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    Summary
    EudraCT Number:2019-002840-26
    Sponsor's Protocol Code Number:CQBW251C12201
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2020-10-01
    Trial 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 ConcernedGermany - BfArM
    A.2EudraCT number2019-002840-26
    A.3Full title of the trial
    A randomized, subject- and investigator-blinded, placebo-controlled, parallel group study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of QBW251 in patients with bronchiectasis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of safety, tolerability, pharmacokinetics and pharmacodynamics of QBW251 in subjects with bronchiectasis
    A.4.1Sponsor's protocol code numberCQBW251C12201
    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 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 AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma GmbH
    B.5.2Functional name of contact pointMedizinischer Infoservice (MCC)
    B.5.3 Address:
    B.5.3.1Street AddressRoonstrasse 25
    B.5.3.2Town/ cityNuremberg
    B.5.3.3Post code90429
    B.5.3.4CountryGermany
    B.5.4Telephone number+49 911 273-12100
    B.5.5Fax number+49 911 27312160
    B.5.6E-mailinfoservice.novartis@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 INNIcenticaftor
    D.3.9.2Current sponsor codeQBW251
    D.3.9.3Other descriptive nameCFTR potentiator
    D.3.9.4EV Substance CodeSUB73414
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number300
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Bronchiectasis
    E.1.1.1Medical condition in easily understood language
    Bronchiectasis
    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 23.0
    E.1.2Level LLT
    E.1.2Classification code 10083611
    E.1.2Term Non-cystic fibrosis bronchiectasis
    E.1.2System Organ Class 100000004855
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To determine the efficacy of QBW251 compared to placebo with respect to change from baseline in bacterial load of colony forming units (CFU/mL, 1CFU/mL= 1 CFU/g) of potentially pathogenic microorganisms in spontaneous sputum at week 12.
    E.2.2Secondary objectives of the trial
    -To assess the change of QBW251 compared to placebo on sputum bacterial clearance
    -To assess the change on patient reported outcomes on bronchiectasis symptom assessment
    -To assess the change of fibrinogen plasma concentration
    -To assess the change in rescue medication use
    -To assess the change on lung function
    -To assess the change in airway structure and function
    -To assess the pharmacokinetics of QBW251 in patients with bronchiectasis
    -To assess the safety and tolerability of QBW251 in patients with bronchiectasis
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Written informed consent must be obtained before any assessment is performed.
    -Male or female patients aged ≥18 years at screening.
    -Proven diagnosis of bronchiectasis by chest CT at screening as determined by the investigator. The screening HRCT can be used to
    confirm the diagnosis if no historical chest CT is available.
    -Evidence of sputum bacterial load of ≥10^6 CFU/mL with at least one
    potentially pathogenic microorganism at screening (H. Influenzae, M
    catarrhalis, S aureus, S pneumoniae, Enterobacteriaceae, P aeruginosa, Stenotrophomonous maltophilia, or any potential pathogenic nonfermenting Gram negative bacteria measured by dilution/outgrowth.)
    NOTE: H. parainfluenzae is not considered pathogenic in this study
    and therefore growth of this organism alone does not satisfy this criterion.
    -Documented history of at least one bronchiectasis exacerbation
    between January 2019 and study screening.
    -Patients with bronchial hypersecretion, defined as productive cough
    that occurs on most days (defined as >50% days) for at least three
    consecutive months within 12 months prior to screening, as assessed by documentation of patient recollection (anamnesis) or documented in patients' record.
    -Patients are allowed to stay on fixed or free combinations of
    LABA/LAMA or LABA/ICS or LABA/LAMA/ICS as maintenance therapy if they are treated with them at a stable doses for the last 3 months prior to screening. Patients are also allowed to stay on macrolides as
    maintenance therapy if they are treated with them at a stable dose 3
    months before screening. Patients will be allowed to use mucolytics or hyperosmolar agents if they were treated with them before study start.
    - If prescribed, patients are included in the study with unchanged chest physiotherapy for at least 4 weeks prior to screening.
    -Clinically stable pulmonary status in the opinion of the investigator and unlikely to require any change in the standard regimen of care during the course of the study.
    -Able to perform reliable, reproducible pulmonary function test
    maneuvers per American Thoracic Society/European Respiratory Society (ATS/ERS) guidelines at screening. At screening, patients who have failed to meet ATS/ERS requirements for acceptability and
    reproducibility for spirometry will be allowed one additional repeat
    testing session during the screening period.
