Clinical Trial
NCT03212677
This is a multi-site study of how nutrition is delivered to critically ill patients in pediatric intensive care units (PICUs) around the world. Each site will include mechanically ventilated children ...
In this research study, investigators want to learn more about the factors that influence children's breathing during sleep and their sleep quality. Specifically, investigators are interested in ...
This is a phase II open label study that will evaluate children ≥ 1 year of age and adults with neurofibromatosis type 1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor, binimetinib. ...
The main goal of this study is to quantitatively assess the sucking and feeding activity of infants at high risk of neurological impairment (preterm infants and term infants at risk of abnormal ...
Part 1 of this trial will enroll healthy volunteers into a single ascending dose (SAD), multiple ascending dose (MAD), and Food Effect (FE) treatment groups. The SAD treatment group is comprised of ...
Acute kidney injury (AKI) is common in children after cardiac surgery with a reported incidence of 20-40%. Pediatric AKI has been found to be associated with important short and long-term adverse ...
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in participants with cystic ...
The purpose of the study is to acquire additional data on efficacy, safety, tolerability, immunogenicity, pharmacokinetic (PK) and other parameters of HYQVIA in pediatric (age ≥ 2 to \<16 years) ...
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat ...