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    Summary
    EudraCT Number:2015-004917-26
    Sponsor's Protocol Code Number:PS-G202
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-06-29
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2015-004917-26
    A.3Full title of the trial
    A Phase 2a, Two-part, Randomized, Double-blind, Placebo-controlled, Incomplete Block Crossover Study to Evaluate the Safety and Efficacy of VX-371 Solution for Inhalation With and Without Oral Ivacaftor in Subjects With Primary Ciliary Dyskinesia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate whether VX-371 is safe and makes breathing easier in patients with primary ciliary dyskinesia (study also known as CLEAN-PCD or PS-G202)
    A.3.2Name or abbreviated title of the trial where available
    PS-G202
    A.4.1Sponsor's protocol code numberPS-G202
    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 SponsorParion Sciences, 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 supportParion Sciences, Incorporated
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationParion Sciences
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address2800 Meridian Parkway
    B.5.3.2Town/ cityDurham
    B.5.3.3Post code27713
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1919313-1203
    B.5.5Fax number+1919313-1190
    B.5.6E-mailasimmons@parion.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameVX-371 / 4.2% NaCl
    D.3.4Pharmaceutical form Inhalation 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 INNVX-371
    D.3.9.2Current sponsor codeVX-371
    D.3.9.4EV Substance CodeSUB181006
    D.3.10 Strength
    D.3.10.1Concentration unit µg/ml microgram(s)/millilitre
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number30
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameVX-371 / 0.17% NaCl
    D.3.4Pharmaceutical form Inhalation 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 INNVX-371
    D.3.9.2Current sponsor codeVX-371
    D.3.9.4EV Substance CodeSUB181006
    D.3.10 Strength
    D.3.10.1Concentration unit µg/ml microgram(s)/millilitre
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number30
    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 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 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 name4.2% NaCl
    D.3.4Pharmaceutical form Inhalation 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 INN4.2% NaCl/Inhalation solution
    D.3.9.2Current sponsor code4.2% NaCl/Inhalation solution
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/V) percent weight/volume
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4.2
    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 Yes
    D.2.1.1.1Trade name Kalydeco
    D.2.1.1.2Name of the Marketing Authorisation holderVertex Pharmaceuticals (Europe) Ltd
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameIvacaftor
    D.3.2Product code 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 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 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.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 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
    Primary Ciliary Dyskinesia
    E.1.1.1Medical condition in easily understood language
    Primary Ciliary Dyskinesia
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10069713
    E.1.2Term Primary ciliary dyskinesia
    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
    Part A
    To evaluate the safety and efficacy of treatment with VX-371, administered with and without 4.2% hypertonic saline (HS) in subjects with primary ciliary dyskinesia (PCD) who are ≥12 years of age.

    Part B
    To evaluate the safety and efficacy of treatment with ivacaftor and VX-371, administered with and without 4.2% HS in subjects with PCD who are ≥12 years of age.
    E.2.2Secondary objectives of the trial
    Part A
    To evaluate the effect of VX-371, administered with and without 4.2% HS, on quality of life (QOL) in subjects with PCD who are ≥12 years of age.

