A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
To find out the effects of Maralixibat on the treatment of PFIC.
To find out the effects of Maralixibat on the treatment of PFIC.
We want to learn more about how cancer treatment, such as immunotherapy (i.e., CAR T cell treatment), affects the brain, learning, and behavior in patients with Acute Lymphoblastic Leukemia (ALL).
To evaluate long-term safety of Humira in pediatric patients (between the ages of 6 and 17 years inclusive at the time of enrollment) with moderately to severely active CD who are prescribed and treated in accordance with the approved local Humira product label under the conditions of routine clinical setting.
Safety of Fecal Microbiota Transplantation (FMT) in children with Crohn's disease in remission.
To know if stool transplant from a healthy person into an ulcerative colitis patient can help treat the disease.
To improve patient care and achieve the best health outcomes possible for children with IBD.
The purpose of the study is to collect information from children 0-18 years of age with all types of pancreatitis to determine the number of children diagnosed with this condition, understand the causes and progression of the disease, and review laboratory and imaging findings as well as responses to any treatments given.
To test a new experimental drug Temisirolimus - Temsirolimus (also known as Torisel®) is approved for kidney cell cancer treatment in adults - in combination with approved chemotherapy drugs - in the hopes of finding a drug combination that may be effective against leukemia and non-hodgkin’s lymphoma that has come back after initial treatment. To find the highest dose that can be given without casing severe side effects.
To find out the effects of a chemical in Stevia on the symptoms of NAFLD.
Carfilzomib (also known as Kyprolis®) is approved to be used in certain adult patients with relapsed or refractory multiple myeloma (myeloma that returns after a successful course of treatment or myeloma that does not respond to treatment). Multiple myeloma is a type of blood cancer. Carfilzomib is not approved to treat ALL. It has not been given in combination with the other drugs used in this study. This study is being done to find out if carfilzomib can be safely given before and during treatment with standard chemotherapy drugs.