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Dose-finding Study to Assess the Efficacy, Safety and Tolerability of Tobramycin Inhalation Powder in Patients With Non-Cystic Fibrosis Bronchiectasis and Pulmonary P. Aeruginosa Infection (iBEST-1)

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ClinicalTrials.gov Identifier: NCT02712983
Recruitment Status : Completed
First Posted : March 18, 2016
Results First Posted : August 14, 2020
Last Update Posted : August 25, 2020
Sponsor:
Collaborators:
Queen's University Belfast, UK
University Hospital Antwerp, BE
University of Milan, IT
Fundacion Clinic per a la Recerca Biomedica
Erasmus Medical Center
Papworth Hospital Cambridge, UK
Royal Brompton Hospital Trust, UK
University of Dundee
University of Edinburgh
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Tracking Information
First Submitted Date  ICMJE February 5, 2016
First Posted Date  ICMJE March 18, 2016
Results First Submitted Date  ICMJE March 17, 2020
Results First Posted Date  ICMJE August 14, 2020
Last Update Posted Date August 25, 2020
Actual Study Start Date  ICMJE February 8, 2017
Actual Primary Completion Date March 20, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 13, 2020)
Change From Baseline to Day 29 in Pseudomonas Aeruginosa (P. Aeruginosa) Density in Sputum (log10 CFUs) [ Time Frame: Baseline (Visit 101/Day 1), Visit 102 (Day 8), Visit 103 (Day 29) ]
Microbiological data was collected to understand the direct impact of the drug on the pathogens. Sputum samples were cultured for the presence of three Pseudomonas aeruginosa (P. aeruginosa) biotypes measured were mucoid, dry and small colony variant. Change was determined using the formula = (Post-baseline value - baseline value). If no P. aeruginosa was isolated for a visit, log10 colony forming units (CFU) was imputed with log10 (19) for all biotypes. Only values for all morphotypes presented.
Original Primary Outcome Measures  ICMJE
 (submitted: March 14, 2016)
P. aeruginosa density in sputum [ Time Frame: Baseline to Day 29 ]
Change in P. aeruginosa bacterial load in sputum as assessed by the change in log10 colony forming units (CFUs) from baseline.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 13, 2020)
  • Change From Baseline to Each Post-baseline Visit in Pseudomonas Aeruginosa (P. Aeruginosa) Density in Sputum (log10 CFUs) [ Time Frame: Baseline (Visit 101/Day 1), Visit 104 (Day 57), Visit 105 (Day 85), Visit 106 (Day 113), End of Treatment (EOT), Visit 201 (Day 141), Visit 202 (Day 169) ]
    Microbiological data was collected to understand the direct impact of the drug on the pathogens. Sputum samples were cultured for the presence of three Pseudomonas aeruginosa (P. aeruginosa) biotypes measured were mucoid, dry and small colony variant. Change was determined using the formula = (Post-baseline value - baseline value). If no P. aeruginosa was isolated for a visit, log10 colony forming units (CFU) was imputed with log10 (19) for all biotypes. Only values for all morphotypes are presented.
  • Time to First Onset of Pulmonary Exacerbation by Exacerbation Category [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    The time to first onset of pulmonary exacerbation compared to placebo was analyzed. Participants with pulmonary exacerbation were categorized as: a) Overall, b) Category 1 (Oral): treated with oral antibiotics only and c) Category 2 (Parenteral): treated with parenteral Antibiotics and/or requiring hospitalization. Participants were censored at the time of completion of study or early discontinuation if they did not have a pulmonary exacerbation during the study period.
  • Duration of Pulmonary Exacerbation by Exacerbation Category [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    The duration of pulmonary exacerbation compared to placebo was analyzed. Participants with pulmonary exacerbation were categorized as: a) Overall, b) Category 1 (Oral): treated with oral antibiotics only and c) Category 2 (Parenteral): treated with parenteral Antibiotics and/or requiring hospitalization.
