The objective of this study is to learn how anemia may affect oxygen delivery to the brain as measured by magnetic resonance imaging (MRI). By studying this, we hope to better identify people who may benefit from correction of their anemia.
This study is a randomized, double-blind, active-controlled, titrated, parallel arm, multicenter study. It will compare the efficacy, safety and tolerability of twice daily Chronocort with twice daily IRHC (Cortef®) over a randomized treatment period of up to 52 weeks in participants aged 16 years and over with known classic CAH due to 21-hydroxylase deficiency. The primary efficacy assessment of biochemical responder rate and the key secondary assessments of dose responder rate and mean total daily dose will be assessed after 52 weeks of randomized treatment.
To evaluate the impact of non-Newtonian behavior on hemodynamic efficiency in children and young adults who have undergone the Fontan procedure.
We are studying the role of gut microbiome diversity on the mucosal and systemic responses to Ty21a vaccine. This is to determine if a given microbiota composition is associated with specific immune responses and to evaluate the impact of an oral typhoid vaccination on the human gut microbiota.
The purpose of this study is to see how young athletes perform sport tasks and to see what movement patterns are related to future injury risk.
To improve patient care and achieve the best health outcomes possible for children with IBD.
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.
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.
