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    Summary
    EudraCT Number:2010-020413-90
    Sponsor's Protocol Code Number:VX09-809-102
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2010-10-15
    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
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2010-020413-90
    A.3Full title of the trial
    A Phase 2, Multicenter, Double-Blind, Placebo-Controlled, Multiple-Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of Lumacaftor Monotherapy, and Lumacaftor and Ivacaftor Combination Therapy in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
    A.4.1Sponsor's protocol code numberVX09-809-102
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01225211
    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 SponsorVertex Pharmaceuticals 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 supportVertex Pharmaceuticals Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVertex Pharmaceuticals Incorporated
    B.5.2Functional name of contact pointClinical Trials and Medical Info
    B.5.3 Address:
    B.5.3.1Street Address130 Waverly St
    B.5.3.2Town/ cityCambridge, MA
    B.5.3.3Post code02139
    B.5.3.4CountryUnited States
    B.5.4Telephone number1877634.8789
    B.5.5Fax number15105958183
    B.5.6E-mailmedicalinfo@vrtx.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/3/10/761
    D.3 Description of the IMP
    D.3.1Product nameVX-809
    D.3.2Product code VX-809, VRT-826809
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNlumacaftor
    D.3.9.1CAS number 936727-05-8
    D.3.9.2Current sponsor codeVX-809
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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.IMP: 2
    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/3/08/556
    D.3 Description of the IMP
    D.3.1Product nameKalydeco
    D.3.2Product code VX-770, VRT-813077
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNivacaftor
    D.3.9.1CAS number 873054-44-5
    D.3.9.2Current sponsor codeVX-770
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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.IMP: 3
    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/3/08/556
    D.3 Description of the IMP
    D.3.1Product nameKalydeco
    D.3.2Product code VX-770, VRT-813077
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNivacaftor
    D.3.9.1CAS number 873054-44-5
    D.3.9.2Current sponsor codeVX-770
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number150
    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.IMP: 4
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameLumacaftor/Ivacaftor
    D.3.2Product code VX-809/VX-770
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNLumacaftor
    D.3.9.1CAS number 936727-05-8
    D.3.9.2Current sponsor codeVX-809
    D.3.9.4EV Substance CodeSUB30152
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNIvacaftor
    D.3.9.1CAS number 873054-44-5
    D.3.9.2Current sponsor codeVX-770
    D.3.9.4EV Substance CodeSUB33103
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125
    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 placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 3
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 4
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    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 16.1
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Cohorts 1, 2 and 3 :
    - To evaluate the safety and tolerability when lumacaftor (VX-809) is administered alone or in combination with ivacaftor (VX-770)
    - To evaluate the effect of lumacaftor administered alone or in combination with ivacaftor on sweat chloride
    Cohort 4 :
    -To evaluate the safety and tolerability of lumacaftor in combination with ivacaftor
    -To evaluate the efficacy of lumacaftor in combination with ivacaftor
    E.2.2Secondary objectives of the trial
    Cohort 1 :
    - To evaluate the effect of lumacaftor administered alone or in combination with ivacaftor on pulmonary function
    - To evaluate the effect of lumacaftor administered alone on sweat chloride
    - To assess the PK of lumacaftor and its major metabolite, M28-lumacaftor, alone and in combination with ivacaftor (including the M1-ivacaftor and M6-ivacaftor)
    Cohorts 2 and 3:
    -To evaluate the effect of lumacaftor administered alone or in combination with ivacaftor on pulmonary function
    -To evaluate the effect of increasing doses of lumacaftor administered alone on sweat Chloride
    -To assess the PK of lumacaftor and M28-lumacaftor alone and in combination with ivacaftor (including M1-ivacaftor and M6-ivacaftor)
    -To evaluate the effect of lumacaftor administered alone or in combination with ivacaftor on CFQ-R score
    Cohort 4 :
    To assess the PK of lumacaftor, M28-lumacaftor, ivacaftor, M1-ivacaftor, and M6-ivacaftor
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Cohorts 1, 2 and 3
    1. Male or female subjects with confirmed diagnosis of CF
    2. Homozygous subjects must have the F508del-CFTR mutation in both alleles (Cohort 1, 2 and 3). Heterozygous subjects (Cohort 2 only) must have the F508del-CFTR mutation on 1 allele, as appropriate for the cohort. For the heterozygous subjects, the second CFTR allele must encode a mutation predicted to either result in the lack of CFTR production or in the production of a CFTR that is not responsive to ivacaftor.
