1) To determine if 5 doses of Epo (Erythropoietin) 1000 U/kg (birth weight) intravenous (IV) reduces the rate of death or neurodevelopmental impairment (mild, moderate, or severe) at 24 months of age.
2) To assess safety of Epo.
3) To determine whether Epo decreases the severity of HIE-induced brain injury as evidenced by early MRI and plasma biomarkers of brain injury.
To improve patient care and achieve the best health outcomes possible for children with IBD.
We are interested in observing whether and how infants learn when interacting with a robot during a learning assessment contingency paradigm.
5,000 transgender women are testing a LifeSkills Mobile intervention web-app to see if it helps reduce condomless sex, increase PrEP use, and decrease HIV infections over several years.
Our goal is early identification of deviation from healthy brain development to allow targeted early intervention and improve developmental outcomes.
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.
Data Collection only. By reviewing the charts of CAH infants who come to our facility for treatment, we hope to discover which treatments, all of which are standard of care, lead to better outcomes for patients (primarily in the first year of life).
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 an observational study that employs qualitative methods (13 focus groups and 22 individual interviews) targeting 130 sexually active and non-sexually active adolescent and young adult (AYA) females, living with chronic illness or disability. The study is designed to explore the extent to which their experiences living with chronic illness and engagement in ongoing medical management has an impact on their contraception decision making and perception of LARC as a viable option.