  • Exposure Adjusted Rate of Pulmonary Exacerbations (PE) Over the Entire Study Period [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    The exposure adjusted rate of pulmonary exacerbation compared to placebo was analyzed. Participants with pulmonary exacerbation were categorized as: a) Overall, b) Category 1 (Oral): treated with oral antibiotics only and c) Category 2 (Parenteral): treated with parenteral Antibiotics and/or requiring hospitalization. The Exposure adjusted rate = (Number of pulmonary exacerbations reported during the study period) / (sum of study duration in days for all participants/ 365.25). Only descriptive analysis performed.
  • Percentage of Participants With at Least One Pulmonary Exacerbation by Exacerbation Category [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    Pulmonary exacerbations are defined as events requiring antibiotic therapy AND for which at least 3 of the following 6 symptoms, signs, or findings were present outside of normal variation: 1. Increased sputum volume, or change in viscosity/consistency or purulence for more than 24 hours; 2. Increased shortness of breath at rest or on exercise for more than 24 hours; 3. Increased cough for more than 24 hours; 4. Fever of ≥38° Celsius within the last 24 hours; 5. Increased malaise/fatigue/lethargy for more than 24 hours; 6. A reduction in forced expiratory volume in the first second of expiration (FEV1) or forced vital capacity (FVC) of least 10% from screening. Participants were categorized as: a) Overall, b) Category 1: treated with oral antibiotics only and c) Category 2: treated with parenteral Antibiotics and/or requiring hospitalization. Only descriptive analysis performed.
  • Percentage of Participants Who Permanently Discontinued Study Drug Due to Pulmonary Exacerbation [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    The percentage of participants who permanently discontinued study drug due to pulmonary exacerbation compared to placebo was analyzed.
  • Time to Permanent Study Drug Discontinuation Due to Pulmonary Exacerbation [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    The time to permanent study drug discontinuation due to Pulmonary exacerbation. Participants were censored at the time of last contact if they did not permanently discontinue study drug due to pulmonary exacerbation requiring during the study period. Only descriptive analysis performed.
  • Time to First Use (Overall, Oral, and Parenteral) of Anti-pseudomonal Antibiotics Usage [ Time Frame: From Baseline (Visit 101/Day 1) up to approximately Day 173 ]
    The time to first use of anti-pseudomonal antibiotics administered compared to placebo was analyzed. Participants were censored at the time of last contact if they did not have anti-pseudomonal antibiotics over the entire study period.
  • Percentage of Participants Requiring Anti-pseudomonal Antibiotics [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    The percentage of participants requiring anti-pseudomonal antibiotics compared to placebo was analyzed. Only descriptive analysis performed.
  • Duration of Anti-pseudomonal Antibiotics Usage [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    The total number of days of new anti-pseudomonal antibiotic use compared to placebo was analyzed. Only descriptive analysis
  • Percentage of Participants Requiring Hospitalization Due to Serious Respiratory-related Adverse Events [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    The percentage of participants requiring hospitalization due to serious respiratory-related adverse events (other than those regularly scheduled hospitalization that were planned prior to study start) was analyzed to define severity of pulmonary exacerbations compared to placebo. Only descriptive analysis performed.
  • Duration of Hospitalization Due to Serious Respiratory-related Adverse Events [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    The duration of hospitalization due to serious respiratory-related adverse events (other than those regularly scheduled hospitalization that were planned prior to study start) was analyzed to define severity of pulmonary exacerbations compared to placebo. Only descriptive analysis performed.
  • Number of Hospitalization Due to Serious Respiratory-related Adverse Events [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    The number of hospitalization due to serious respiratory-related AEs was analyzed to define severity of pulmonary exacerbations compared to placebo. Respiratory related adverse events were identified using the AEs captured under system organ class 'Respiratory, thoracic and mediastinal disorders' and 'Infections and infestations'.
  • Time to First Hospitalization Due to Serious Respiratory-related Adverse Events [ Time Frame: Baseline (Visit 101/Day 1) to Visit 202 (Day 169) ]
    Time to first hospitalization due to serious respiratory-related AEs was analyzed to define severity of pulmonary exacerbations compared to placebo. Participants were censored at the time of last contact if they did not have a hospitalization due to serious respiratory-related adverse events over the entire study period.