    3. Age 18 years or older on the date of informed consent.
    4. FEV1 >=40% of predicted normal for age, gender, and height (Knudson standards) pre bronchodilator value at Screening
    5. Hematology and serum chemistry of blood and urine results with no clinically significant abnormalities that would interfere with the study assessments (as judged by the investigator).
    6. Able to understand and comply with protocol requirements, restrictions, and instructions, and likely to complete the study as planned.
    7. If sexually active, male subjects who are able to father a child and female subjects of childbearing potential must meet the contraception requirements
    8. Willing and able to remain on a stable medication regimen for the duration of study participation.
    9. Signed informed consent form (ICF).
    10. If the subject received prior treatment in a previous clinical study with either lumacaftor or ivacaftor, the last dose of that study drug must have been administered at least 3 months prior to Day 1 of the current study. Subjects who participated in Cohort 1 of this study are not eligible for participation in Cohort 2 or Cohort 3. Subjects who participated in Cohort 2 of this study are not eligible for participation in Cohort 3.

    Cohort 4:
    1. Signed ICF.
    2. Male and females, age 18 years or older on the date of informed consent.
    3. Male or female subjects with confirmed diagnosis of CF35 defined as:
    - A sweat chloride value not less than 60 mmol/L by quantitative pilocarpine iontophoresis OR CF-causing mutations (all as documented in the subject’s medical record) AND
    - chronic sinopulmonary disease OR gastrointestinal/nutritional abnormalities
    4. Subjects must have the F508del-CFTR mutation on 1 allele, as appropriate for the cohort.
    The second CFTR allele must encode a mutation predicted to either result in the lack of CFTR production or in the production of a CFTR that is not responsive to ivacaftor.The CFTR mutations must be confirmed before randomization.
    5. FEV1 40% to 90% of predicted normal for age, gender, and height (Hankinson standards)
    6. Stable CF disease as judged by the investigator.
    7. Hematology and clinical chemistry of blood and urine results with no clinically significant abnormalities that would interfere with the study assessments (as judged by the investigator).
    8. Able to understand and comply with protocol requirements, restrictions, and instructions, and likely to complete the study as planned.
    9. Willing and able to remain on a stable medication regimen for the duration of study participation.
    E.4Principal exclusion criteria
    Cohort 1, 2 and 3:
    - History of any illness or condition that, in the opinion of the investigator might confound the results of the study or pose an additional risk in administering study drug to the subject (e.g., cirrhosis with portal hypertension).
    - An acute illness including acute upper or lower respiratory infection, pulmonary exacerbation or changes in therapy (including antibiotics) for pulmonary disease within 14 days before receiving the first dose of study drug.
    - History of solid organ or hematological transplantation.
    - Pregnant or nursing females; females of childbearing potential who are unwilling or unable to use an acceptable method of nonhormonal contraception, as outlined in this protocol

    Cohort 4:
    1-History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
    2-Any clinically significant laboratory abnormalities at screening that would interfere with the study assessments or pose an undue risk for the subject (as judged by the investigator).
    3- Any of the following abnormal values at screening:
    - Hemoglobin <10 g/dL
    - Abnormal liver function
    - Creatinine clearance <50 mL/min/m2 using the Modification of Diet in Renal Disease calculation equation
    4. An acute illness including acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before receiving the first dose of study drug.
    5. Colonization with organisms associated with a more rapid decline in pulmonary status (e.g., Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus).
    6. A 12-lead ECG demonstrating QTcF >450 at the Screening Visit. If QTcF exceeds 450 msec for the screening ECG, the ECG should be repeated 2 more times during the Screening Period, and the average of the 3 QTcF values should be used to determine the subject's eligibility.
    7. History of solid organ or hematological transplantation.
    8. History of alcohol abuse or drug addiction in the past year, including cannabis, cocaine, and opiates, as deemed by the investigator.