    E.4Principal exclusion criteria
    -Use of other investigational drugs at the time of enrollment, or within 5 half-lives of enrollment, or within 30 days, whichever is longer; or longer if required by local regulations. Current or planned participation in another clinical trial during this study
    - History of hypersensitivity to the study drugs or to drugs of similar chemical classes or excipients
    -Patients with a history of long-QT syndrome or the QTcF interval at Screening or baseline is prolonged (QTcF >450 ms in males, >460 ms in females)
    -Patients who have a clinically significant ECG abnormality before randomization, as determined by investigator. Note: Clinically significant abnormalities may include but are not limited to the following: left bundle branch block, Wolff-Parkinson-White syndrome, clinically significant arrhythmias (e.g. atrial fibrillation, ventricular tachycardia)
    -Patients with a history or current treatment for hepatic disease including but not limited to acute or chronic hepatitis, cirrhosis or hepatic failure. A history of resolved Hepatitis A is not exclusionary. Patients with prothrombin time international normalized ratio(PT/INR) of more than 1.5xULN at screening. Patients excluded for the PT/INR of more than 1.5xULN can be re-screened when the values have returned to normal
    -History of lung transplant or malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin. Patients with segmentectomy for other reasons than cancer are allowed to be included in the study. Patients with a history of cancer and 5 years or more disease-free survival time may be included in the study by agreement with Novartis Medical Monitor on a case-by-case basis
    -Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory blood test
    -Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using acceptable effective methods of contraception during study participation
    -Clinically significant laboratory values abnormalities (including G-GT, AST, ALT, total bilirubin or creatinine) in the opinion of the investigator at screening
    -Patients requiring long-term oxygen therapy for chronic hypoxemia. This is typically patients requiring oxygen therapy >12 h per day delivered by home oxygen cylinder or concentrator. Note: Nocturnal oxygen therapy for transient oxygen desaturations during sleep is allowed
    -Patients with bronchiectasis who have had a pulmonary exacerbation with a deterioration in three or more of key symptoms for at least 48 h and a clinicians determines that a change bronchiectasis treatment is required within 4 weeks prior to screening
    -Hemoptysis, requiring medical intervention at any time within 4 weeks prior to screening
    -Bronchiectasis predominantly characterized by isolated cavitary lung lesions
    -Patients with bronchiectasis requiring therapy that may interfere with the assessment of QBW251 efficiency or that are unlikely to respond to QBW251
    -Current or ex-smokers with severe emphysema as determined by the investigator
    -Patients with another concomitant pulmonary disease according to the definition of the International ERS/ATS guidelines, including but not limited to IPF, sarcoidosis or other granulomatous or infectious process. Concomitant COPD and asthma with characteristics of airway hyperresponsiveness as well as COPD-Asthma overlap syndrome are allowed as long as it is not the main, primary diagnosis as determined by investigator
    -Patients currently receiving treatment for NTM pulmonary disease. Patients with a diagnosis of PCD may be allowed to participate with approval of Sponsor
    -Patients with a known history of non-compliance to medication or who are unable or unwilling to complete an electronic patient diary or patient reported outcome questionnaire
    -Recent (within three years of screening) or recurrent history of clinically significant autonomic dysfunction (e.g., recurrent episodes of fainting, palpitations, etc...) as determined by investigator
    -Patients with a major vascular surgery in the 6 months prior to the screening visit
    -Patients who have clinically significant renal, cardiovascular (such as but not limited to unstable ischemic heart disease, NYHA Class III/IV left ventricular failure, myocardial infarction), neurological, endocrine, immunological, psychiatric, gastrointestinal, or hematological abnormalities, which could interfere with the assessment of the efficacy and safety of the study treatment, or patients with uncontrolled Type I diabetes or uncontrolled Type II diabetes
    -Known or suspected history of ongoing, chronic or recurrent infectious disease of HIV, Hepatitis B/C
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in bacterial load of colony forming units ( CFU/mL, 1 CFU/mL = 1 CFU/g) of potentially pathogenic microorganisms in spontaneous sputum with QBW251 compared to placebo at week 12.
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 weeks of treatment
    E.5.2Secondary end point(s)
    -Proportion of subjects with absence of any CFU of potentially pathogenic bacteria in sputum culture after 12 weeks of treatment.
    -Changes from baseline in Quality of Life Questionnaire for Bronchiectasis (QOL-B) (Respiratory symptoms domain) after 12 weeks of treatment.
    -Change from baseline in fibrinogen plasma concentration after 12 weeks of treatment.
    -Change from baseline in rescue medication use (salbutamol/albuterol) after 12 weeks of treatment.
    -Changes from baseline in pre- and postbronchodilator FEV1, FVC after 12 weeks treatment, measured by spirometry.
    -Change from baseline in airway wall and lumen parameters along with extent of global and regional air trapping after 12 weeks of treatment, as measured by HRCT.
    -Assessment of drug exposure (Cmax, AUC) on Days 1 and 28; pre- and post-dose concentration (Ctrough and Cmax) on Days 56 and 84 (for a subset of patients at selected sites). Pre- and post- dose concentration (Ctrough and around Cmax) on Days 1, 28, 56 and 84 (for all other patients).
    -All safety endpoints (including adverse events, vital signs, ECG, and safety laboratory changes) during the study.
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 weeks of treatment
    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 Yes
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    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) 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 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 EEA15
    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
    China
    Spain
    Germany
    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
    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 months7
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months8
    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: 36
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 36
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 72
    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)
    None
    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 Decision2021-01-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-01-15
    P. End of Trial
    P.End of Trial StatusCompleted
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