The purpose of this research study registry is to identify patients with advanced sarcoidosis. This is an observational research study, which means you will not take study medication(s). The study will determine how many and how often patients are diagnosed with advanced disease in sarcoidosis clinics. The study will determine how many and how often patients are diagnosed with advanced disease in sarcoidosis clinics. This study will look at genetic markers; determine the risk factors for advanced sarcoidosis and the natural course of symptoms in advanced disease. You will be in the research study for approximately 36 months (3 years)and will be seen every 6 (six) months during the research study.
The purpose of this study is to evaluate the effectiveness of inhaled treprostinil in improving exercise ability in patients with PH (Pulmonary Hypertension) -COPD (Chronic Obstructive Pulmonary Disease) when compared to an inactive solution, or placebo. This is a 34-week study. Approximately 136 subjects will be enrolled at approximately 60 US centers in the Original Design. If the study is adapted to the Contingent Design, approximately 314 subjects will be enrolled at approximately 80 US centers.
Alpha-1 antitrypsin (Alpha-1, AAT) deficiency is an inherited disease which results from a defect in the alpha-1 gene. Severe AAT deficiency causes emphysema predominant chronic obstructive pulmonary disease (COPD). This study is designed to test the effectiveness of an drug (Alvelestat) on lung damage caused by Alpha-1 Antitrypsin Deficiency. This is blinded study and there is a 50% chance of receiving a placebo.
The goal of this cohort and biorepository is to collect data and blood specimens on individuals with Nontuberculous Mycobacteria (NTM), pulmonary disease, and healthy adults to better understand the illness and ultimately improve the care and survival of those with these conditions.
Alpha-1 Antitrypsin (AAT) is a naturally occurring protein involved in the protection of lungs from inflammation. A mutation in the AAT gene (a change in the body's genetic instructions on how to make AAT) causes it to be made incorrectly and very little of it gets into the bloodstream.This results
in the lung damage known as emphysema. ARO-AAT is an investigational drug, which means that it is not approved by the Food and Drug Administration. ARO-AAT works by interrupting a step in the production of AAT. In a patient with AATD, this would stop the mutated protein from being made. This study is being carried out to see how safe and well tolerated ARO-AAT is, and to see if low, medium and high doses of the study treatment will decrease Alpha-1 Antitrypsin in the blood and in the liver compared to a placebo, or dummy injection. The Study medication is given via injection on Day 1, 29 and 133 and then every 84 days. The study includes approximately 17 visits over a period of 24 month. Compensation will be provided for study site visits. .
The purpose of this study is to test the effects of the study drug, RVT-1601, in research subjects with persistent cough due to idiopathic pulmonary fibrosis (IPF) in improving cough (frequency and severity) and quality of life. RVT-1601 is delivered as a mist through an eFlow® nebulizer that research subjects will inhale through a mouthpiece attached to the nebulizer. The Screening period will last up to 4 weeks, in order to give the study doctor time to determine if you qualify for the study. After the Screening Period, you will participate in the study for approximately 24 weeks (6 months). Your total study participation is expected to be 28 weeks (7 months).
Other than Nd:YAG laser, no other comparative studies of thermal ablative therapies with PDT for malignant airway obstruction have been performed. Little has been published about need for additional therapies and changes in quality of life following airway de-obstruction procedures in a randomized fashion comparing different modalities. The aim of this multicenter, prospective, randomized clinical study is to evaluate the efficacy of PDT vs APC.
The purpose of this study is to learn more about the effectiveness and safety of VX-561 used in patients with cystic fibrosis. Patients who have cystic fibrosis and are currently taking a stable dose of ivacaftor (IVA) are invited to take part in this research study. VX-561 is a form of IVA, an approved medication for some types of CF, that lasts longer in the body, allowing the medicine to be taken once a day instead of twice a day. Participation could last for approximately 20 weeks. There will be approximately 88 people participating in this study.
The main purpose of this study is to investigate how well GLPG1690, an experimental drug being investigated for the treatment of Idiopathic Pulmonary Fibrosis (IPF), is tolerated and to see how well GLPG1690 works together with your current treatment of pirfenidone or nintedanib. Approximately 750 participants will take part in the study. It has been described that patients with IPF have increased levels of autotaxin which leads to inflammation of the lungs. It is being investigated whether GLPG1690 can, by decreasing the activity of autotaxin, reduce safely the inflammation in the lungs that occurs in people with IPF, and therefore affect the progression of the disease.