    Percentage of patients with hospitalization due to respiratory-related events

    •Length of hospital stay due to respiratory-related events [ Time Frame: Day 1 to day 168 ]
    The number of days in length of hospital stay per patient due to respiratory-related events will be measured.

    •Time to first usage of anti-pseudomonal antibiotic [ Time Frame: Day 1 to day 168 ]
    Time to first usage of anti-pseudomonal antibiotic per patient will be assessed by number of days

    •Percentage of patients who use anti-pseudomonal antibiotic [ Time Frame: Day 1 to day 168 ]
    Percentage of patients who use anti-pseudomonal antibiotic will be assessed.

    •Duration of use of anti-pseudomonal antibiotic [ Time Frame: Day 1 to day 168 ]
    Number of days of use of anti-pseudomonal antibiotic per patient will be assessed.

    •Change from baseline in tobramycin minimal inhibitory concentration for Pseudomonas aeruginosa [ Time Frame: Baseline and day 168 ]
    Change from baseline in tobramycin minimal inhibitory concentration for Pseudomonas aeruginosa will be measured by laboratory testing.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Time Frame: Day 1 to day 168
    Time Frame: Baseline and day 168
    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 No
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 No
    E.8.1.5Parallel group Yes
    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
    TOBI Podhaler cyclical use
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    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.8.9.2In all countries concerned by the trial months7
    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: 3
    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: 1
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 28
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 200
    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)
    TIP administered once daily on a continuous (ie, non-cycled) basis, versus TIP administered BID in 28 day on / 28 day off cycles.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Cystic Fibrosis Foundation Therapeutics, Inc.
    G.4.3.4Network Country United States
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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