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 (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 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.
This will be a Phase I, double-blind, randomized, placebo-controlled study conducted in 2 parts, single and multiple inhaled dose, at multiple sites. The purpose of this study is to find out about the safety and tolerability of TRK-250 after single and multiple inhaled doses in subjects with idiopathic pulmonary fibrosis (IPF). TRK-250 is an experimental drug which is not approved by the United States Food and Drug Administration (FDA) for the treatment of idiopathic pulmonary fibrosis. This study will be the first time that TRK-250 is administered to humans. This study will be conducted in 2 parts, single inhaled dose (Part A) and multiple
inhaled doses (Part B). Part A of this study will have 6 study visits over a period of up to no more than 9 weeks. Part B of this study will have 7 study visits over a period of 10 weeks.
This study is being done to learn more about the safety of the drug ATYR1923, to see if taking this drug ATYR1923 will allow you to reduce your steroid dose, and to find out more about the effects of different doses of this drug on the lungs of people who have pulmonary sarcoidosis. This study will also look at how the body responds to the drug. These things will be measured by taking samples of your blood, through medical examinations performed by your study doctor, by lung function tests and by reviewing images of your lungs. This study also includes taking blood samples for biomarkers, antibodies, and tryptase and complement levels. A skin lesion biopsy portion of the study is optional. Participation will last up to 28 weeks. You will have a total of 7 study site clinic visits and 8 telephone contacts during the treatment period.
This is a Phase 2 study to determine the efficacy and safety of an experimental study drug called sotatercept. Enrolled patients will receive either sotatercept or placebo (substance that is made to resemble drugs but does not contain an active drug) in combination with your current Pulmonary arterial hypertension (PAH) medication(s), which falls within currently accepted standard of care (SOC) for PAH. There are 4 parts to this research study, a screening period (28 days), a 6-month double-blind treatment period, an 18-month extension period, and an 8-week follow up period. If qualified to continue with treatment, the subject will receive subcutaneous (under the skin) injection(s) of the study treatment through a needle at each dosing visit (multiple injections may be required).
This study is designed to follow individuals who participated in "Phase 1/2 Study of Intravenous or Intrapleural Administration of a Serotype rh.10 Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human Alpha-1 Antitrypsin cDNA to Individuals with Alpha-1 Antitrypsin Deficiency" for 5 years after receiving treatment.