Timothy R. Aksamit, M.D.

The Bronchiectasis Research Registry is a consolidated database of non-cystic fibrosis (non-CF) bronchiectasis patients from major clinical and research institutions. The goal of the Registry is to support collaborative research and assist in the planning of multi-center clinical trials for the treatment of non-CF bronchiectasis, a progressive, chronic disease of the lungs which afflicts thousands of patients. The Registry is also to be used to provide better insight into the diagnosis, pathophysiology etiologies, and management approaches of the different types of bronchiectasis.

The purpose of this study is to conduct analytic and clinical validation of a CRISPR-based blood test for MAC cellfree DNA as a measure of MAC pulmonary disease activity and treatment response. This biomarker could ultimately be used to guide diagnostic and therapeutic decision-making with regard to MAC pulmonary disease.

The purpose of this study is e 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). Secondary objectives are to create a data repository to be linked to future claims, electronic health record, and/or other existing databases and a biorepository of blood samples.

The purpoes of this study is to evaluate the safety of ORC-13661 and its ability to significantly mitigate or prevent hearing loss in patients being treated with intravenous (IV) amikacin.

To assess the efficacy and safety of 12 months of therapy with inhaled colistimethate sodium in the treatment of subjects with non-cystic fibrosis bronchiectasis chronically infected with Pseudomonas aeruginosa.

The DRCFI treatment will influence the time to first pulmonary exacerbation compared to placebo.

The purpose of this study is to test the hypothesis that benralizumab will reduce exacerbation rates compared with placebo on top of standard-of-care therapy in adult patients with non-cystic fibrosis bronchiectasis with eosinophilic inflammation (NCFB+EI). All patients who complete the 52-week double-blind treatment period on investigational product (IP) may be eligible to continue into an open-label extension (OLE), during which all patients will receive benralizumab. The OLE treatment period is intended to allow patients at least one year of treatment with open label benralizumab.

The purpose of this study is to evaluate the effectiveness, safety, and phage kinetics of multiple inhaled doses of AP-PA02 administered as monotherapy and administered in combination with inhaled antibiotics compared to placebo and inhaled antibiotics alone.

The purpose of this study is to evaluate the effect of brensocatib at 10 mg and 25 mg compared with placebo on the rate of pulmonary exacerbations (PEs) over the 52-week treatment period.The study protocol allows for children ages 12-18 years to be enrolled; however, Mayo Clinic will not be enrolling any subjects <18 years old.

The purpose of this study is to evaluate the effectiveness, safety and tolerability of Amikacin for patients who have a mycobacterial lung disease that is not tuberculosis, and is difficult to treat.

A study to evaluate the effectiveness of Liposomal Amikacin for Inhalation (LAI) when added to multi-drug regimen in subjects with Nontuberculous Mycobacterial (NTM) lung infection caused by Mycobacterium Avium Complex (MAC).

The purpose of this study is to generate evidence demonstrating the domain specification (via modern psychometric methods), reliability, validity, and responsiveness (within-subject meaningful change) of the patient-reported outcome (PRO) endpoints.

The purpose of this study is to evaluate the effectiveness of ALIS + azithromycin (AZI) + ethambutol (ETH) compared to the empty liposome control (ELC) + AZI + ETH on patient reported respiratory symptoms at Month 13.

The purpose of this extension study is to assess the safety, tolerability, and effectiveness of Liposomal Amikacin for Inhalation added to a multi-drug regimen in subjects with non-tuberculous mycobacterium lung infections due to Mycobacterium avium complex who were resistant to therapy in Study INS-212.

The purpose of this study is to determine whether 2-drug therapy is non-inferior to 3-drug therapy against pulmonary mycobacterium avium complex (MAC) disease, and whether 2-drug therapy is better tolerated than 3-drug therapy.

The purpose of this study is to investigate Pseudomonas Aeruginosa and Staphylococcus Aureus bacterial
load, patient characteristics and exploratory biomarkers in adult patients with Cystic Fibrosis or
Non-Cystic Fibrosis Bronchiectasis.

The purpose of this study is to evaluate if the time to first pulmonary exacerbation of bronchiectasis or its frequency can be prolonged by inhalation of ciprofloxacin for 28 days every other 28 days or for 14 days every other 14 days over 48 weeks.