TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three-arm clinical trial of immunosuppressive therapy for children with acute liver failure. This is a double-blind study, so the study doctors and participants will not know to which arm the participants are assigned. The study will determine if corticosteroids or equine anti-…
This study is for individuals who have Cushings disease and want to join voluntarily in this study to find out if a study drug called osilodrostat (LCI699) is safe and has beneficial effects in people who have Cushings disease. Cushings disease is a rare but debilitating disease. Cushings disease is caused by a benign tumor of the pituitary gland. The tumor…
The purpose of this study is to determine if measuring blood levels of proteins called POMC, AgRP, and chromogrannin A (CGA) can be used to diagnose and track tumor growth and recurrence in patients that have ACTH-dependent Cushing's syndrome, non-secreting pituitary tumors, and other neuroendocrine tumors. We invite you to participate if you have: a…
The purpose of this research study is to determine if an investigational drug, VS-01, will help in the treatment of acute-on-chronic liver failure (ACLF) compared to supportive care (standard of therapy). ACLF is a potentially life-threatening condition characterized by a group of signs that occur together (syndrome) in patients with a late stage of…
The objective of the LIFE-BTK RCT is to evaluate the safety and efficacy of the ESPRIT BTK device (which is a bioresorbable polymeric scaffold with the everolimus drug and a bioresorbable polymeric coating mounted on a balloon dilatation catheter) compared to Percutaneous Transluminal Angioplasty (PTA), which is is a procedure that can open up a blocked…
The purpose of this study is to confirm the effectiveness and safety of LCI699 in treating patients with Cushing's disease. Patients will be treated with the invesitigational drug LCI699 at doses up to 60 mg a day, and possibly for a short period of time with placebo. A placebo is a dummy drug ("pill") with no active medicine inside.