Bronchiolitis is broadly defined as a clinical syndrome that occurs in children < 2 years of age and is characterized by upper respiratory symptoms followed by lower respiratory infection with inflammation, which results in wheezing and or crackles (rales). Bronchiolitis typically is caused by viruses. In ICU setting, the standard therapy of patient presents with acute bronchiolitis are bronchodilation medications. Recent recommendations of American Academy of Pediatrics stated that no evidence to support usage of any of bronchodilators,The goal of this study is validate the effectiveness of bronchodilators medications.
This study will evaluate the efficacy and safety of 2 dose levels of NTRA-2112 on intestinal malabsorption in preterm infants as compared to placebo. NTRA-2112 is a powder of insulin formulation for reconstitution in breast milk, infant formula, normal and half-normal saline administered with the preterm infant's formula, donor breast milk, or own mother's breast milk for local gastrointestinal (GI) therapy. The effect on intestinal malabsorption will be evaluated by comparing the ability of preterm infants to achieve full enteral (EN) feeding for 3 consecutive days.
The study will enroll preterm infants weighing at least 500g born between 26 and 32 weeks of pregnancy who meet inclusion and exclusion criteria. Infants will be treated for 28 days or until discharged from the hospital, whichever happens first. Patients will be randomly-assigned to one of three treatment arms: Treatment A 400 ?U/ml ? equivalent dose of insulin or NTRA-2112; Treatment B to
obtain 2000 ?U/ml dose of insulin; or placebo as calculated according to planned daily enteral intake. During the treatment period, infants will undergo daily evaluation of nutrition, general growth, gastric residuals, development progression, and any side effects.
Patients will be evaluated at day of discharge along with follow-up clinic visits performed at 3 months, 12 months, and 24 months corrected age.
This is a Randomized , Double blinded study to evaluate how effective a single dose of experimental drug called MEDI8897 is at preventing lung disease caused by RSV disease in healthy preterm infants born between 29 weeks 0 days and 34 weeks 6 days.This study also evaluate safety, tolerability and pharmacokinetics (PK) of MEDI8897 in healthy preterm infants compared with placebo.
This study tests an intervention to help children with sickle cell disease ages 0-7 years and their families. The intervention is available on a smartphone or tablet, and includes an app for keeping track of symptoms and messaging with a health care provider. For more information, please contact Shannon at 843-792-9379.
This study is a longitudinal, observational follow-up of MUSC subjects previously enrolled in the NIH funded Tolsurf study. It is being conducted to determine the developmental impact of NICU exposures to chemical plasticizers called phthalates that are found in the NICU environment. Subjects who participated in the Tolsurf study are part of a larger group of NIH funded studies. Participants in each of these NIH funded NICU studies have urine samples from their NICU stay stored in a biobank. These urine samples will be tested for phthalates. If the families agree to let their children participate in the study, results of pulmonary testing and developmental testing will be compared to levels of phthalates in the urine collected during their NICU stay. Previous studies have linked phthalates in-utero to developmental delays, asthma, and altered onset of puberty. The aims of the DINE study are to identify the sources of phthalates in the NICU and measure the impact of phthalates in the NICU.
The purpose of this research protocol is to collect information about individuals living with sickle cell disease to help improve the care of those patients. We hope to understand more about the disease itself, the best ways to treat the disease, and the best ways to help patients with sickle cell disease get care. The goal is to have clinical information about every person diagnosed with sickle cell disease in South Carolina so that we can better treat the disease and help develop new ways to improve outcomes.
Transforming health care and outcomes for children with rare diseases is difficult within the current health care system. There is great variation in care delivery, inadequate and slow application of existing evidence, and ineffective use of available data to generate new knowledge. Individual care centers have inadequate numbers of patients for robust learning and improvement. In order to redesign the system, changes must take place at multiple levels, including the patient and family, clinician, practice and the network. The purpose of this project is to design, develop, and test further refinements to an improvement and research network focused on HLHS, the most severe congenital heart defect, and to use a registry to simultaneously improve clinical care, redesign care delivery systems and to conduct quality improvement, health services, outcomes, and comparative effectiveness research. The purpose of this initiative, specifically, is to improve care and outcomes for infants with HLHS by: 1) expanding the established NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, 2) improving implementation of consensus standards, tested by teams, into everyday practice across pediatric cardiology centers, and 3) engaging parents as partners in improving care and outcomes. We utilize a quality improvement methodology, known as the adapted learning collaborative model, which expedites the implementation of tools and strategies that facilitate changes such as systematic care coordination, cardiovascular monitoring, and nutritional monitoring into every day practice. The NPC-QIC registry is used to document the impact of these changes on various care processes and outcomes (e.g., mortality rate, readmissions, and weight gain).
The Sponsor is conducting a clinical study with STP206 for the prevention of Necrotizing Enterocolitis (NEC) in preterm infants weighing less than 4.4 lbs. (2000g). NEC is the most common serious disease of the gut in preterm infants. The purpose of this study is to look at the safety of STP206 in babies born early, and to get early information on whether STP206 may prevent NEC.
STP206 is an investigational product, meaning it is not yet approved by the US Food and Drug Administration for use in the United States. STP206 contains live bacteria and is considered a Live Biotherapeutic. The two types of bacteria are Lactobacillus and Bifidobacteria. These bacteria are used in foods such as cheese, yogurt, sauerkraut, and pickles, and have been consumed safely for years and are commonly contained in probiotic products that are currently sold throughout the world. These bacteria also are normally present in parts of the body such as bowel, mouth, skin, and the vagina. These bacteria generally do not cause disease. There have been studies of probiotic bacteria in premature babies that suggest these types of bacteria are effective in preventing NEC. The purpose of this study is to determine the safety of STP206 and if it is effective in preventing NEC.
Infants born early who are in the neonatal intensive care unit will be included if they meet national guidelines for retinopathy of prematurity (ROP) screening exams. Informed consent will be given to the parent(s) or legal guardians. 1.5-2 ml of blood will be drawn from a vein when the child is enrolled in the study and may be drawn again if the child requires treatment of eye disease. A cheek swab will also be obtained. These biologic samples will be shipped overnight to the University of Utah for genetic analysis. Analysis will determine if a change in gene expression causes retinopathy of prematurity. Infants enrolled in the study will be followed clinically per established ROP screening guidelines. They will not require additional study exams.