Remodulin for the treatment of Persistent Pulmonary Hypertension of the Newborn (PPHN)
To explore the safety and treatment effect of intravenous (IV) Remodulin as add on therapy in neonates with PPHN compared to placebo.
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.
Intervention Study for Type 2 Diabetes.