Fecal Microbiota Transplantation (FMT) in Ulcerative Colitis
To know if stool transplant from a healthy person into an ulcerative colitis patient can help treat the disease.
To know if stool transplant from a healthy person into an ulcerative colitis patient can help treat the disease.
Safety of Fecal Microbiota Transplantation (FMT) in children with Crohn's disease in remission.
To assess the survival rate of subjects at 6 and 12 months following implantation of the Bioabsorbable pulmonary valve conduit. To assess the rate of conduit failures at 6 months, requiring re-intervention or reoperation.
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.
The purpose of this study is to (1) determine the minimum effective dose of abiraterone acetate that normalizes androstenedione levels in prepubertal children with CAH secondary to 21-hydroxylase deficiency (Phase 1 trial), and (2) assess the utility of abiraterone acetate in prepubertal children with CAH as adjunctive therapy to minimize excessive androgen secretion and allow more physiological glucocorticoid replacement (Phase 2 trial).
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.
To find out the effects of dupilumab (Dupixent) on skin infection in eczema.
To determine the safety and feasibility of autologous umbilical cord blood-derived mononuclear cells delivered into the myocardium of the right ventricle during planned Stage II Glen surgical procedure for individuals with hypoplastic left heart syndrome.
To test the effectiveness of an addiction-based weight loss intervention, embodied first as a smartphone app with telephone coaching (AppCoach) compared to (1) addiction model based weight-loss app alone (App) and (2) multi-disciplinary in-clinic weight loss intervention (Clinic) on weight outcomes of overweight and obese adolescents at 3, 6, 12 and 18 months post enrollment.
To collect information on the long-term effects of XLH on children and adults who have participated in burosumab clinical trials, adults and children who have not participated in burosumab clinical trials, and who may or may not have been treated with burosumab during the course of the study.