This study aims to use new technology involving molecular and imaging tests to help detect liver cancer early in patients. Participants enrolled will participate in it for approximately 5 years. The purpose of this study is to determine if an experimental drug, INT-787, is safe and effective in patients who are hospitalized due to severe alcohol-associated…
The purpose of this study is to compare the effects, good or bad, of tiragolumab plus atezolizumab plus bevacizumab versus placebo plus atezolizumab plus bevacizumab on patients with HCC. In this study, participants will be randomized you will get either tiragolumab plus atezolizumab plus bevacizumab or placebo plus atezolizumab plus bevacizumab. A placebo…
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem cells, modifies them by CRISPR technology, and infuses them back to the…
The purpose of this study is to learn about whether the study drug (PF-07209326) is safe and effective in people with sickle cell disease and learn about what the right dose is. We will look at drug levels in the blood, see how fast drug levels rise and fall, and what effects the drug has, all of which are known as the pharmacokinetics (PK) and…
This study involves two infusions of either rituximab or placebo, two right heart catheterizations (6 months apart), and a variety of other clinical study procedures and exams.
The purpose of this research is to learn how to help parent youth pairs work together to help youth with sickle cell disease take hydroxyurea every day as a daily habit. This study is for children and teens ages 10-18 with Sickle Cell Anemia taking hydroxyurea, and their parents (or guardians). Many children and teens do not use hydroxyurea every day. Daily…
The purpose of this study is to learn more about TPST-1120. The aims of the study are to find out if TPST-1120 can be given safely to people, to determine a safe dose of the study drug, if it is safe when given in combination with other anti-cancer drugs, what effects the drug may have, and to learn more about how the body processes the study drug. This is…
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle cell disease. Patients will receive busulfan…
Patients will be asked to participate in this research study because they have been diagnosed with hepatocellular carcinoma (HCC) which is a type of liver cancer. By doing this study, researchers hope to: Find out more about patients that have HCC including their age, race/ethnicity, and other medical conditions they may have; learn more about how…
The purpose of this study is to study the effects of a new study drug known as Riociguat in people diagnosed with pulmonary hypertension (PH) associated with idiopathic interstitial pneumonia (IIP). Patients will be in the study for approximately 26 weeks in which they will take the pill Riogciguat three times a day.
This is a clinical research study designed to determine the safety and effectiveness of the TIVUS system, an investigational device used in the treatment of PAH. The TIVUS System is intended to treat patients with pulmonary artery hypertension by injuring the sympathetic nerves surrounding the pulmonary vasculature (denervation) using ultrasonic energy. The…