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.
Hydroxychloroquine is given as an investigational drug to relatives of someone with type 1 diabetes who have positive diabetes antibodies in an attempt to prevent or delay the onset of type 1 diabetes. See website for more details https://www.trialnet.org/our-research/prevention-studies/hydroxychloroquine-hcq
To determine if MEDI8897 reduces the amount of visits to see a medical professional due to a lower respiratory tract infection caused by RSV. We will be looking at this in healthy preterm infants entering their first RSV session.
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.
Develop non-invasive measures to identify abnormal placental function in babies with and without congenital anomalies.
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 research is being done to test the safety and effectiveness of intranasal carbetocin to treat Prader Willi Syndrome.
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.
