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 ...
The primary objective of the 1701-202 STRONG SCD study is to evaluate the safety and tolerability of different dose levels of IW-1701 compared with placebo when administered daily for approximately 12 ...
The purpose of this study is to assess AR101's safety, tolerability and efficacy over an extended dosing period.
This study evaluates the ability of contrast-enhanced ultrasound to improve the diagnosis of acute appendicitis in children compared to conventional ultrasound.