The TRANSFORM network is focused on improving child health outcomes in SC through research on conditions of highest priority for future generations. These include conditions like asthma, autism, diabetes, obesity and early childhood outcomes. To accelerate research study recruitment activities for future projects that TRANSFORM sites may participate in, the TRANSFORM network sites will create a research recruitment registry of families interested in volunteering for research. Participants will enter their own as well as their child(ren)'s information into the electronic registry which can be used to identify people to contact for future studies.
This is a prospective, randomized, open-label, Phase II, multi-center clinical trial for pediatric subjects with congenital or acquired heart disease who are on blood thinners. Subjects will be assigned by chance (2:1) to apixaban,vitamin K antagonist or low molecular weight heparin.
Recruitment will start first for subjects 2 years old to less than 18 years old.
Recruitment will then be followed by subjects 3 months to less than 2 years old.
Recruitment for subjects from 0 to less than 3 months of age will be delayed until study data for infants less than 3 years is available.
We will combine state-of-the-art brain imaging techniques in 140 Single Ventricle Reconstruction Trial (SVR) III patients and 100 control subjects with innovative brain connectome or "graph" analyses to determine if brain connectivity graph measurements will provide novel neuroimaging biomarkers for neurodevelopmental disabilities and improve our understanding of their inciting mechanisms in the SVR survivors. Only 6 of the SVR III patients and no controls will be enrolled at MUSC.
Multiple studies have shown a progressive decline in measured aerobic exercise capacity during adolescence in survivors of the Fontan procedure. When maximal oxygen consumption falls below a threshold of approximately 50% of predicted for age and gender the risk of cardiopulmonary failure, death, or need for heart transplantation increases significantly. A therapy which improves or reduces this observed decline in exercise capacity might forestall the onset of heart failure and prolong transplant-free survival. This is a randomized, double-blind, placebo-controlled clinical trial of a 6-month (26 week) treatment with udenafil (87.5 mg twice daily). The primary efficacy measurement will be improvement in aerobic exercise performance relative to baseline. The secondary efficacy measurements will examine 1) Improvement on echocardiographic indices of systolic and diastolic ventricular performance relative to baseline; 2) Improvement on endothelial function relative to baseline; 3) Improvement on serum brain-type natriuretic peptide (BNP) level, a biomarker of heart failure, relative to baseline; 4)To determine if six months of treatment with oral udenafil will alter functional health status in adolescents following the Fontan procedure. Safety will be assessed by monitoring adverse events and vital signs, clinical laboratory test results, 12-lead ECG, and physical examinations.
The majority of drugs administered to children are used off label and PK studies to define appropriate dosing are lacking across pediatric age groups. Challenges associated with clinical trials in children limit the ability to conduct PK and dosing trials in this population. Studies capitalizing on standard of care procedures have proven successful in characterizing the PK of drugs used in children.
The purpose of this study is to characterize the PK of understudied drugs administered to children per standard of care as prescribed by their treating caregiver.
This study will serve as a tool to better understand drug exposure in children receiving drugs per standard of care. The data collected through this initiative will provide valuable PK and dosing information drugs in different pediatric age groups as well as special pediatric populations (i.e. obese).