Part A of this study will compare the effects of Clofazimine Inhalation Suspension to placebo. The purpose of this study is to find out if Clofazimine Inhalation Suspension can treat NTM lung disease by getting rid of the bacteria from the lungs and to make sure that Clofazimine Inhalation Suspension is safe for use. The participant will continue with current treatment for NTM lung disease in this study. To test if the bacteria are gone from the lungs, sputum will be tested on a regular basis (every month) to see if the culture changes from positive to negative. Participants will be randomly assigned by chance (like drawing numbers from a hat) to either Clofazimine Inhalation Suspension or placebo.
After the participant completes the study treatment in Part A, they will be eligible to receive Clofazimine Inhalation Suspension in Part B.
Approximately 234 participants will take part in the study at approximately 120 sites globally.
This study is to evaluate an investigational study drug, itepekimab, for the treatment of bronchiectasis. The main purpose for this study is to assess the safety, efficacy, and tolerability of itepekimab in bronchiectasis in addition to the current background treatment you are receiving which may include bronchodilators, inhaled corticosteroids, mucolytics, and/or maintenance antibiotics. You will receive either the study drug or a placebo if you participate in this research. This study will include about 300 participants with bronchiectasis across approximately 20 countries worldwide.
This research seeks to gather important insights about patients' experiences of NCFBE-related exacerbations and brensocatib treatment by conducting semi-structured qualitative interviews in the USA. A total of up to 30 interviews of approximately one hour are planned. Patient must be at least 18 years old and currently participating in the brensocatib PTA program for at least 3 months.
This is a prospective, longitudinal, multicenter, nonrandomized observational study to obtain research quality data across key outcome measures in people with Cystic Fibrosis who are ineligible and/or not taking a approved CFTR modulator and who are not receiving an investigational therapy.
Participants will be seen at study sites for research visits to include spirometry, patient reported outcomes (PROs), and blood collections on Day 0, Day 90 (3 months), Day 180 (6 months), and Day 360 (12 months). Participants will complete home spirometry as well during this period.
We will follow 146 new parents of children <5 years of age at 18 participating US
adult CF centers to assess the primary outcome of ppFEV1 up to 5 years after
becoming a parent. A prospective approach will capture the immediate and longterm impact of the use of the highly effective CFTR modulator ETI by ~90% of US
adults with CF. By combining objective health measures and participant surveys,
we can comprehensively assess the psychosocial impacts of parenthood and
explore the interplay between the parenting role and physical and mental health.
We anticipate identifying modifiable factors that may ameliorate negative health
impacts of parenthood.
Nontuberculous mycobacteria (NTM) cause a chronic pulmonary infection associated with cough, fatigue, and shortness of breath. Our primary objectives are to better understand the trajectory of patient-reported outcomes (PROs), e.g.
respiratory symptoms and health-related quality of life (HRQoL), across the entire disease course and measure toxicity and tolerability using patient-reported symptomatic adverse events in treated patients with nontuberculous mycobacterial pulmonary disease (NTM-PD).
This is a double-blind, randomized, placebo-controlled study of CMTX-101 in pwCF who are 18 years of age or older and chronically infected with P. aeruginosa. This study will evaluate the safety and tolerability, PK, immunogenicity, reduction of pulmonary P. aeruginosa burden, and exploratory endpoints of CMTX-101 in up to 41 participants. This study has 2 parts.
The purpose of this study is to evaluate the safety and tolerability of single ascending doses (SAD) of VX-52 in patients with Cystic Fibrosis and the CFTR genotype who have not been responsive to CFTR modulator therapy. This is a first in human study.
This is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety, tolerability, and efficacy of AP-PA02 administered by inhalation. This study will evaluate AP-PA02 administration in stable NCFB (non-cystic fibrosis bronchiectasis) patients. Subjects will either be included in Cohort A or Cohort B. For Cohort A, subjects will be randomized to receive either inhaled AP-PA02 or placebo. Cohort A will include individuals with NCFB and confirmed chronic P. aeruginosa infection but not on chronic inhaled antibiotics. These individuals will receive wither AP-PA02 or placebo for 10 days twice a day.
Cohort B will include individuals who with NCFB and confirmed P. aeruginosa infection but who are on chronic antibiotics. These individuals will receive either AP-PA02 or placebo for 10 days plus their current inhaled antibiotics for 28 days.
The Program will allow access to brensocatib for patients who have completed the INS1007-301 ASPEN Clinical Trial. Patients will receive brensocatib 10 mg orally once daily. Eligible, compliant patients may receive brensocatib in this program until the drug is commercially available or until Insmed terminates the program.