  • Serum Tobramycin Concentration [ Time Frame: Baseline (Visit 101/Day 1), Visit 102 (Day 8) and Visits 103 (Day 29): 0-1 hours and 1-2 hours post-dose. ]
    The serum pharmacokinetic (PK) properties of tobramycin were assessed by evaluating tobramycin concentrations in serum collected from the non-cystic fibrosis bronchiectasis population post administration of o.d. or b.i.d. doses of TIP. Serum specimens for PK tobramycin concentration were assessed at Visit 101 (Day 1/start of treatment) 0 to 1 and 1 to 2 hours post-dose and Visit 102 (Day 8) 0 to 1 and 1 to 2 hours post-dose. Prior to protocol amendment #2, PK samples were assessed on Visits 103 (Day 29) rather than Visit 102. Only descriptive analysis performed.
  • Sputum Tobramycin Concentration [ Time Frame: Baseline (Visit 101/Day 1): 0-1 hours and 1-2 hours post-dose; Visit 102 (Day 8):0-2 hours and 5-6 hours post-dose; Visits 103 (Day 29): 5 to 6 hours post-dose, Visit 104 (Day 57) and Visit 105 (Day 85): 3-4 hours post-dose. ]
    The sputum pharmacokinetic (PK) properties of tobramycin were assessed by evaluating tobramycin concentrations in sputum collected from the non-cystic fibrosis bronchiectasis population post administration of o.d. or b.i.d. doses of TIP. Only descriptive analysis performed.
  • Change From Baseline in Quality of Life Questionnaire for Bronchiectasis (QOL-B)-Physical Functioning [ Time Frame: Baseline, Visit 102 (Day 8), Visit 103 (Day 29), End of Treatment (Day 113) and Visit 202 (Day 169) ]
    The QoL-B consists of 37 items across 8 domains: Physical Functioning (PF), Role Functioning (RF), Vitality, Emotional Function (EF), Social Functioning (SF), Treatment Burden (TB), Health Perception (HP) and Respiratory Symptoms (RS). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardized on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and HRQoL. A total score is not calculated since functioning can vary greatly from one domain to another. Only descriptive analysis performed.
  • Change From Baseline in Quality of Life Questionnaire for Bronchiectasis (QOL-B)-Role Functioning [ Time Frame: Baseline, Visit 102 (Day 8), Visit 103 (Day 29), End of Treatment (Day 113) and Visit 202 (Day 169) ]
    The QoL-B consists of 37 items across 8 domains: Physical Functioning (PF), Role Functioning (RF), Vitality, Emotional Function (EF), Social Functioning (SF), Treatment Burden (TB), Health Perception (HP) and Respiratory Symptoms (RS). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardized on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and HRQoL. A total score is not calculated since functioning can vary greatly from one domain to another. Only descriptive analysis performed.
  • Change From Baseline in Quality of Life Questionnaire for Bronchiectasis (QOL-B)-Vitality [ Time Frame: Baseline, Visit 102 (Day 8), Visit 103 (Day 29), End of Treatment (Day 113) and Visit 202 (Day 169) ]
    The QoL-B consists of 37 items across 8 domains: Physical Functioning (PF), Role Functioning (RF), Vitality, Emotional Function (EF), Social Functioning (SF), Treatment Burden (TB), Health Perception (HP) and Respiratory Symptoms (RS). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardized on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and HRQoL. A total score is not calculated since functioning can vary greatly from one domain to another. Only descriptive analysis performed.
  • Change From Baseline in Quality of Life Questionnaire for Bronchiectasis (QOL-B)-Emotional Functioning [ Time Frame: Baseline, Visit 102 (Day 8), Visit 103 (Day 29), End of Treatment (Day 113) and Visit 202 (Day 169) ]
    The QoL-B consists of 37 items across 8 domains: Physical Functioning (PF), Role Functioning (RF), Vitality, Emotional Function (EF), Social Functioning (SF), Treatment Burden (TB), Health Perception (HP) and Respiratory Symptoms (RS). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardized on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and HRQoL. A total score is not calculated since functioning can vary greatly from one domain to another. Only descriptive analysis performed.