    Part B
    To evaluate the effect of ivacaftor and VX-371 administered with and without 4.2% HS on QOL in subjects with PCD who are ≥12 years of age.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects who meet all of the following inclusion criteria will be eligible.
    1. Subject (or subject’s legally appointed and authorized representative) will sign and date an informed consent form (ICF) and, where appropriate, assent form.
    2. Willing and able to comply with scheduled visits, treatment plan, study restrictions laboratory tests, contraceptive guidelines, and other study procedures.
    3. Willing and able to use the nebulization device as directed by the instructions for use.
    4. The subject must have evidence supportive of a PCD diagnosis, based on the following:
    A. Subjects ≥12 to <18 years of age must meet 2 or more of the following PCD clinical criteria:
    ● Unexplained neonatal respiratory distress (at term birth) with need for respiratory support with CPAP and/or oxygen for >24 hours
    ● Any organ laterality defect confirmed by historical chest imaging – situs inversustotalis, situs ambiguous, or heterotaxy
    ● Daily, year-round wet or productive cough starting in first year of life or bronchiectasis on historical chest imaging
    ● Daily, year-round nasal congestion starting in first year of life or pansinusitis on historical sinus imaging
    B. Subjects ≥18 years of age must have bronchiectasis on historical chest imaging
    C. All subjects must ALSO have documentation of at least one of the following historical tests:
    ● For patients with no laterality defect, nNO level, measured with a chemiluminescent NO analyzer, during plateau <77 nL/min on 2 occasions, at least 2 months apart, with CF excluded by sweat chloride or genetic testing
    ● For patients with a laterality defect, nNO level, measured with a chemiluminescent NO analyzer, during plateau <77 nL/min on at least 1 occasion
    ● Diagnostic ciliary ultrastructural defect on transmission electron micrograph (TEM).
    ● 2 loss of function and/or known mutations in a single PCD-associated gene.
    Prior to randomization, all subjects must have a confirmed diagnosis of PCD (including central review, as required) based on one of the following:
    ● 2 loss of function and/or known mutations in a single PCD-associated gene identified by the central genetic testing laboratory from the specimen obtained at the Screening Visit; previous genotype results cannot be used to determine eligibility for randomization.
    ● Diagnostic ciliary ultrastructural defect on transmission electron micrograph. A previously prepared TEM specimen will be reviewed centrally; a new specimen will not be obtained.
    ● Laterality defect that includes dextrocardia plus bronchiectasis in more than 1 lobe on historical chest imaging.
    5. Subjects with ppFEV1 of ≥40 to <90 percentage points adjusted for age, sex, and height according to the Global Lung Function Initiative (GLI) predicted values at the Screening Visit, taken 4 hours or more after last dose of short-acting bronchodilators (β-agonists and/or anticholinergics)
    6. Non-smoker for the past 90 days prior to the Screening Visit and less than a 5 pack-year lifetime history of smoking, and willing to not smoke while enrolled in the study.
    7. Stable regimen of medications and chest physiotherapy for the 28 days prior to Day 1, and no anticipated need for changes during the study period (other than stopping inhaled HS).
    8. If currently using daily inhaled HS, must be able to discontinue its use for the duration of the study.
    9. If taking daily chronic or chronic cycling antibiotics, has been on a consistent regimen for at least 4 months prior to the Screening Visit. The cycling regimen of antibiotics can be either intermittent monotherapy (e.g., 28 days on/28 days off) or continuous alternating therapy (e.g., 28-day cycles of 2 alternating antibiotics).
    10. Clinically stable (as deemed by the investigator) for at least 14 days prior to the Screening Visit with no evidence of significant new or acute respiratory exacerbations, excluding symptoms of allergic (perennial or seasonal) or non-allergic rhinitis.
    11. Female subjects of childbearing potential must have a negative serum pregnancy test at the Screening Visit. Females of childbearing potential must have a negative urine pregnancy test at the Day 1, Day 57 and Day 85 visits before receiving the first dose of study drug in each Treatment Period, respectively. Subjects of childbearing potential and who are sexually active must meet the contraception requirements outlined in Section 11.6.5.1.
    E.4Principal exclusion criteria
    Subjects who meet any of the following exclusion criteria will not be eligible.
    1. Diagnosis of CF, including at least 1 of the following:
    a. Documented sweat chloride test ≥60 mM by quantitative pilocarpine iontophoresis
    b. Abnormal nasal transepithelial potential difference (NPD) test
    c. 2 CF-causing genetic mutations in the CFTR gene
    2. Subjects with only 1 mutation in the CFTR gene and a sweat chloride test ≥60 mM by quantitative pilocarpine iontophoresis.
    3. History of any organ transplantation or lung resection or chest wall surgery.
    4. Significant congenital heart defects, other than a laterality defect, at the discretion of the investigator.
    5. Diagnosis of Cri du chat syndrome (chromosome 5p deletion syndrome).
    6. Inability to withhold short-acting bronchodilator use for 4 hours prior to clinic visit and long-acting bronchodilator use the night before the first and last clinic visit of each treatment period.
    7. History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug(s) to the subject. This may include, but is not limited to history of clinically significant and uncontrolled adrenal, neurologic, gastrointestinal, renal, hepatic, cardiovascular (including hyper/hypotension and tachy/bradycardia), psychological, pulmonary (other than PCD), metabolic, endocrine, or hematological/coagulation disorder or disease, or scoliosis of such severity that it impacts pulmonary function or any other major disorder or disease, in the opinion of the investigator.
    8. Use of diuretics (including amiloride) or renin-angiotensin antihypertensive drugs (e.g., spironolactone, angiotensin converting enzyme [ACE] and/or neural endopeptidase (NEP)-inhibitors, or angiotensin receptor blockers [ARBs]) or trimethoprim or drospirenone in the 28 days before Day 1 or anticipate need for these medications during the study.
    9. Had symptoms of acute upper or lower respiratory tract infection or had an acute pulmonary exacerbation requiring treatment or was treated with systemic antibiotics for ear or sinus disease within 28 days before Day 1 (topical otic antibiotics allowed).
    10. History of significant intolerance to inhaled HS as determined by the investigator.
    11. History of drug or alcohol abuse, in the opinion of the investigator.
    12. Known hypersensitivity to any of the study drugs or amiloride.
    13. Used ivacaftor within 28 days prior to Day 1 or anticipate need for ivacaftor during the study.
    14. Pregnant and/or nursing females.
    15. Any clinically significant laboratory abnormalities at the Screening Visit as judged by the investigator, or any of the following:
    a. Plasma or serum potassium > upper limit of normal (ULN)
    b. Abnormal renal function, defined as creatinine clearance rate <50 mL/min using the Bedside Schwartz equation (for subjects 12 to 17 years of age) or <50 mL/min using the Cockcroft-Gault equation (for subjects ≥18 years of age)
    c. Abnormal liver function, defined as ≥3 x ULN for alanine aminotransferase (ALT), aspartate aminotransferase (AST), or 2 x ULN for total bilirubin, unless accounted for by Gilbert’s syndrome (benign indirect hyperbilirubinemia)
    d. Hemoglobin concentration <10.0 g/dL
    16. Unwilling or unable to follow the contraception guidelines as outlined in Section 11.6.5.1.
    17. History of at least 2 sputum or throat swab cultures yielding B. cepacia complex or M. abscessus or M. avium within the previous 2 years.
    18. Has had surgery within 3 months of Day 1 that required general anesthesia and hospitalization.
    19. Has previously participated in an investigational study involving administration of any investigational compound or use of an investigational device within 28 days prior to the Screening Visit (Note: Participation in a past or concurrent observational study is acceptable).
    20. Has any surgical or medical condition which in the judgment of the investigator might interfere with the absorption, distribution, metabolism, or excretion of the study drug or with safety evaluations.
    21. Has any other condition or circumstances that, in the opinion of the investigator, should disqualify this subject for this study.
    22. The subject or a close relative of the subject is the investigator or a sub-investigator, research assistant, pharmacist, study coordinator, or other staff directly involved in the conduct of the study.
    Additional Exclusion Criteria for Part B:
    In addition to the exclusion criteria above, subjects who participate in Part B and meet any of the following exclusion criteria will not be eligible to continue into Part B.
    1. Unable to swallow tablets.
    2. Concomitant use of strong or moderate inhibitors or inducers of cytochrome P450 (CYP) 3A, including consumption of certain herbal medications (e.g., St. John's Wort), and grapefruit/grapefruit juice.
    3. Known hypersensitivity to ivacaftor.
    E.5 End points
    E.5.1Primary end point(s)
    Part A
    -Results of safety and tolerability assessments of adverse events (AEs), clinical laboratory values (urine, serum and plasma chemistry, and hematology), 12-lead electrocardiograms (ECGs), spirometry, vital signs, and pulse oximetry.
    -Absolute change in percent predicted FEV1, from study baseline after 28 days of treatment in Part A.

