Fetal Substudy (Congenital Heart Disease & Control)
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.
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.
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.
To explore the safety and treatment effect of intravenous (IV) Remodulin as add on therapy in neonates with PPHN compared to placebo.
This is a randomized, double-blind, placebo-controlled study that will evaluate the potential of tildacerfont to reduce GC burden in adult subjects with classic CAH who have LLD ≤ A4 ≤ 2.5x ULN and are on supraphysiologic doses of GC therapy (≥30 mg/day and ≤60 mg/day HCe). This will be the first study of tildacerfont to evaluate GC dose reduction. In addition, this study will characterize clinical outcomes after up to 76 weeks of treatment with tildacerfont. An optional Open-Label Extension Period will provide an open-label treatment with tildacerfont at 200 mg QD for up to 240 weeks.