  • Change From Baseline in Quality of Life Questionnaire for Bronchiectasis (QOL-B)-Social Functioning [ Time Frame: Baseline, Visit 102 (Day 8), Visit 103 (Day 29), End of Treatment (Day 113) and Visit 202 (Day 169) ]
    The QoL-B consists of 37 items across 8 domains: Physical Functioning (PF), Role Functioning (RF), Vitality, Emotional Function (EF), Social Functioning (SF), Treatment Burden (TB), Health Perception (HP) and Respiratory Symptoms (RS). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardized on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and HRQoL. A total score is not calculated since functioning can vary greatly from one domain to another. Only descriptive analysis performed.
  • Change From Baseline in Quality of Life Questionnaire for Bronchiectasis (QOL-B)-Treatment Burden [ Time Frame: Baseline, Visit 102 (Day 8), Visit 103 (Day 29), End of Treatment (Day 113) and Visit 202 (Day 169) ]
    The QoL-B consists of 37 items across 8 domains: Physical Functioning (PF), Role Functioning (RF), Vitality, Emotional Function (EF), Social Functioning (SF), Treatment Burden (TB), Health Perception (HP) and Respiratory Symptoms (RS). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardized on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and HRQoL. A total score is not calculated since functioning can vary greatly from one domain to another. Only descriptive analysis performed.
  • Change From Baseline in Quality of Life Questionnaire for Bronchiectasis (QOL-B)-Health Perceptions [ Time Frame: Baseline, Visit 102 (Day 8), Visit 103 (Day 29), End of Treatment (Day 113) and Visit 202 (Day 169) ]
    The QoL-B consists of 37 items across 8 domains: Physical Functioning (PF), Role Functioning (RF), Vitality, Emotional Function (EF), Social Functioning (SF), Treatment Burden (TB), Health Perception (HP) and Respiratory Symptoms (RS). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardized on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and HRQoL. A total score is not calculated since functioning can vary greatly from one domain to another. Only descriptive analysis performed.
  • Change From Baseline in Quality of Life Questionnaire for Bronchiectasis (QOL-B)-Respiratory Symptoms [ Time Frame: Baseline, Visit 102 (Day 8), Visit 103 (Day 29), End of Treatment (Day 113) and Visit 202 (Day 169) ]
    The QoL-B consists of 37 items across 8 domains: Physical Functioning (PF), Role Functioning (RF), Vitality, Emotional Function (EF), Social Functioning (SF), Treatment Burden (TB), Health Perception (HP) and Respiratory Symptoms (RS). Each of the 37 items is scored from 1 to 4, and each of the 8 scale scores is standardized on a 0-100 point scale, with higher scores representing fewer symptoms or better functioning and HRQoL. A total score is not calculated since functioning can vary greatly from one domain to another. Only descriptive analysis performed.
Original Secondary Outcome Measures  ICMJE
 (submitted: March 14, 2016)
  • Frequency of pulmonary exacerbations [ Time Frame: 112 days of treatment and 56 days of follow-up ]
    Defined as frequency of pulmonary exacerbations compared to placebo.
  • Time to first use of antipseudomonal antibiotics [ Time Frame: 112 days of treatment and 56 days of follow-up ]
    Measured by the time to first use of anti-pseudomonal antibiotics compared to placebo
  • Serum tobramycin concentration [ Time Frame: Day 29 ]
  • Sputum tobramycin concentration [ Time Frame: Day 85 ]
  • P. aeruginosa density in sputum over the entire study duration [ Time Frame: 112 days of treatment and 56 days of follow-up ]
    Change in P. aeruginosa bacterial load in sputum from baseline as assessed by the change in colony forming units (CFUs) of P. aeruginosa.
