Find a Research Study

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.

Abatacept is given as an investigational study drug to relatives of someone with type 1 diabetes who have positive diabetes antibodies in an attempt to prevent or delay the onset of type 1 diabetes. See TrailNet.org website for more details.

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

Develop better ways of screening and diagnosing certain types of brain injuries in infants and children who are born with and without congenital heart disease.

Hydroxychloroquine is given as an investigational drug to relatives of someone with type 1 diabetes who have positive diabetes antibodies in an attempt to prevent or delay the onset of type 1 diabetes. See website for more details https://www.trialnet.org/our-research/prevention-studies/hydroxychloroquine-hcq

To determine if MEDI8897 reduces the amount of visits to see a medical professional due to a lower respiratory tract infection caused by RSV. We will be looking at this in healthy preterm infants entering their first RSV session.

The collection of the research data we hope will help better screening, diagnosing procedures and treatment of brain injury in newborns and identify a connection between MR imaging and neurodevelopmental outcomes.

Develop non-invasive measures to identify abnormal placental function in babies with and without congenital anomalies.

To explore the safety and treatment effect of intravenous (IV) Remodulin as add on therapy in neonates with PPHN compared to placebo.