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    Summary
    EudraCT Number:2011-005085-37
    Sponsor's Protocol Code Number:CQBW251X2101
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-05-24
    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 ConcernedUK - MHRA
    A.2EudraCT number2011-005085-37
    A.3Full title of the trial
    A randomized, double blind placebo-controlled study to assess the safety, tolerability, pharmacokinetics and preliminary pharmacodynamics of single and multiple ascending doses of QBW251 in healthy subjects and multiple doses in cystic fibrosis patients.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety, tolerability, pharmacokinetics and preliminary pharmacodynamics of QBW251 in healthy subjects and cystic fibrosis patients.
    A.4.1Sponsor's protocol code numberCQBW251X2101
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorNovartis Pharma Services AG
    B.1.3.4CountrySwitzerland
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportNovartis Pharma Services AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharmaceuticals UK Ltd
    B.5.2Functional name of contact pointMedical Collaboration Centre
    B.5.3 Address:
    B.5.3.1Street AddressFrimley Business Park
    B.5.3.2Town/ cityFrimley, Camberley, Surrey
    B.5.3.3Post codeGU16 7SR
    B.5.4Telephone number00441276698370
    B.5.5Fax number00441276698449
    B.5.6E-mailmedinfo.uk@novartis.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation 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.3.2Product code QBW251
    D.3.4Pharmaceutical form Capsule, hard
    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 INNNot assigned
    D.3.9.1CAS number Not assigned
    D.3.9.2Current sponsor codeQBW251
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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.3.2Product code QBW251
    D.3.4Pharmaceutical form Capsule, hard
    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 INNNot assigned
    D.3.9.1CAS number Not assigned
    D.3.9.2Current sponsor codeQBW251
    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.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    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 placeboCapsule, hard
    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] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level PT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part 3: To assess the safety and tolerability of multiple ascending oral doses in cystic fibrosis patients and to evaluate the preliminary efficacy of multiple doses of QBW251 on change from baseline LCI in cystic fibrosis patients at Day 15.
    Part 4: Evaluate the efficacy of multiple doses of QBW251 on change from baseline in percent of predicted FEV1 in cystic fibrosis patients at Day 29.
    E.2.2Secondary objectives of the trial
    •To evaluate the pharmacokinetics of multiple doses in cystic fibrosis patients (Parts 3 and 4).
    •Part 3: To evaluate the preliminary efficacy of multiple ascending doses of QBW251 on percent predicted FEV1 in cystic fibrosis patients at Day 15.
    • Part 4: To assess the safety and tolerability of multiple doses of QBW251 in cystic fibrosis patients.
    • Part 4: to evaluate the efficacy of multiple doses of QBW251 on LCI in cystic fibrosis patients at Day 29.
    • Part 4: To evaluate the efficacy of multiple doses of QBW251 in cystic fibrosis patients as reflected in changes in the Cystic Fibrosis Questionnaire-Revised (CFQ-R PRO) at Day 14 , Part 3; at Day 28, Part 4.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Parts 3 and 4 (cystic fibrosis patients) - see protocol for full list of inclusion criteria
    1. Written informed consent must be obtained before any assessment is performed
    2. Male and female patients of 18 to 55 years of age (inclusive) with a confirmed diagnosis of cystic fibrosis as per the Cystic Fibrosis Foundation (CFF) consensus guidelines
    3. A documented CFTR genotype defined as a class III, IV, V, or VI mutation on one allele and on the other allele any other CFTR mutation with the exception of F508del/F508del due to its designation as either a class II or III mutation
    4. Patients must be willing to undergo genetic testing to confirm CFTR genotype
    5. At screening and baseline, vital signs (systolic and diastolic blood pressure and pulse rate) will be assessed in the sitting position after the subject has rested for at least three minutes and again after three minutes in the standing position. Sitting vital signs should be within the following ranges:
    •oral body temperature between 35.0-37.5°C
    •systolic blood pressure, 90-140 mm Hg
    •diastolic blood pressure, 50-90 mm Hg
    •pulse rate, 40 - 90 bpm
    Patients should be excluded if their standing vital signs (relative to sitting) show findings which, in the opinion of the Investigator, are associated with clinical manifestation of postural hypotension (i.e. absence of any other cause)
    6. Patients must have a body mass index (BMI) within the range of 15-35 kg/m2
    7. FEV1 at screening must be 50 to 90% predicted (inclusive) for Part 3 and 40 to 100% predicted (inclusive) for Part 4 by NHANES/Hankinson standards
    8. Oxygen saturation (O2) at screening must be >=90% on room air
    9. Able to perform reliable, reproducible pulmonary function test maneuvers per American Thoracic Society/European Respiratory Society (ATS/ERS) guidelines (Miller et al 2005) at screening
    10. Able to perform reliable, reproducible Lung Clearance Index test maneuvers as evaluated by over reading by EcoMedics at screening
    11. Able to communicate well with the investigator, to understand and comply with the requirements of the study. Subjects should be able to understand and sign the written informed consent
