The purpose of this study is to evaluate the safety and tolerability of VX-522 in participants 18 years of age and older with cystic fibrosis and a cystic fibrosis transmembrane conductance regulator ...
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...
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 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).
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...
