Congenital heart defects (CHD) are the most common major human birth malformation, affecting ~8 per 1,000 live births. CHD are associated with significant morbidity and mortality, and are second only...
The Wiskott-Aldrich Syndrome (WAS) is an inherited disorder that results in defects of the blood and bone marrow. It affects boys because the genetic mistake is carried on the X chromosome. Normal...
The primary objective of this study is to evaluate if Broncho-Vaxom® given to high risk infants for 10 days, monthly, for two consecutive years can increase time to occurrence of the first episode of...
Patients are being asked to be in this research study because medical researchers hope that by gathering information about a large number of children with pulmonary hypertension over time, their...
Neonates, children with single ventricle congenital heart disease, and those undergoing multiple complex cardiac surgeries are at high risk of increased perioperative blood loss, and blood product...
This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders.
This study was designed to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of riociguat at age-, sex- and body-weight-adjusted doses of 0.5 mg, 1.0 mg, 1.5 mg, 2.0 mg and 2.5...
Few studies are specifically designed to address health concerns relevant during pregnancy. The consequence is a lack of evidence on best clinical practice. This includes mothers and their babies when...
The investigators propose a robust therapeutic intervention to ameliorate myocardial ischemia/ reperfusion injury and significantly decrease morbidity and mortality in patients requiring...
The purpose of this initiative is to improve care and outcomes for infants with HLHS by expanding the NPC-QIC national registry to gather clinical care process, outcome, and developmental data on...
PumpKIN is a multicenter, prospective, single-arm feasibility study; Evaluating the investigational Jarvik 2015 VAD in pediatric patients with heart failure. This feasibility trial will enroll 10...
This is a multi-center prospective, single-arm, non-randomized, pivotal study that will continue to access the feasibility of the Xeltis Bioabsorbable Pulmonary Valved Conduit in subjects requiring...
Patients under the age of 5, with a diagnosis of hypoplastic left heart syndrome (HLHS), unbalanced atrioventricular canal (uAVC), or borderline left heart who are undergoing staged LV recruitment...
The goal of this study is to identify significant clinical and laboratory risk factors in pediatric patients with significant upper gastrointestinal bleeding. This is defined as bleeding that...
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 TEAMMATE Trial will enroll 210 pediatric heart transplant patients from 25 centers at 6 months post-transplant and follow each patient for 2.5 years. Half of the participants will receive...
Klippel-Feil syndrome (KFS) was first described in 1912 by Klippel and Feil as a classic triad are comprised of a short neck, a low posterior hairline and restricted motion of the neck. This disease...
CMV infection and disease remain a significant clinical challenge for pediatric solid organ transplant (SOT) recipients. Current prevention strategies are limited to prophylaxis in which antiviral...
The goal of this study is to test the hypothesis that fiberoptic confocal microscopy (FCM) imaging during repair of common congenital heart defects is a useful adjunct for avoidance of conduction...
