The goal of this pilot prospective cohort study is to examine the reproducibility of gastric volume and emptying as measured by gastric ultrasound and its correlation to a gold-standard test for...
The TSC Biosample Repository collects and stores samples of blood, DNA, and tissues that scientists can request to use in their research. The samples we collect are all linked to clinical data in the...
The goal of this study is to evaluate the impact of a clinical decision support system (CDSS) in children receiving mechanical ventilation (MV) after surgery for congenital heart disease (CHD). The...
This is a phase II randomized double-blind placebo-controlled trial that aims at evaluating the safety and tolerability of oral encapsulated fecal microbial transplantation therapy (MTT) in peanut...
This study will evalute the effect of a low glycemic load (LGL diet on dysglycemia, insulin requirements, DXA-derived body composition, gastrointestinal symptoms and quality of life measures in adults...
The primary objective of this investigation is to evaluate the feasibility and safety of implantable loop recorder implantation (using the LUX-DX™) in newborns undergoing stage 1 palliation. The...
The purpose of this study is to determine if postpyloric feedings effectively improve objective measures of pulmonary health in preterm infants with chronic lung disease when compared with nasogastric...
Crohn's disease (CD) is a condition that causes inflammation (swelling, redness) of the lining and wall of the small intestine, large intestine, or both. CD may be associated with abdominal cramps...
The main purpose of this study is to investigate efficacy, pharmacokinetics and safety of the drug in pediatric participants with moderately to severely active ulcerative colitis (UC).
The purpose of this study is to examine if it is feasible to administer decitabine and filgrastim after allogenic hematopoietic stem cell transplant (HCT) in children and young adults with...
This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).
The primary objective of the clinical investigation is to demonstrate successful clinical use of the ThecaFlex DRx™ System in delivering nusinersen in subjects with spinal muscular atrophy (SMA).
All...
The goal of this research study is to understand the acceptability and feasibility of the Sleep ALL Night intervention among children with Acute Lymphoblastic Leukemia (ALL) in hopes of improving the...
Unrecognized coarctation of the aorta (CoA) is a life-threatening component of congenital heart disease (CHD) in which narrowing of the aorta causes obstructed blood flow to the lower half of the body...
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo...
The proposed study includes a newborn developmental intervention to improve neurodevelopmental (ND) and medical outcomes for infants with congenital heart disease (CHD) with improved parent well-being...
The purpose of this study is to learn more about changes in glucose levels in hospitalized infants with intestinal failure receiving parenteral nutrition or PN (nutrients delivered intravenously), as...
This is a phase 2a, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NST-6179 in adult subjects...
