Find a Research Study

To find out the effects of Maralixibat on the treatment of PFIC.

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.

This study will evaluate the effect of Denosumab on lumbar spine mineral density (BMD), as assessed by dual-energy X-Ray absorptiometry (DEXA) at 12 months in children 5 to 17 years of age with GIOP.

This study is a randomized, double-blind, active-controlled, titrated, parallel arm, multicenter study. It will compare the efficacy, safety and tolerability of twice daily Chronocort with twice daily IRHC (Cortef®) over a randomized treatment period of up to 52 weeks in participants aged 16 years and over with known classic CAH due to 21-hydroxylase deficiency. The primary efficacy assessment of biochemical responder rate and the key secondary assessments of dose responder rate and mean total daily dose will be assessed after 52 weeks of randomized treatment.

To find out safety and effectiveness of dupilumab (Dupixent) in infants with severe 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.