This study proposes to quantify and describe the quality of life of children with intestinal failure, and to identify the medical and socio-economic factors that impact this quality of life, using
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Open-label, dose escalation, multi-center, Phase I/II clinical trial to assess the safety/tolerability and determine the recommended Phase II Dose (RP2D) of ET140203 T-cells in pediatric subjects who
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Background:
During the first few decades of life, the brain changes dramatically in shape and function. Sleep lets researchers measure these changes. Researchers want to create a database of sleep
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This study primarily aims to determine whether patients diagnosed with osteochondritis dissecans (OCD) of the knee experience psychological stress due to their diagnosis and treatment plan.
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The Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP) is focused on gathering longitudinal clinical data as well as biological samples (skin and/or blood and/or
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Individuals with Sturge-Weber Syndrome (SWS) sometimes have brain involvement which can result in seizures, stroke-like episodes and neurologic deficits. The purpose of this study is to integrate
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Surgical site infections (SSIs) are one of the major hospital acquired infections and responsible for the most cost among the hospital acquired infections. The objective of this study is to assess the
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TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three arm, randomized, controlled trial of immunosuppressive therapy for children with acute liver failure. The study will
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The purpose of this study is to test whether Lomecel-B™ works in treating patients with hypoplastic left heart syndrome (HLHS) and to gather additional information about the safety of Lomecel-B.
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This research study is a genomic profiling and repository study for children and young adults who have leukemia, myelodysplastic syndrome (MDS) or myeloproliferative syndrome (MPS). Genes are the part
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This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of Cannabidiol administered as ZYN002 for the treatment of children, adolescent, and
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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
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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
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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
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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
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Creation of a pediatric robotic spine surgery registry will allow for data collection and analysis on the coupled use of robotics and navigation, as well as patient-specific rods in pediatric spine
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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
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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
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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
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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
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