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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).

This is a Phase 1/2, first-in-human, open-label, dose-escalation study designed to evaluate the safety, tolerability, and efficacy of BBP-631 administered to up to 25 adult participants diagnosed with classic congenital adrenal hyperplasia (CAH) (simple virilizing or salt-wasting, Group 1) or with classic salt-wasting CAH (Group 2) due to 21-hydroxylase deficiency (21-OHD) and who are monitored for 24 weeks post-treatment. All participants who receive BBP-631 will be followed for an additional 4.5 years for safety and efficacy in a separate long term follow up protocol (Study CAH-399). In total, all participants will be followed for at least 5 years after the date of treatment with BBP-631.

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.

To find out the effects of dupilumab (Dupixent) on skin infection in eczema.

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 understand how environmental factors affect a child's early brain development. This includes positive factors like social support and parent-child bonding and challenges like poverty, stress and early life exposures.

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 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.