This is a study to determine the use of recombinant Von Willebrand Factor (rVWF) in the treatment and control of nonsurgical bleeding episodes and bleeding during elective and emergency surgery in children with severe Von Willebrand Disease. The study will last approximately 14 months and will involve regular visits to a research clinic.
When a chest tube is placed, it can be hard for the fluid to drain. Tissue plasminogen activator and DNase are given through the chest tube to help with draining the fluid. We are doing this research to see if early addition of tPA-DNase will help with better fluid draining.
The acute respiratory distress syndrome (ARDS) affects 150,000 people in the US. Because of these mechanical abnormalities of the lung, ARDS patients may require mechanical ventilation. Patients with ARDS that are on mechanical ventilation have a 50% mortality rate. The overall objectives of this study are to demonstrate whether a protocol for the early use of inhaled N-acetylcysteine improves patient care and hospital stay.
This study is for patients that have been diagnosed with 1 of the following types of cancer : Chronic Lymphocytic Leukemia (CLL), Small-Cell Lymphocytic Lymphoma (SLL), or of Non-Hodgkin Lymphoma (NHL) of which there are 2 mains types: B-Cell NHL or T-Cell NHL. The investigational drug being studied is PRT062070; it is also called "cerdulatinib."The primary purpose of Phase 2a of this study is to assess safety and the activity of the study drug in patients who have CLL, SLL, or B-cell NHL, or T-cell NHL. You will take the study drug until you withdraw your agreement to continue to take part in this study, your cancer becomes worse, you have severe side effects, the research study ends, or you have completed dosing with the study drug and all follow-up study visits.
This is a prospective, randomized, open-label, Phase II, multi-center clinical trial for pediatric subjects with congenital or acquired heart disease who are on blood thinners. Subjects will be assigned by chance (2:1) to apixaban,vitamin K antagonist or low molecular weight heparin.
Recruitment will start first for subjects 2 years old to less than 18 years old.
Recruitment will then be followed by subjects 3 months to less than 2 years old.
Recruitment for subjects from 0 to less than 3 months of age will be delayed until study data for infants less than 3 years is available.
This study is for patients who have received a kidney, liver, or pancreas transplant and who have developed resistant Cytomegalovirus infection. There will be two treatment groups to compare the improvement or resolution of Cytomegalovirus infection. Subjects will be randomized to receive 8 weeks of either the investigational drug, Maribavir, or physician assigned standard of care treatment.
This study is for participants who have been diagnosed with head and neck cancer that requires surgery as well as post-operative radiation and possibly cisplatin. The investigational drug in this study is Pembrolizumab. Pembrolizumab is a medication that is currently being tested in multiple cancers. It works by turning on your immune system to recognize and fight your cancer. The purpose of this study is to test the safety and the benefit of adding pembrolizumab to treatment that participants will already be getting for their cancer. Participants can be expected to be in this study for up to 3 years.
A 52 week trial to evaluate the safety and effectiveness of the study drug ifetroban versus placebo, in treating patients with diffuse cutaneous systemic sclerosis or systemic sclerosis associated Pulmonary Arterial Hypertension. Subjects over age 18, with disease duration 60 months or less, along with other inclusion criteria will receive study drug or placebo for one year, along with safety testing.
This study will examine the effect of N-Acetylcysteine (NAC), an over-the-counter antioxidant supplement on brains of youth (ages 15-19) using magnetic resonance imaging (MRI). 55 adolescents will receive, in a counterbalanced order, a 10-day course of NAC 1200 mg twice daily and a subsequent 10-day course of matched placebo twice daily, separated by 11 days. Urine samples will be collected at screening and urine and blood samples will be collected before and after each course of medication treatment. Participants will receive a 1- hour MRI scan at baseline and after each treatment trial.
You/your child could be eligible to participate if he or she is:
Between the ages of 15 and 19.
May or may not drink alcohol.
Participants must provide informed consent and youth under 18 must have parental consent to participate.
Compensation is available to those who qualify.
The purpose of this research study is to provide or continue to provide inhaled treprostinil to eligible subjects who participated in the RIN-PH-201 study. The study will also investigate the safety and how well inhaled treprostinil works in subjects with pulmonary hypertension associated with interstitial lung disease or combined pulmonary fibrosis and emphysema. This study will look at the ability to exercise after taking the study drug. Adults over the age of 18 are eligible. This study is voluntary and will last until you discontinue from the study or the study ends.