  • Respiratory Symptom Scale Quality of Life Questionnaire for Bronchiectasis (QOL-B) [ Time Frame: 112 days of treatment and 56 days of follow-up ]
    Change from baseline on the Respiratory Symptom Scale QOL-B. Respiratory Symptoms is 1 out of 8 domains of the QOL-B instrument and is graded on a 4-point Likert scale.
  • Rate of pulmonary exacerbations [ Time Frame: 112 days of treatment and 56 days of follow-up ]
    Defined as rate of pulmonary exacerbations compared to placebo.
  • Severity of pulmonary exacerbations [ Time Frame: 112 days of treatment and 56 days of follow-up ]
    Defined as severity of pulmonary exacerbations compared to placebo.
  • Time to onset of pulmonary exacerbations [ Time Frame: 112 days of treatment and 56 days of follow-up ]
    Defined as time to onset of pulmonary exacerbations compared to placebo.
  • Proportion of patients requiring anti-pseudomonal antibiotics [ Time Frame: 112 days of treatment and 56 days of follow-up ]
    Measured by proportion of patients requiring anti-pseudomonal antibiotics compared to placebo
  • Duration of anti-pseudomonal antibiotic treatment [ Time Frame: 112 days of treatment and 56 days of follow-up ]
    Measured by duration of use of anti-pseudomonal antibiotics compared to placebo
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Dose-finding Study to Assess the Efficacy, Safety and Tolerability of Tobramycin Inhalation Powder in Patients With Non-Cystic Fibrosis Bronchiectasis and Pulmonary P. Aeruginosa Infection
Official Title  ICMJE A Randomized, Blinded, Parallel Group, Multi-center Dose-finding Study, to Assess the Efficacy, Safety and Tolerability of Different Doses of Tobramycin Inhalation Powder in Patients With Non-Cystic Fibrosis Bronchiectasis and Pulmonary P. Aeruginosa Infection
Brief Summary The purpose of this study was to support the selection of a safe and tolerable tobramycin inhalation powder (TIP) dose, and regimen that exhibits effective bacterial reduction of P. aeruginosa in non-cystic fibrosis bronchiectasis (BE) patients with P. aeruginosa colonization.
Detailed Description

This was a blinded, randomized, dose and regimen finding trial utilizing a three treatment cohort design where active TIP and TIP/Placebo cyclical (3 capsules o.d [Cohort A], 5 capsules o.d. [Cohort B] or 4 capsules b.i.d. [Cohort C]) versus Placebo were administered for a total of 112 days.

Novartis decided to close the recruitment of new subjects into this study earlier than scheduled. Subjects who had a signed informed consent form and entered screening by 10-Sep-2018 still participated in the study. The latest possible randomization was on 08-Oct-2018. All subjects enrolled in the study (107 enrolled subjects out of 180 planned) continued as planned through to their last scheduled visit. The early recruitment halt of the study was not due to safety or lack of efficacy.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Non-cystic Fibrosis Bronchiectasis
Intervention  ICMJE
  • Drug: TIP
    TIP dose regimen
  • Drug: TIP and placebo
    TIP and inhaled placebo dose regimen
  • Drug: Placebo
    Inhaled placebo dose regimen
Study Arms  ICMJE
  • Experimental: Cohort A (3 capsules o.d.): TIP
    Cohort A (3 capsules o.d.): Tobramycin inhalation powder (TIP)
    Intervention: Drug: TIP
  • Experimental: Cohort A (3 capsules o.d.): TIP/PBO
    Cohort A (3 capsules o.d.): Tobramycin inhalation powder (TIP) and inhaled placebo (PBO) cyclical
    Intervention: Drug: TIP and placebo
  • Placebo Comparator: Cohort A (3 capsules o.d.): PBO
    Cohort A (3 capsules o.d.): Inhaled placebo (PBO)
    Intervention: Drug: Placebo
  • Experimental: Cohort B (5 capsules o.d.): TIP
    Cohort B (5 capsules o.d.): Tobramycin inhalation powder (TIP)
    Intervention: Drug: TIP
  • Experimental: Cohort B (5 capsules o.d.): TIP/PBO
    Cohort B (5 capsules o.d.): Tobramycin inhalation powder (TIP) and inhaled placebo (PBO) cyclical
