The purpose of this study is to assess safety of the study drug, inhaled treprostinil, and the ability to tolerate long-term treatment with the study drug in patients with PH-COPD. The study will also evaluate the effectiveness of the study drug over long-term treatment. Inhaled treprostinil is a medication that will be given to you by a handheld inhalation device (study nebulizer) called the Tyvaso® Inhalation System. The study is estimated to last 2 to 3 years but could be much shorter or longer. Depending on the final number of subjects who participated in the RIN-PH-304 study, either approximately 136 or approximately 314 subjects may be eligible to participate in this study from the U.S. study centers.
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. Severe AAT deficiency (lack of AAT in the blood) causes emphysema, which causes holes in the lungs. This study is being done to learn more about the safety, tolerability and effectiveness of VX-814 in patients with Alpha-1 Antitrypsin Deficiency. There are four groups in the study, one placebo group and three treatment groups with varying doses. This study is double blind with an 80% chance of being assigned to a treatment group and a 20% chance of being assigned to placebo.
This study is for patients that have been diagnosed with Small-Cell Lung Cancer. The purpose of this study is to find out if this approach is better or worse than the usual approach. The usual approach is defined as care that most people get for small cell lung cancer. Participants can expect to be on this study for two years.
The purpose of this study is to determine whether the using specific breathing strategies during anesthesia for patients having surgery on their liver and intestines in an effort to help them breathe better after surgery. This will be tested with an incentive spirometer before and after surgery and those results will be compared to people having similar surgery without these extra strategies.
This study is being done to compare the safety, effectiveness, and side effects of different doses of the drug being studied, BI 1265162, with a placebo, in subjects with Cystic Fibrosis (CF). You will be in this study for about 7 weeks. This study will involve study procedures such as, physical and chest exams, blood pressure and pulse, electrocardiograms, blood tests, pregnancy urine tests, breathing tests (pulmonary function tests and multiple breath washout tests), and completing a diary and questionnaires. Each dose of the study drug will consist of 2 puffs which will be taken 2 times per day using the Respimat® inhaler. This is an add-on therapy therefore subjects will continue to take their usual medications (including any therapy), and the treatment period for taking this add-on therapy is 4 weeks.
The purpose of this study is to learn more about the effectiveness and safety of the investigational drug called fenretinide (LAU-7b). The FDA has approved the testing of fenretinide in this study. Researchers hope that treatment with fenretinide will preserve lung function by reducing the persistent inflammation in the lung and to improve its capacity to defend against bacteria such as Pseudomonas aeruginosa. The drug will be administered by capsules. Your participation in this study will last approximately 7 months and includes 8 study visits to the study center (which will last approximately 2-5 hours) and 6 telephone calls. The phone calls are expected to last 10-15 minutes and depend on the amount of information you report.
This study is designed to evaluate ABBV-3067 alone and in combination with ABBV-2222 to see how safe they are and how well they work for treating cystic fibrosis. The study will also help select the minimum effective dose of both medicines. Both drugs are investigational and will be taken orally. Both help to restore Cystic Fibrosis transmembrane conductance regulator (CFTR) activity, which is when there isn't enough water on membrane surfaces for those who have cystic fibrosis. You will either receive the drug or a placebo. The study will be done in approximately 3 months. Approximately 189 patients with cystic fibrosis who are older than 18 years of age will be enrolled in this study.
The primary purpose of conducting this study is to see if TLD (Targeted Lung Denervation) Therapy in addition to standard optimal medical care is better at reducing a moderate or severe exacerbation (also known as a COPD flare-ups or worsening of symptoms) and related hospitalizations than optimal medical care alone. TLD Therapy is done by passing a bronchoscope, with a special device (catheter) inserted through it, into the lungs. This special catheter delivers a type of electrical energy called radiofrequency (or RF) energy to the nerves located on the outside of the airways. As with many bronchoscopic procedures, this is done while under anesthesia. TLD Therapy does cause a permanent change to a person's lungs. To test this, participating patients will be randomly assigned (in a 1:1 ratio) to receive one of two different treatments, either TLD Therapy in addition to optimal medical care or optimal medical care alone.
Some sites, including MUSC, will also be collecting 3 airway brushes to look at inflammatory biomarkers in the lungs. A biomarker is anything that can be used as an indicator of a particular disease state.
If you choose to participate in this study, it is estimated that you will be involved in this study for approximately 62 months. Participation will take around 11 clinical site visits and 9 follow up phones calls over a period of 5 years. The participant and person obtaining consent will sign the informed consent form and the participant will receive a copy before any study procedures occure.
This is a trial for cardiac sarcoidosis in patients seen during routine clinical care who have not been clinically suspected to have cardiac sarcoidosis according to conventional criteria. The addition of echocardiogram and ambulatory ECG to usual clinical follow-up will be compared. This is a multicenter study, coordinated by the Foundation for Sarcoidosis Research. Up to 12 sites in the US and Europe will participate. Each site will enroll 50 consecutive consenting individuals to a total of 600 enrolled subjects in a randomized trial of additional screening versus usual care.
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.