The Gene Discovery Core at The Manton Center for Orphan Disease Research based at Boston Children's Hospital studies families with rare, poorly understood or undiagnosed, but suspected genetic...
Pediatric ACL: Understanding Treatment Outcomes (PLUTO) is a multi-center, prospective cohort study. Specific aims of PLUTO are to evaluate the safety and comparative effectiveness of non-operative...
The purpose of this study is to improve the ability of the investigators to monitor brain health in newborn babies at risk of brain injuries. The researchers will be using an investigational system of...
Parents of infants who have been thrombocytopenic for 3-4 days will be approached for consent to enter the study. For the purposes of the study, thrombocytopenia will be defined as a platelet count ...
The goal of Advanced Fetal Imaging - Phase II is to advance fetal MRI imaging by designing MRI coils specifically for pregnant women and testing recently developed MRI image acquisition techniques....
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...
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 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...
This is a prospective, multicenter, open-label, randomized, controlled, parallel Phase 3 study with an open-label single-arm extension period to evaluate pharmacokinetics (PK), safety and efficacy of...
In proximal urea cycle disorders (UCD), particularly ornithine transcarbamylase deficiency (OTCD), hyperammonemia (HA) causes increased brain glutamine (Gln) which perturbation is thought to be at the...
This will be a non-randomized, open-label, Phase 1/2 study of the safety and efficacy of AGTC-402, administered to one eye by subretinal injection in individuals with achromatopsia caused by mutations...
The primary aim of this study is to compare the propofol requirements of children who receive propofol with that of children who receive dexmedetomidine prior to propofol, for sedation for upper and...
PumpKIN is a multicenter, prospective, single-arm feasibility study; Evaluating the investigational Jarvik 2015 VAD in pediatric patients with heart failure. This feasibility trial will enroll 10...
This trial of pitavastatin will determine efficacy and safety in this high risk population and provide evidence for clinicians to target this treatable risk factor to achieve an impact on early...
The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia...
Post-traumatic headaches (PTH) are the most common complaint after traumatic brain injury, possibly generated by a number of stressors to the trigeminovascular and cervical plexus networks, including...