    Intervention: Drug: TIP and placebo
  • Placebo Comparator: Cohort B (5 capsules o.d.): PBO
    Cohort B (5 capsules o.d.): inhaled placebo (PBO)
    Intervention: Drug: Placebo
  • Experimental: Cohort C (4 capsules b.i.d.): TIP
    Cohort C (4 capsules b.i.d.): Tobramycin inhalation powder (TIP)
    Intervention: Drug: TIP
  • Experimental: Cohort C (4 capsules b.i.d.): TIP/PBO
    Cohort C (4 capsules b.i.d.): Tobramycin inhalation powder (TIP) and inhaled placebo (PBO) cyclical
    Intervention: Drug: TIP and placebo
  • Placebo Comparator: Cohort C (4 capsules b.i.d.): PBO
    Cohort C (4 capsules b.i.d.): inhaled placebo (PBO)
    Intervention: Drug: Placebo
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: May 17, 2019)
107
Original Estimated Enrollment  ICMJE
 (submitted: March 14, 2016)
180
Actual Study Completion Date  ICMJE March 20, 2019
Actual Primary Completion Date March 20, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Written informed consent must be obtained before any assessment is performed.
  • Male and female patients of ≥ 18 years of age at screening (Visit 1).
  • Proven diagnosis of non-CF BE as documented by computed tomography or high-resolution computed tomography
  • At least 2 or more exacerbations treated with oral antibiotics OR 1 or more exacerbation requiring intravenous antibiotic treatment within 12 months prior to screening.
  • FEV1 ≥ 30% predicted at screening (Visit 1).
  • P. aeruginosa, must be documented in a respiratory sample at least 1 time within 12 months and also present in the expectorated sputum culture at Visit 1.

Key Exclusion Criteria:

  • Patients with a history of cystic fibrosis.
  • Patients with a primary diagnosis of bronchial asthma.
  • Patients with a primary diagnosis of COPD associated with at least a 20 pack year smoking history.
  • Any significant medical condition that is either recently diagnosed or was not stable during the last 3 months, other than pulmonary exacerbations, and that in the opinion of the investigator makes participation in the trial against the patients' best interests.
  • Clinically significant (in the opinion of the investigator) hearing loss that interferes with patients' daily activities (such as normal conversations) or chronic tinnitus. Patients with a past history of clinically significant hearing loss in the opinion of the investigator may be eligible only if their hearing threshold at screening audiometry is 25dB or lower at frequencies 0.5-4 kHz. The use of a hearing device is reflective of a clinically significant hearing loss; hence patients using hearing aids at screening are not eligible.
  • Patients with active pulmonary tuberculosis.
  • Patients currently receiving treatment for nontuberculous mycobacterial (NTM) pulmonary disease.
  • Patients who are regularly receiving inhaled anti-pseudomonal antibiotic (during the study inhaled anti-pseudomonal antibiotics are not allowed other than the study drug).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   France,   Germany,   Italy,   Spain,   United Kingdom
Removed Location Countries Netherlands
 
Administrative Information
NCT Number  ICMJE NCT02712983
Other Study ID Numbers  ICMJE CTBM100G2202
2015-003040-39 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Responsible Party Novartis ( Novartis Pharmaceuticals )
Study Sponsor  ICMJE Novartis Pharmaceuticals
Collaborators  ICMJE
  • Queen's University Belfast, UK
  • University Hospital Antwerp, BE
  • University of Milan, IT
  • Fundacion Clinic per a la Recerca Biomedica
  • Erasmus Medical Center
  • Papworth Hospital Cambridge, UK
  • Royal Brompton Hospital Trust, UK
  • University of Dundee
  • University of Edinburgh
Investigators  ICMJE
Study Director: Novartis Pharma Novartis Pharmaceuticals
PRS Account Novartis
Verification Date August 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP