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Clinical Trials

Characterization of Angelman Syndrome

Angelman Syndrome (AS) is a developmental disorder that is caused by a deficiency of a maternally transmitted gene. It is inherited at birth, and affects movement, speech, and social demeanor. This...

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Transfer Impedance in Cystic Fibrosis

This study aims to determine whether respiratory system transfer impedance (Ztr) may fill an important clinical function by providing a reproducible, valid, and sensitive measure of airway obstruction...

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Natural History Study of SCID Disorders

This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine...

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Patients Treated for SCID (1968-Present)

Individuals with a past diagnosis of severe combined immune deficiency (including many cases of "leaky SCID", Omenn syndrome, and reticular dysgenesis) who have undergone blood and marrow transplant,...

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SVRII Family Factors Study

The purpose of this study is to learn about the relationship between family factors and developmental and psychosocial outcomes in children with congenital heart disease at 6 years of age. A secondary...

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Neuroblastoma Biology Study

Medical scientists want to find better ways to treat neuroblastoma and to find ways to prevent the tumor from growing back. To do this, they need more information about the characteristics of...

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Genetics of Severe Early Onset Epilepsies

Investigators at Boston Children's Hospital are conducting research in order to better understand the genetic factors which may contribute to disorders related to epilepsy. These findings may help...

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Orchidopexy Randomized Clinical Assessment

This is a randomized trial designed to evaluate testicular outcomes after a single stage versus a two-stage surgical procedure to fix an undescended, one-sided testicle located in the abdomen. Also to...

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MRI Assessment of Placental Health

The ultimate goal of this project is to develop methods that allow informed decision-making on the delivery time of fetuses that are at increased risk of stillbirth due to IUGR. In placenta related...

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ATHN 2: Factor Switching Study

This is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched...

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Preventing Asthma in High Risk Kids

This trial is a randomized, double-blind, placebo controlled trial designed to test whether two years treatment of preschool children aged 2-3 years of age at high risk for asthma with omalizumab ...

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NIRS Monitoring in Premature Infants

This study uses frequency domain near-infrared spectroscopy coupled with diffuse correlation spectroscopy (FDNIRS-DCS) technology for monitoring cerebral blood flow (CBF) and cerebral oxygen...

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Gene Discovery Core, The Manton Center

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...

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Home Away From Home - Medical Outcomes

Treatment for pediatric acute myeloid leukemia (AML) involves intensive chemotherapy regimens that result in periods of profound neutropenia leaving patients susceptible to severe infectious...

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Advanced Fetal Imaging - Phase II

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....

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Neuroblastoma Precision Trial

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...

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Family Engagement in Safety Reporting

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....

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Pumps for Kids, Infants, and Neonates

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...

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Aerobic Exercise for Concussion

The use of aerobic exercise treatment in the chronic phase of concussion recovery is well-described in the literature but there are limited existing data on the effect of exercise treatment in the...

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Validating Adolescent SBIRT Measures

The purpose of this study is to address the needs for efficient and informative measurement for evaluating adolescent Screening, Brief Intervention, and Referral to Treatment (SBIRT) strategies. The...

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PALISADE Follow-on Study (ARC004)

The purpose of this study is to demonstrate the safety, tolerability, and efficacy of AR101 through oral immunotherapy (OIT) in peanut-allergic children and adults who have completed the ARC003 study.

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A Gene Transfer Study for Hemophilia A

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...

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Pediatric Femur Fracture Registry

Prospective data collection and evaluation of complete data sets will be performed in the course of routine clinical care of a cohort of consecutive patients (children up to 16 years old) presenting...

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Gene Transfer for Sickle Cell Disease

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat...

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MIBG With Dinutuximab +/- Vorinostat

131I-Metaiodobenzylguanidine (131I-MIBG) is one of the most effective therapies utilized for neuroblastoma patients with refractory or relapsed disease. In this pediatric phase 1 trial, 131I-MIBG will...

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