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    Summary
    EudraCT Number:2011-000441-20
    Sponsor's Protocol Code Number:TR02-108
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-04-20
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2011-000441-20
    A.3Full title of the trial
    Randomized, open-label, active-controlled, multicenter study to assess the efficacy, safety and tolerability of Arikace™ in Cystic Fibrosis patients with chronic infection due to Pseudomonas aeruginosa
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy, safety and tolerability of Arikace™ compared to TOBI in Cystic Fibrosis patients with chronic infection due to Pseudomonas aeruginosa
    A.4.1Sponsor's protocol code numberTR02-108
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01315678
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/31/2011
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorInsmed Incorporated
    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 supportInsmed Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationInsmed Limited
    B.5.2Functional name of contact pointDenise Rue
    B.5.3 Address:
    B.5.3.1Street Address5-10 St. Paul's Churchyard
    B.5.3.2Town/ cityLondon
    B.5.3.3Post codeEC4M 8AL
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+1732997 4526
    B.5.5Fax number+1732997 4599
    B.5.6E-mailcf@insmed.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 No
    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/3/06/387
    D.3 Description of the IMP
    D.3.1Product nameLiposomal Amikacin (Arikace™)
    D.3.4Pharmaceutical form Nebuliser suspension
    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 INNAMIKACIN SULFATE
    D.3.9.1CAS number 39831555
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number70
    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 Yes
    D.3.11.13.1Other medicinal product typeSterile, liposomal dispersion of amikacin sulfate.
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleComparator
    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 300mg/5ml 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 Yes
    D.2.5.1Orphan drug designation numberEU/3/03/140
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Nebuliser solution
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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.3.10 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 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
    Pseudomonas aeruginosa pulmonary infection / colonisation in patients with cystic fibrosis
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level PT
    E.1.2Classification code 10011763
    E.1.2Term Cystic fibrosis lung
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The objectives of the study are to evaluate the efficacy, safety and tolerability of 3 cycles (28 days on-treatment and 28 days off treatment) of Arikace™ therapy. Change in pulmonary function, time to first pulmonary exacerbation, need for antipseudomonal antibiotics, change in sputum quantitative microbiology, hospitalization, patient reported symptoms/outcomes, and time off work/school will be assessed. Safety and tolerability will be evaluated throughout the study.
    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Pharmacokinetic assessments will be conducted in a subgroup of study subjects who consent to PK evaluation.

    The objective will be to characterize the pharmacokinetics, including bioavailability, and systemic exposure of amikacin for inhalation (ArikaceTM) in CF subjects.
    E.3Principal inclusion criteria
    1) Written informed consent or assent obtained from the patient, parent or legal guardian prior to the performance of any study related procedures
    2) Male or female study subjects ≥ 6 years of age (or older, if restricted by the local IRB/EC) at Screening
    3) Diagnosis of CF confirmed by a positive sweat test > 60 mEq/liter or > 60 mmol/L or by DNA analysis revealing both mutated alleles consistent with CF disease
    4) History of chronic infection with Pa confirmed by three documented positive cultures for Pa within the 2 years prior to Screening with at least one obtained within 6 months prior to Screening. The cultures could be obtained from the following respiratory secretions: sputum, deep throat swabs, or broncho-alveolar lavage fluid specimens
    5) Sputum culture positive for Pa at Screening
    6) Forced expiratory volume in 1 second (FEV1) ≥ 25% of predicted value at Screening using spirometer provided by sponsor
    7) Oxygen saturation (SaO2) ≥ 90% while breathing room air at Screening
    8) Ability to comply with study drug use, study visits, and study procedures as judged by the Investigator
    9) Ability to expectorate ≥ 0.4 mL of sputum
    10) Willingness to have specimens stored (no genetic testing)
    11) Women of childbearing potential must have a negative result on their serum pregnancy test at Screening and use reliable methods of contraception (e.g., abstinence, hormonal or barrier methods, partner sterilization, or IUD) throughout the study duration. Women not of childbearing potential are defined as prepubescent, postmenopausal (i.e., amenorrhea for at least 1 year), or surgically or naturally sterile
    E.4Principal exclusion criteria
    1) Forced Expiratory Volume in 1 second (FEV1) <25% of predicted at Screening using spirometer provided by Insmed
    2) History of hypersensitivity to aminoglycosides including tobramycin solution for inhalation
    3) Prior exposure to Arikace™ (including clinical study)
    4) History of major complications of lung disease (including atelectasis, pneumothorax, major pleural effusion), within 8 weeks prior to Screening
    5) Hemoptysis of ≥60 mL in a 24-hour period within 4 weeks prior to Screening
    6) History of acute pulmonary exacerbation requiring antibiotic treatment within 4 weeks prior to Screening
    7) History of upper respiratory tract infection within 2 weeks prior to Screening
    8) Use of antipseudomonal antibiotics (IV antibiotics, inhalation antibiotics, or oral) within 4 weeks prior to Day 1
