The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem ...
Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less ...
The investigators propose a robust therapeutic intervention to ameliorate myocardial ischemia/ reperfusion injury and significantly decrease morbidity and mortality in patients requiring ...
The purpose of this initiative is to improve care and outcomes for infants with HLHS by expanding the NPC-QIC national registry to gather clinical care process, outcome, and developmental data on ...
To Assess the Activity and Safety of SMT C1100 (Ezutromid) in Paediatric Male Participants with Duchenne Muscular Dystrophy (DMD).
The main objective of this proposed investigation is to evaluate blood ropivacaine concentrations in infants and toddlers following the initiation of, and over the course of, continuous paravertebral ...
This is a single site, non-randomized, prospective, open-label, interventional pilot/feasibility study. Patients recruited will have medically-refractory focal neocortical epilepsy, defined on the ...
This proposal sets forth the platform for a Precision Medicine clinical trial through the New Approaches to Neuroblastoma Therapy (NANT) consortium. The plan is to utilize NANT's established multi ...
Miscommunications are a leading cause of serious medical errors in hospitals, contributing to more than 60% of sentinel events, the most serious adverse events reported to the Joint Commission. ...
In this research study, the investigators want to learn more about whether the Respiratory Volume Monitor (ExSpiron) can accurately measure breathing in infants less than 12 months of age. The ...