The is a Phase 3 study for children, ages 0-17 years old, with severe von Willebrand disease (VWD). In this study, the study drug will be used prophylactically for the treatment of bleeding events. Prophylactic treatment means the study drug will be used to prevent or stop a bleed before it happens.
This study is going to look at how safe the study drug is and how well the study drug (recombinant von Willebrand factor (rVWF, vonicog alfa)), works to prevent and control bleeding.

This research study aims to compare usual care with a patient navigation intervention for children with sleep-disordered breathing (SDB). SDB is a range of symptoms from snoring to severe obstructive sleep apnea. In the REPOSE intervention, a centralized patient navigator will find out the dynamic individual barriers that families face in caring for their child with SDB. They will also provide resources and social support for parent-child dyads. In addition, the navigator facilitates bidirectional SDB care coordination between clinical teams and parents to achieve evidence-based care. The patient navigation intervention group will be compared to the group of children receiving standard of care.

A study in the United States that looks at the safety and effectiveness of Pradaxa Pellets in children aged 3 months to less than 12 years who need treatment of a blood clot or who have had a blood clot and are at risk of developing another blood clot. This study is for children prescribed Pradaxa oral pellet as a standard of care.
Research visits will occur when your child starts taking Pradaxa Pellets and at 6 weeks, 3 months, 6 months, and 12 months. During those visits questions about your child's medical history and current medication and health during the study will be asked.

This study is a registry for patients who have sickle cell disease (SCD), including pregnant women. A registry is a database of patient health and demographic information that is used to understand how a disease affects different people based on, for example, their other medical conditions, their age, or their treatment regimen.
This study is being done to collect information about how SCD affects you and to try to understand what characteristics of your disease may predict whether you have complications from your disease in the future.

The purpose of this research is to assess a group-based telehealth parenting program for child ages 2-6 years old with a neurodevelopmental diagnosis and behavior problems. Families will complete an online intake assessment, 6 group-based telehealth PCIT sessions, and an online post intervention assessment. Families will be compensated for their time.

Each day in the United States, approximately 41 children experience in-hospital cardiac arrest (IHCA) requiring cardiopulmonary resuscitation (CPR). A lack of situation awareness was cited as the most common contributing factor to serious safety events within children's hospitals. Situation awareness is the ability to monitor what is happening, integrate information to develop a comprehensive picture, and extrapolate forward to project the future. The primary objective of this study is to implement unit based interventions (SAMURAI PICU bundle) to reduce the incidence of IHCA in pediatric intensive care units using quality improvement methodology.

This study is for patient that have been diagnosed with High Risk Neuroblastoma. The investigational drug in this study is naxitamab. The purpose of this study is to test the safety of adding an Anti-GD2 Immunotherapy agent (naxitamab) to your standard therapy during the induction phase of care. Read the sections on risks and benefits carefully and be sure you understand them. This study will also look at the effectiveness of this treatment.

Adolescence is a time of development in the brain and microbiome, including the microbiome present in the mouth called the oral microbiome. Alcohol use often begins and escalates during adolescence, which can cause changes to the oral microbiome leading to changes in the brain. The alcohol specific changes to the oral microbiome and the brain may make adolescents more vulnerable to a lifelong struggle with alcohol use disorder. This study will collect (1) saliva to assess the composition of the oral microbiome and (2) brain measurements using magnetic resonance imaging (MRI) across adolescents who may or may not use alcohol. Findings from this study will help form a more thorough understanding of the biological consequences of adolescent alcohol use, with the long-term goal of informing novel prevention and intervention efforts.

This study will evaluate the association between feeding recovery and clinical assessment of infant behaviors as measured in a brief survey to assess irritability, ability to be soothed, alertness, non-nutritive sucking, and muscle tone. Forty infants will be recruited from the PCICU-CSD at MUSC. This data will be assessed using the Neoneur Feeding System to create a model of feeding recovery.

Hematopoietic cell transplantation (HCT) and solid organ transplantation (SOT) recipients are at high risk for respiratory viral infection (RVI). This study aims to establish a comprehensive RVI diagnostic and disease progression predictive model in children undergoing HCT and SOT. Findings will result in the first ever evidence-based pediatric guidelines.