The primary purposes of this study are to:
•Provide access to cord blood units for recipients whose best choice for a cord blood unit(s) do not meet all FDA standards, but do meet standards set by the NMDP on this study.
•Assess how well and how quickly blood counts return to normal after transplant in recipients on this study.
Genetic Testing of neonates undergoing surgery for single ventricle cardiac defects (SVCD) and other congenital cardiac defects. DNA testing with an aim to identifying genetic factors that aid survival and recovery in SCD patients.
Genetic contribution to patient outcomes: Over the past two decades, there has been dramatic improvement in the survival and functional outcome of patients with all forms of congenital cardiac defects. Yet, there exists significant variability in outcomes that becomes more pronounced as the level of surgical intervention increases and the exposure to adverse hemodynamic conditions becomes more prolonged and more profound. This is particularly noticeable in the SCD patient group where there are continued high levels of mortality and levels of disability that can be quite severe. While these poor outcomes can on occasion be attributed to technical difficulties, complex cardiac anatomy or patient co-morbidities, more commonly they occur in patients that do not superficially appear to be any different than those that will ultimately have excellent outcomes. What is becoming increasingly apparent is that every patient differs in their ability to tolerate the challenges presented by the peri-operative environment. Therefore, significant improvements in outcomes may depend on identification of the genetic factors that place some patients at greater risk and designing treatment protocols to minimize those risks.
This study is being offerred to patients that have acute myelogenous leukemia (AML) which is a cancer of the blood and these patients are going to have a stem cell transplant. This study is looking to determine how accurate two different laboratory tests are at detecting residual, or small numbers of cancer cells in the body before and after stem cell transplant, as well as whether or not results of these two tests show how well a recipient might do after transplant.
To increase adherence among a group of twenty 3-9 year old children to their prescribed diet and insulin regimen at the Pediatric Endocrine Outpatient Nutrition clinic and for caregivers to be able to verbalize understanding of diet recommendations and meal preparation techniques as instructed during the inpatient hospital education (and referenced to in the children's storybook). We are utilizing standard of care data (routine ht., wt., BMI-for-age and A1C) collections for this study.
This study if for patients that have a blood disease and it's been determined that the best option for treating that blood disease is a cord blood transplant. Cord blood (CB) is blood that is taken from the umbilical cord and placenta of healthy newborn babies after childbirth. The cord blood collected from a newborn baby is called a cord blood unit. The United States Food and Drug Administration (FDA) considers cord blood to be a biological drug. These are considered “investigational” products. This study will evaluate the safety of administration of the investigational cord blood units by carefully documenting all infusion-related problems.
This is an 8 week drug study for children 6 to 17.5 years of age who have been diagnosed with hypertension. The purpose is to test the safety, tolerability and effictiveness of the study medication, Aliskiren. The study has 3 phases:
Screening Phase: All subjects will "wash out" (be on a placebo and no hypertensive medication) for a period of 7 - 21 days, during which they will be closely monitored the the physicians and research study staff.
Phase 1: (4 weeks/28 days) After the wash out period, all subjects will given the study drug, Aliskiren, with dosing based on weight.
Phase 2: (4 weeks/days). Patients will be randomized to receive either placebo or continue the aliskiren treatment assigned during Phase 1.
Once subjects have completed the 8 week study, they will be eligible to enroll in a 52 week extension study which looks at the long term safety, efficacy and tolerability of the study drug.
Capillary leakage leading to persistent pleural effusions and multiorgan dysfunction continue to be a significant post-operative problem in children following cardiopulmonary bypass (CPB). Studies to be performed as part of this research program will test the hypothesis that children with persistent pleural effusions have insufficiencies in an HDL-associated lipid, sphingosine-1-phosphate (S1P), that controls blood vessel barrier maintenance. If plasma levels of S1P are found to be predictive of persistent pleural effusions and prolonged post-operative recovery time after CPB then exogenous administration of S1P may hold promise in improving post-operative recovery after CPB.
A follow-up study (Fontan 2) at an average of 6 years after Fontan 1 has recently been completed. A limited re-evaluation of the original Fontan 1 cohort utilized the following outcomes: vital status, functional health status, interim medical events, access to health care and self-reported availability and willingness to participate in future studies. We are continuing to follow this unique group of individuals in order to assess how they are coping with this chronic disease process.
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).