    Part B
    - Results of safety and tolerability assessments of adverse events AEs, clinical laboratory values (urine, serum and plasma chemistry, and hematology), 12-lead ECGs, spirometry, vital signs, and pulse oximetry.
    - Absolute change in ppFEV1 from study baseline and Part B baseline, after 28 days of treatment in Part B.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part A
    For Treatment Period 1 it will be evaluated at day 28 and for Treatment Period 2 it will be evaluated at day 85 --> both compared to study baseline.

    Part B
    For Treatment Period 3 it will be evaluated at Day 113 --> compared to study baseline and Part B baseline.
    E.5.2Secondary end point(s)
    Part A
    - Change in QOL score as measured by the PCD Quality of Life Questionnaire (QOL-PCD) and the St. George’s Respiratory Questionnaire (SGRQ) after 28 days of treatment in Part A.

    Part B
    - Change in QOL score as measured by the QOL-PCD and SGRQ, from study baseline and Part B baseline, after 28 days of treatment in Part B
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part A
    For Treatment Period 1 it will be evaluated at day 28 and for Treatment Period 2 it will be evaluated at day 85 --> both compared to study baseline.

    Part B
    For Treatment Period 3 it will be evaluated at Day 113 --> compared to study baseline and Part B baseline.
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    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 No
    E.8.1.6Cross over Yes
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    In Part B, administration of ivacaftor will be open-label.
    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 trial4
    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 EEA11
    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
    Canada
    Denmark
    Germany
    Italy
    Netherlands
    Poland
    United Kingdom
    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.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months1
    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 Yes
    F.1.1Number of subjects for this age range: 40
    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.1.1.6.1Number of subjects for this age range: 40
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 100
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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
    For 12-17 year olds and there will parental consent.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 75
    F.4.2.2In the whole clinical trial 150
    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)
    There are no plans for treatment or care following subject completion of participation in the trial.
    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 Decision2016-08-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-09-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-11-20
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