Pragmatic Pediatric Trial of Balanced vs. Normal Saline Fluid in Sepsis (PRoMPT BOLUS)
To find out which of two commonly used IV fluids given in the Emergency Department for sepsis is most effective.
To find out which of two commonly used IV fluids given in the Emergency Department for sepsis is most effective.
Two main purposes of the study: To see if serious bacterial infections occur less than once a year in each study subject. This will determine if study drug is effective. Also to find out if study drug is safe and tolerable for study subjects.
To demonstrate that an ultra-fast-acting formulation of insulin lispro is non-inferior to Humalog on measures of glycemic control when administered 0 to 2 minutes before eating in combination with basal insulin.
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 Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of NBI-74788 versus placebo administered bid with breakfast and the evening meal (doses separated by approximately 12 hours) for 24 weeks in approximately 165 adult subjects with classic CAH due to 21-hydroxylase deficiency.
To determine the safety and feasibility of autologous umbilical cord blood-derived mononuclear cells delivered into the myocardium of the right ventricle during planned Stage II Glen surgical procedure for individuals with hypoplastic left heart syndrome.
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.
To find out the effects of tenofovir alafenamide (TAF) on children and adolescents with chronic hepatitis B.
Carfilzomib (also known as Kyprolis®) is approved to be used in certain adult patients with relapsed or refractory multiple myeloma (myeloma that returns after a successful course of treatment or myeloma that does not respond to treatment). Multiple myeloma is a type of blood cancer. Carfilzomib is not approved to treat ALL. It has not been given in combination with the other drugs used in this study. This study is being done to find out if carfilzomib can be safely given before and during treatment with standard chemotherapy drugs.
The goals of this study are to improve survival rates in children and young adults with relapsed AML through the combination of DEC and VOR.