The purpose of this study is to assess the safety, tolerability and efficacy of emapalumab in children and adults with macrophage activation syndrome (sHLH/MAS) in Still's disease (including systemic...
This is a Phase 1b/2, randomized, double-blind, multi-center study to evaluate the safety, tolerability, and preliminary clinical efficacy of STMC-103H in neonates and infants at risk for developing...
Prospective, single-arm, multi-center study to evaluate safety and effectiveness of the Autus Size-Adjustable Valve in pediatric patients aged 18 months to 16 years requiring surgical pulmonary valve...
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in...
Current methods of measuring the response to new treatments for muscular dystrophies involve the examination of small pieces of muscle tissue called biopsies. The investigators are interested in...
A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.
The BEAM study is a multicenter, prospective, observational study in children supported on extracorporeal membrane oxygenation (ECMO). The primary goals of this study are to develop and refine a brain...
Cervical spine injuries (CSI) are serious, but rare events in children. Spinal precautions (rigid cervical collar and immobilization on a longboard) in the prehospital setting may be beneficial for...
Creation of a pediatric robotic spine surgery registry will allow for data collection and analysis on the coupled use of robotics and navigation in pediatric spine deformity surgery across...
This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants.
This...
The primary objectives of the study are to identify a setrusumab dosing strategy in participants with OI and to evaluate the effect of setrusumab vs placebo on reduction in fracture rate.
The purpose of this study is to identify the optimal deimplementation strategies for an overused practice: continuous pulse oximetry monitoring of children hospitalized with bronchiolitis who are not...
Randomized Controlled Trial comparing a coach-enhanced digital cognitive behavioral intervention (d-CBI) (RxWell) + treatment as usual (TAU) versus TAU alone for moderate depression as determined by...
This research study is exploring the use of genomic sequencing in the newborn period to screen healthy babies for current and future health risks. The study will enroll a diverse cohort of 500 healthy...
The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein...
Individuals with repaired Tetralogy of Fallot (rTOF) remain at risk for sudden cardiac death from ventricular tachycardia (VT). Transcatheter pulmonary valve replacement (TPVR) indications continue to...
In this research study the investigators want to learn more about how well the investigators can visualize scar tissue in the heart by MRI. In patients with congenital heart disease who need a...
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC...
COMPASS is a prospective multicenter randomized interventional trial. Participants with ductal-dependent pulmonary blood flow will be randomized to receive either a systemic-to-pulmonary artery shunt...
