Non-Cystic Fibrosis Bronchectasis - Lung disease
Conditions
Interventions
Each subject will receive study treatment for 24 weeks. The entire study is
scheduled to take a maximum of 32 weeks for each individual subject from
Screening (Visit 1) to the End of Study (Visit 10
Sponsors
Insmed Incorporated
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Subjects who have given their signed, informed consent, are male or female between 18 and 85 years of age (inclusive) with a body mass index > 18.5 at Screening (Visit 1) and have a clinical history consistent with NCFBE will be eligible for enrollment in the study.
Exclusion criteria
Exclusion criteria: Subjects who have a primary diagnosis of chronic obstructive pulmonary disease or asthma, have bronchiectasis due to cystic fibrosis, hypogammaglobulinemia, common variable immunodeficiency, or *1-antitrypsin deficiency, are current smokers as defined per Centers for Disease Control and Prevention criteria, or currently being treated for a non tuberculous mycobacterial lung infection, allergic bronchopulmonary aspergillosis, tuberculosis or have a clinical diagnosis of Papillon-Lefèvre Syndrome will not be eligible for enrollment in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint is the time to the first pulmonary exacerbation over the 24-week treatment period. A pulmonary exacerbation in this study is defined as having 3 or more of the following symptoms for at least 48 hours resulting in a physician*s decision to prescribe antibiotics: 1 Increased cough; 2 Increased sputum volume or change in sputum consistency; 3 Increased sputum purulence; 4 Increased breathlessness and/or decreased exercise tolerance; 5 Fatigue and/or malaise; 6 Hemoptysis. Subjects on chronic macrolide therapy whose only change in therapy is dose or frequency adjustment will not meet the definition of exacerbation. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Endpoints: 1. Change from Baseline in QOL-B Respiratory Symptoms Domain score over the 24-week treatment period. 2. Change from Screening in post-bronchodilator FEV1 over the 24-week treatment period. 3. Change in concentration of active NE in sputum from pre-treatment (defined as the average of Screening and Day 1 concentrations) to on-treatment (defined as the average of Week 12 and Week 24 concentrations). 4. Rate of pulmonary exacerbations (number of events per person-time) over the 24-week treatment period. Safety Endpoints: The safety endpoints will include AEs, 12-lead ECG measurements, clinical laboratory testing results, vital sign measurements, physical examination results, and PFT measurements. Adverse events of special interest (AESIs) which will be reported include hyperkeratosis, periodontitis/gingivitis, and other infections. Pharmacokinetic Endpoints: The PK parameters, as estimated using non-compartmental analysis methods, will include maximum plasma concentration (Cmax), time to maximum plasma concentration (Tmax), average plasma concentration at steady state (Css), maximum plasma concentration at steady state (Cmaxss), time to maximum plasma concentration at steady state (Tmaxss), minimum plasma concentration at steady state (Cminss), and area under the plasma concentration curve from 0 to 8 hours (AUC0-8). | — |
Countries
Netherlands
Outcome results
None listed