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.
The main purpose of this study is to see if important factors can be identified in each of the three categories (patient, catheter, and catheter management) that are linked to blood clotting complications. This is a multicenter, prospective observational study to evaluate risk factors associated with complications of peripherally inserted central catheter (PICCs) and midline catheters. Both adult and pediatric patients with CF who receive care at participating centers will be eligible for participation. This study can last for 2 weeks.
Sepsis is an acute life-threatening illness. Specific aims of this study are to demonstrate the efficacy of combination therapy using vitamin C, thiamine and corticosteroids to reduce the duration of cardiovascular and respiratory organ dysfunction in critically ill patients with sepsis. Additionally, this research study aims to demonstrate the efficacy of combination therapy using vitamin C, thiamine and corticosteroids to reduce 30-day mortality in critically ill patients with sepsis.
The purpose of this study is to compare an imaging procedure called high-resolution computed tomography (HRCT) against the standard pulmonary function tests (PFTs) to see if HRCT is better at showing if the standard glucocorticoid treatment is working. This study also includes testing of blood samples for biomarkers which are measurable indicators of the severity or presence of some disease state. The maximum time you will be in the study is 3 months. During this time, you will need to visit the study site 6 times for 6 site study visits. This study will enroll subjects with a diagnosis of pulmonary sarcoidosis at stage II or III who have shortness of breath (dyspnea) and have not received prednisone or prednisolone (glucocorticoids) treatment or other sarcoidosis therapy within the last three months.
The purpose of this research study is to evaluate the long-term safety and tolerability of the study drug called nintedanib in subjects with Systemic Sclerosis and associated Interstitial Lung Disease. Patients who have completed (did not prematurely discontinue trial medication) SENSCISTM (1199.214) can participate in this study. Unlike 1199.214, patients will will know the dose of the study drug. Treatment duration for each patient will be variable. Treatment will be stopped if a reason for withdrawal is met The patient will be in this study about 3 years and have about 26 visits to the study center. These visits include physical exams, blood and urine samples, urine pregnancy testing, collection of unused study drug, and receiving the study drug supply at the reduced dose.
The purpose of this study is to evaluate the efficacy and safety of open-label exposure of gNO in patients with NTM lung disease. Subjects will receive the study drug by inhaling through a nasal mask. Subjects will be treated for 3 weeks (5 days per week) and followed monthly for 3 months.
This is a research study to find out the safety and efficacy of a drug called BG00011 and to find the best dosage. The study drug will be given by an injection under the skin, also called subcutaneously (SC), once a week during the treatment period. Study treatment will be given to you and you will be trained on how to inject yourself with the study drug/placebo in a prefilled syringe. You can ask for re-training at any time during the study. You will be in the study for up to 65 weeks (including a Screening visit, a Study Treatment Period of 52 weeks, and a Safety Follow-Up visit). This study is a randomized, double blind, placebo controlled study.
This is a research study of different ways to use intravenous fluids (fluids given through a small tube placed in the vein) and vasopressors (medicines used to raise blood pressure) to treat sepsis, which is a serious infection. We are asking you to be in this study because: a) you have been diagnosed with low blood pressure due to an infection and b) your blood pressure has stayed low after your doctors gave you fluids. We do not know which approach is better in this situation: a)starting medicines to raise blood pressure first and then giving more fluids (if needed), or b) giving a larger amount of fluids first and then giving medicines to raise blood pressure if needed. Right now, the choice of approach is left to the doctors. Some doctors use medicines to raise blood pressure followed by extra fluids, and others use extra fluids followed by medicines to raise blood pressure. Some doctors use a combination of the two. This treatment part of the study will last for 24 hours, and then we will follow you until you go back to where you live. We want to find out whether one of these approaches compared to the other can improve a patient's chances of survival.
The purpose of this stuThe purpose of this study is to learn more about the safety and effectiveness of VX-445 used in combination with TEZ and VX-561 in patients with cystic fibrosis. Participation could last for approximately 100 weeks. This will include a treatment period that will last approximately 96 weeks, and a safety follow-up period that will last approximately 4 weeks after the last dose of Study Drug. Approximately 400 people will participate in this study.