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.
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.
The primary aim of this registry will be to identify genes and/or biologic and environmental factors that either cause these tumors or increase one's risk for developing them. Ultimately, research using this registry may result in improved diagnosis, more effective treatments and possibly prevention.
The objective of this study is to better understand how a single ventricular heart with Fontan physiology may affect brain health.
The objective of this study is to better understand the effects of chronic diseases, such as congenital heart disease, on blood vessel function and on blood characteristics.
To investigate the connection between diet, genetics, and diseases in Hispanic / Latino youth.
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).
To find out the effects of ApoE4 on the Preterm Brain.
Learn more about healthy baby development and develop new tools for pediatricians so they can better support the unique development of each child.
To explore the safety and treatment effect of intravenous (IV) Remodulin as add on therapy in neonates with PPHN compared to placebo.