    9. Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of the Screening Visit. A washout period of 5 terminal half-lives of the previous investigational study drug, or 30 days, whichever is longer, must elapse before the Screening Visit. The duration of the elapsed time may be longer if required by local regulations. Heterozygous subjects who participated in Cohort 2 and meet the eligibility criteria for Cohort 4 may participate in Cohort 4.
    10. Use of moderate to strong inducers of CYP3A AND use of strong inhibitors, including consumption of certain herbal medications (e.g., St. John's Wort) and certain fruit and fruit juices within 14 days before Day 1
    11. Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at the Screening Visit.
    12. Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements
    13. Evidence of lens opacity or cataract as determined by the ophthalmologic examination at the Screening Visit.
    14. The subject or a close relative of the subject is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study. An adult (18 years of age or older) who is a relative of a study staff member may be enrolled in the study provided that the adult otherwise qualifies to be enrolled, lives independent of and does not reside with the study staff member, and receives medical care and participates in the study at a site other than the study staff member’s site.
    E.5 End points
    E.5.1Primary end point(s)
    Cohorts 1, 2 and 3
    - Safety and tolerability assessments based on adverse events, hematology, serum chemistry, coagulation, urinalysis, standard 12-lead electrocardiograms (ECGs), and vital signs
    - Change in sweat chloride

    Cohort 4:
    - Safety and tolerability assessments based on AEs, hematology, serum chemistry, coagulation studies, urinalysis, standard 12-lead ECGs, vital signs, and pulse oximetry
    - Relative change in percent predicted FEV1
    E.5.1.1Timepoint(s) of evaluation of this end point
    - Safety will be evaluated through follow-up visit
    - Change in sweat chloride with be evaluated from Day 14 to Day 21 in Cohort 1 and Day 28 to Day 56 in Cohort 2 and 3
    - Relative change in percent predicted FEV1 from baseline at Day 56 (cohort 4)
    E.5.2Secondary end point(s)
    Cohorts 1, 2 and 3:
    - Change in percent predicted forced expiratory volume in 1 second (FEV1)
    - Change in sweat chloride
    - PK parameters of VX-809 and metabolite in presence and absence of VX-770
    - PK parameters of VX-770 and metabolite in presence and absence of VX-809
    - Cystic Fibrosis Questionnaire (CFQ-R) Score

    Cohort 4 :
    - Absolute change in percent predicted FEV1
    - Absolute change in body mass index (BMI)
    - Absolute change in CFQ-R score
    - Absolute change in body weight
    - Absolute change in sweat chloride from baseline
    - PK parameters of lumacaftor, M28-lumacaftor, ivacaftor, M1-ivacaftor, and M6-ivacaftor
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Change in percent predicted FEV1 evaluated through Day 21 in Cohort 1 and through D56 in Cohort 2, 3 and 4
    - Change in sweat chloride at increasing doses of VX-809 evaluated from baseline through D14 in Cohort 1 and from baseline through D28 in Cohort 2. Change in sweat chloride evaluated from baseline to Day 14 of lumacaftor administered alone in Cohort 3. Change in sweat chloride evaluated from baseline at D56 in cohort 4.
    - PK of lumacaftor evaluated through D21 in Cohort 1 and through D56 in Cohort 2 and 3
    - PK of ivacaftor evaluated through D21 in Cohort 1 and through D56 in Cohort 2 and 3
    - CFQ-R evaluated through D56 in Cohort 2, 3 and 4
    Cohort 4 only:
    - BMI from baseline at D56; body weight from baseline at D56
    - PK of lumacaftor, ivacaftor + metabolites through D56
    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 Yes
    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 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.8.2.4Number of treatment arms in the trial12
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA3
    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
    Australia
    New Zealand
    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
    End of the trial is defined as database lock.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months3
    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 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 293
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 40
    F.4.2.2In the whole clinical trial 293
    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)
    Following the conclusion of the patient's participation in the clinical study, the patient will return to their standard treatment for Cystic Fibrosis
    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 Decision2010-12-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-02-08
    P. End of Trial
    P.End of Trial StatusCompleted
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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