The NEAT Study is about obesity, appetite, emotions, and how the brain develops in children. The study team is looking for volunteer youth to participate who are either healthy or have a clinical condition called Congenital Adrenal Hyperplasia (CAH).
This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered twice daily (bid) with breakfast and evening meals for28 weeks in approximately 81 pediatric subjects with classic congenital adrenal hyperplasia (CAH) due to21-hydroxylase deficiency. Eligible subjects will be randomly assigned in a 2:1 ratio (active:placebo) to either crinecerfont (25 mg bid via oral solution for subjects 10 to <20 kg, 50 mg bid via oral solution for subjects 20 to <55 kg, or 100 mg bid via oral capsules for subjects ≥55 kg) or matching placebo (oral solution placebo for subjects <55 kg and oral capsule placebo for subjects ≥55 kg). Dose assignment from Day 1 to Week 28 will be based on the subject’s weight at Day 1. After the 28-week placebo-controlled treatment period, there will be a 24-week, open-label treatment period, during which all subjects will receive crinecerfont at doses based on their Week 28 body weight.
To find out which of two commonly used IV fluids given in the Emergency Department for sepsis is most effective.
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.
The purpose of the study is to learn about how to improve access to care during adolescence/young adulthood and to see if Virtual Team Clinic is an acceptable form of care. Virtual Team Clinic is a new approach to provide care to (middle/high) school aged patients living with Type 1 Diabetes and their families. This new approach consists of Virtual Team Clinic appointments (primarily telemedicine, and/or in person as necessary). The study also incorporates group visits/medical appointments with a multidisciplinary care team (e.g., RD, RN, Social Worker, etc.). Each group will have a special theme and learning experience. You will learn how to better take care of your diabetes, get support from others, and hopefully have fun.
