The purpose of this study is to determine the efficacy of enhanced preoperative education using a Pediatric Interactive Relational Agent (PIRA), designed for children aged 4 through 10 years and their ...
This phase I/II clinical trial is an open-label clinical trial design to verify safety and dosing for TAVT-18 (sirolimus) powder for oral solution in TSC infants (N=5).
This is a prospective observational study designed to evaluate Immature Platelet Fraction or Immature Platelet Count and Platelet Function Analyzer-100/200 Closure Time-ADP (in vitro bleeding time) as ...
The incidence of moderate to severe congenital heart disease (CHD) in the United States is estimated to be 6 per 1000 live-born full term infants. Recent advances in pediatric cardiology, surgery and ...
This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor (ETI) triple combination therapy in ...
Dravet syndrome (DS) is an epileptic encephalopathy caused by pathogenic variants in the SCN1A gene resulting in medically refractory epilepsy and psychomotor delays. As a pilot study assessing for ...
The goal of this prospective study is to validate a wearable EEG seizure detection solution compared to video EEG. Subjects that undergo a clinical video EEG are asked to additionally wear a wearable ...
This study proposes to quantify and describe the quality of life of children with intestinal failure, and to identify the medical and socio-economic factors that impact this quality of life, using ...
Open-label, dose escalation, multi-center, Phase I/II clinical trial to assess the safety/tolerability and determine the recommended Phase II Dose (RP2D) of ET140203 T-cells in pediatric subjects who ...
Background: During the first few decades of life, the brain changes dramatically in shape and function. Sleep lets researchers measure these changes. Researchers want to create a database of sleep ...