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    Summary
    EudraCT Number:2006-006693-24
    Sponsor's Protocol Code Number:Moli1901-010B
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2007-01-11
    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 ConcernedAustria - BASG
    A.2EudraCT number2006-006693-24
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Dose-finding Study to Evaluate the Efficacy and Safety of Aerosolized Moli1901 in Adolescents (12 Years of Age or Older) and Adults with Cystic Fibrosis
    A.4.1Sponsor's protocol code numberMoli1901-010B
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAOP Orphan Pharmaceuticals AG
    B.1.3.4CountryAustria
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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/3/02/120
    D.3 Description of the IMP
    D.3.1Product nameMoli1901 (2622U90, duramycin)
    D.3.2Product code Moli1901
    D.3.4Pharmaceutical form Inhalation vapour, solution
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPInhalation use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Information not present in EudraCT
    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 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 Information not present in EudraCT
    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 Information not present in EudraCT
    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 placeboInhalation vapour, 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
    Cystic fibrosis is the most common fatal inherited disease in the Caucasian population, affecting about 4 in 10.000 children. In cystic fibrosis chloride transport across the respiratory epithelium is deficient, so the mucus contains less water and its viscosity is abnormally increased. Moli1901 corrects the abnormal transport of chloride thereby reducing the formation of mucus plugs and improving clearance.
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level PT
    E.1.2Classification code 10011763
    E.1.2Term Cystic fibrosis lung
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Study objectives are to establish minimum effective dose (MED), optimal dose, and maximum safe dose (MSD) in this dose-finding study with three different dosage schedules:
    1. 2,5 mg/day (0.5 mg/ml; 5 ml) Moli1901, daily
    2. 2,5 mg/day (0.5 mg/ml; 5 ml) Moli1901, every other day
    3. 2,5 mg/day (0.5 mg/ml; 5 ml) Moli1901, twice a week
    Additionally, the tolerability of Moli1901 shall be investigated.
    Primary efficacy criterion (confirmatory evaluation): the change in the percentage of the predicted FEV1 (forced expiratory volume in first second) value
    E.2.2Secondary objectives of the trial
    Co-primary criterion (supportive evaluation): the ensemble of two subject reported clinical scores, which are physical dimensions of the self-report, disease-specific quality of life test, the cystic fibrosis questionnaire in the revised version.
    Secondary efficacy criteria:
    The change in the percentage of the predicted FVC (forced vital capacity)
    The change in the percentage of the predicted FEF25-75 (forced expiratory flow 25–75% of FVC)
    The change in the absolute values of FEV1 (forced expiratory volume in first second)
    Number of pulmonary exacerbations
    Number of hospitalization days due to pulmonary exacerbation
    Dose (cumulative dose) and duration of therapy needed during study participation to treat bronchial obstruction, infection or inflammation (i.e. antibiotics, mucolytics, anti-inflammatory drugs).
    Time to first pulmonary exacerbation
    Change in oxygen saturation as measured with pulse oximetry
    Quality of life questionnaire (cystic fibrosis questionnaire-revised)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Male or female subjects of 12 years of age or older
    2.Body mass index equal or above -2 SDS (Standard Deviation Score-According to CDC standards) for 12-19 year old patients and greater/equal 18.5 kg/m2 for adults 20 years old and older
    3.Have a confirmed diagnosis of cystic fibrosis:
    positive sweat chloride value greater/equal 60 mEq/l by quantitative pilocarpine iontophoresis (on at least 2 occasions, if no genotype was determined) and/or
    genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype
    Measurement of nasal potential difference could be considered as an additional diagnostic tool, if results of sweat chloride test and genotype test are not unambiguous. Nasal potential difference test can not replace any of the above tests and should not be interpreted in isolation.
    4.Have Screening FEV1 between 50% and 85% of predicted.
    5.Have oxygen saturation level measured by pulse oximetry (SpO2) greater 90 % on room air
    E.4Principal exclusion criteria
    1. Show bronchial hyperresponsiveness as known from previous visits e.g. the subject needs additional puffs of salbutamol (or other beta-2-agonists) more than twice daily. This also applies to subjects who have shown bronchoconstriction to previously administered inhalation therapies or a decrease of FEV1 greater/equal 20% during or after administration of placebo at the screening visit
    2.Have shown any evidence for unstable lung function, e.g. have had more than 15% FEV1 variation (difference between highest and lowest measured value in % of highest value measured) in the last 3 months before treatment starts (i.e. visit 2)
    3.Have a pulmonary disease such as pneumonia, tuberculosis, or lung cancer
    4.Have had an acute upper respiratory tract infection within the last 2 weeks
    5.Have had an acute lower respiratory tract infection (requiring antibiotics or hospitalization) within the last 4 weeks
    6.Have had an pulmonary exacerbation within the last 4 weeks
    7.Have had any changes from routine maintenance therapy within the last 4 weeks
    8.Have any scheduled changes to inhaled antibiotics regimen during the course of the study
    9.Receive or are planned to receive any treatment via “on-off” regimen (e.g. Tobramycin – TOBI®)
    10.Have received the last dose of any “on-off” treatment within the last 6 weeks
    11.Have any clinically significant liver, renal, cardiac (as defined by QTc >450 msec (or 0.450 seconds) for males or QTc> 470 msec (or 0.470 seconds) for females), neurological, or hematological disease
    12.Have allergic bronchopulmonary aspergillosis or colonization with Burkholderia cepacia
    13.Have poorly controlled diabetes mellitus
    14.Have smoked more than 3 cigarettes per day within the last 12 months.
    15.Have a history of alcohol (> 40g/day) or drug abuse
    16.Have participated in an investigational drug study (including Moli1901) within the last 8 weeks
    17.Are women of child bearing potential and refuse to use effective contraception or are pregnant or lactating
    18.Are unwilling to perform appropriate safe contraception for at least 6 months after investigational product administration
    E.5 End points
    E.5.1Primary end point(s)
    Change in the percentage of the predicted FEV1 (forced expiratory volume in first second) value
    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 Yes
    E.6.10Pharmacogenetic Information not present in EudraCT
    E.6.11Pharmacogenomic Information not present in EudraCT
    E.6.12Pharmacoeconomic Information not present in EudraCT
    E.6.13Others Information not present in EudraCT
    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) 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 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 Yes
    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 concerned2
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    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 months12
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months12
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 120
    F.4.2.2In the whole clinical trial 160
    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)
    Patients will continue with their routine cystic fibrosis treatment and will be eligible to participate in the next Moli1901 study after a wash-out period
    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 Decision2007-05-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2007-01-08
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2009-07-15
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