    9) Radiologic finding of new pulmonary infiltrate(s) within 3 months prior to Screening, or presence of other abnormalities suggesting clinically significant active pulmonary disease other than CF
    10) Initiation of chronic therapy (e.g., TOBI®, Colomycin®, other inhaled antibiotics, high-dose ibuprofen, bronchodilators, inhaled anti-inflammatory agents including steroids, low dose maintenance steroids, rhDNase, hypertonic saline, macrolides) within 4 weeks prior to study Day 1
    11) History of positive culture for Burkholderia cepacia within 2 years prior to Screening
    12) History of pulmonary tuberculosis or non-tuberculous mycobacterial lung disease treated within 2 years prior to Screening or requiring treatment at the time of screening
    13) History of Allergic Broncho-Pulmonary Aspergillosis requiring systemic steroid treatment or any other condition requiring systemic steroids at a dose ≥ equivalent of 10 mg/day of prednisone within 3 months prior to Screening
    14) Presence of any clinically significant cardiac disease as determined by investigator and/or, if QTc data are available, QTc prolongation > 450 msec (0.450 seconds) for males or QTc > 470 msec (0.470 seconds) for females or QTc prolongation > 440 msec (0.440 seconds) for all subjects 6 to 12 years of age
    15) Acquired and primary immunodeficiency syndromes
    16) History of hepatitis C or chronic active hepatitis B infection
    17) Active pulmonary malignancy (primary or metastatic) or any malignancy requiring chemotherapy or radiation therapy within one year prior to Screening or anticipated during the study period
    18) History of biliary cirrhosis with portal hypertension
    19) History of lung transplantation
    20) Elevated AST, ALT or GGT ≥ 3× the upper limit of normal (ULN) at Screening
    21) Absolute neutrophils count ≤ 1000 at Screening
    22) Serum creatinine > 2× ULN at Screening
    23) Daily, continuous oxygen supplementation
    24) Supplemental oxygen requirement of greater than 2 L/min at night
    25) Administration of any investigational products within 8 weeks prior to study Day 1
    26) Psychotic, addictive or other disorder limiting the ability to provide informed consent or to comply with study requirements.
    27) History of alcohol, medication, or illicit drug abuse within the 1 year prior to Screening
    28) Smoking tobacco or any substance within 6 months prior to screening or anticipated inability to refrain from smoking throughout the study
    29) Positive pregnancy test or lactation at Screening. All women of child bearing potential will be tested for pregnancy. Women not of childbearing potential are defined as prepubescent, postmenopausal (i.e., amenorrhea for at least 1 year), or surgically or naturally sterile
    30) Any condition which in the opinion of the investigator interferes with ability to safely complete the study or adhere to study requirements
    E.5 End points
    E.5.1Primary end point(s)
    The relative change in Forced Expiratory Volume in 1 second (FEV1 (liters)) from baseline (Day 1) to the end of the study (Day 168)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Days 1, 168
    E.5.2Secondary end point(s)
    1) Relative change in FEV1 (liters) from Day 1 to the end of study
    2) Relative change in FEV1 (% predicted) from Day 1 to the end of study
    3) Time to first protocol defined pulmonary exacerbation and proportion of subjects experiencing a protocol defined pulmonary exacerbation
    4) Time to first antipseudomonal antibiotic treatment for protocol defined pulmonary exacerbation
    5) Change in log CFU Pa from baseline throughout study
    6) Time to and number of hospitalizations for any cause
    7) Change in respiratory symptoms from baseline throughout study as measured by the Cystic Fibrosis Questionnaire-Revised (CFQ-R)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) Day 1, 14, 28, 57, 84, 113 and 140
    2) Day 1, 14, 28, 57, 84, 113, 140 and 168
    5) Day 1, 14, 28, 57, 84, 113, 140, 168
    7) Day 1, 14, 28, 57, 84, 113, 140 and 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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic Yes
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    tolerability
    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 Yes
    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) Yes
    E.8.2.2Placebo No
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA70
    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
    Austria
    Belgium
    Canada
    Denmark
    France
    Germany
    Greece
    Hungary
    Ireland
    Italy
    Netherlands
    Poland
    Slovakia
    Spain
    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
    Last visit of the last patient.
    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 months11
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months11
    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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 40
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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
    In case of Patients under the age of 18, consent should be also given by parents/guardians.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state25
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 241
    F.4.2.2In the whole clinical trial 300
    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 - Therapeutics Development Network
    G.4.3.4Network Country United States
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation European Cystic Fibrosis Society - Clinical Trial Network
    G.4.3.4Network Country Netherlands
    G.4 Investigator Network to be involved in the Trial: 3
    G.4.1Name of Organisation Cystic Fibrosis - National Knowledge Service (UK CF-NKS)
    G.4.3.4Network Country United Kingdom
    G.4 Investigator Network to be involved in the Trial: 4
    G.4.1Name of Organisation Cystische Fibrose - Netzwerk Klinische Studien (German CF-NKS)
    G.4.3.4Network Country Germany
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-06-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-06-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2013-08-27
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