    E.4Principal exclusion criteria
    Parts 3 and 4 (cystic fibrosis patients) - see protocol for full details:
    1. Use of other investigational drugs at the time of enrollment; or within 5 half-lives or within 30 days of enrollment, or until the expected PD effect has returned to baseline
    2. History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes
    3. A history of clinically significant ECG abnormalities, or ECG abnormalities at screening
    4. History or presence of prolonged QT syndrome
    5. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin) within the past 5 years
    6. Pregnant or nursing (lactating) women
    7. Women of child-bearing potential, unless they are using contraception during the study and until study completion
    8. Sexually active males must use a condom during intercourse while taking drug and for 2 weeks after stopping study medication and should not father a child in this period
    9. Use of any herbal supplements within 4 weeks prior to initial dosing
    10. Smokers
    11. Use of prescription drugs prohibited in Section 5.4.9.2 of the protocol
    12. Donation or loss of 400 mL or more of blood within 8 weeks prior to initial dosing, or longer if required by local regulation
    13. Plasma donation (>400 mL) within 28 days prior to first dosing
    14. Hemoglobin levels below 10.0 g/dl at screening
    15. Clinical instability
    16. Recent and/or recurrent history of autonomic dysfunction
    17. Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism, or excretion of drugs, or which may jeopardize the subject in case of participation in the study
    18. History of immunodeficiency diseases, including a positive HIV test result
    19. A positive Hepatitis B surface antigen or Hepatitis C test result
    20. History of drug or alcohol abuse
    21. Unwilling to avoid direct sun exposure
    22. Any upper respiratory tract infection
    23. Any changes in concomitant medications for 14 days prior to screening throughout the end of study visit
    24. Current or historical Burkholderia cepacia respiratory tract infection and/or Mycobacterium abbesses and/or other atypical mycobacterial species infection
    25. History of lung transplant
    26. History of clinically significant hemoptysis
    27. Patients with known adrenal dysfunction (+/- adrenal replacement therapy)
    28. Patient is currently receiving (or has received within 4 weeks of baseline visit) VX-770/Ivacaftor
    29. Vulnerable subjects
    E.5 End points
    E.5.1Primary end point(s)
    Parts 3-4: safety and tolerability
    Part 3: changes in lung function (LCI) from baseline to Day 15
    Part 4: absolute change from baseline in lung function (percent of predicted FEV1) from baseline to Day 29.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part 3-4: Safety and tolerability
    Part 3: changes in lung function (LCI) from baseline to Day 15
    Part 4: absolute change from baseline in lung function (percent of predicted FEV1) from baseline to Day 29
    E.5.2Secondary end point(s)
    Part 3 and 4:
    •Pharmacokinetics multiple doses in cystic fibrosis patients (Parts 3 and 4).
    •Part 3: percent predicted FEV1 in cystic fibrosis patients at Day 15.
    •Part 4: safety and tolerability of multiple doses of QBW251 in cystic fibrosis patients.
    •Part 4: LCI in cystic fibrosis patients at Day 29.
    •Parts 3 and 4: changes in the Cystic Fibrosis Questionnaire-Revised (CFQ-R PRO) at Day 14 (Part 3) and at Day 28 (Part 4).
    E.5.2.1Timepoint(s) of evaluation of this end point
    •Pharmacokinetics multiple doses in cystic fibrosis patients (Parts 3 and 4).
    •Part 3: percent predicted FEV1 in cystic fibrosis patients at Day 15.
    •Part 4: safety and tolerability of multiple doses of QBW251 in cystic fibrosis patients.
    •Part 4: LCI in cystic fibrosis patients at Day 29.
    •Parts 3 and 4: changes in the Cystic Fibrosis Questionnaire-Revised (CFQ-R PRO) at Day 14 (Part 3) and at Day 28 (Part 4).
    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 Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    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 No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    4-parts dose-escalation study
    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 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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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
    France
    Germany
    Ireland
    Netherlands
    Romania
    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
    The study will complete when the last subject completes their Study Completion visit (EOS) and any repeat assessments associated with this visit have been documented and followed-up appropriately by the Investigator.
    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 months0
    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 months0
    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: 156
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers Yes
    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 state18
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 156
    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 EU Clinical Trial Network
    G.4.3.4Network Country Belgium
    G.4 Investigator Network to be involved in the Trial: 2
    G.4.1Name of Organisation US Clinical Trial Network
    G.4.3.4Network Country United States
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2012-07-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-05-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-11-20
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