Clinical trials

This study will clinically evaluate a newly developed respiratory monitor - pneuRIP. The pneuRIP uses Respiratory Inductance Plethysmography (RIP) bands to measure key breathing indices non-invasively. This study compares the pneuRIP to an existing Respitrace system (Carefusion, Yorba Linda CA). 10 normal children and 10 children with breathing difficulties will be monitored with both systems.

The prevalence of childhood obesity in the United States has more than tripled in the past four decades affecting one in every five adolescent girls and is disproportionally higher among racial and/or ethnic minorities. Normal puberty onset and progression is dependent on normal hypothalamic-pituitary-gonadal (HPG) axis which is affected by whole body metabolism. Gonadotropin-releasing hormone (GnRH) and gonadotropins, LH and FSH, are released in a pulsatile manner for appropriate sex steroids production and gonadal function. Proper pulsatility in the GnRH system is disrupted by a significant change in energy balance such as in obesity. Polycystic Ovary Syndrome (PCOS) is the most common neuroendocrine dysfunction in women of reproductive age. Glucagon-like peptide-1 (GL-1), a peptide hormone secreted by the intestinal enteroendocrine L-cells following glucose and fat intake, stimulates insulin release by the pancreas in response to glucose, decreases gastric emptying and inhibits glucagon secretion. GLP-1 receptors are present in the hypothalamic nuclei and pituitary gland; and it is thought that GLP-1 may directly stimulate GnRH secretion and partially regulate reproduction. In animal studies, GLP-1 was found to stimulate GnRH secretion, to regulate kisspeptin (Kiss-1) mRNA and GnRH mRAN expression. GLP-1 receptor agonists are FDA-approved to treat adults and adolescents with obesity. Although the impact of GLP-1 receptor agonists in reproductive health has been investigated in preclinical trials, and in men with obesity and functional hypogonadism, no studies to date have investigated the impact of GLP-1 receptor agonists in female neuroendocrine function, particularly in youth. The goal of this proposal is to gather critical preliminary data to investigate, in a group of obese adolescent females with PCOS, the impact of GLP-1 agonist administration in addition to lifestyle modifications on the neuroendocrine rhythms - LH frequency and amplitude (principal); body composition, adiposity; and carbohydrate metabolism and insulin sensitivity. To accomplish these aims, we will recruit a cohort of up to 20 adolescents ages 12-18 years, at least 2 years post-menarche, with obesity, PCOS, by NIH criteria, without carbohydrate intolerance and in otherwise good health. Research volunteers will be advised on lifestyle modifications of diet and exercise as per routine, and a GLP-1 agonist will be started according to the product's label as per FDA guidelines in children with obesity. Medication will be titrated to maximal therapeutic dose, as per routine clinical practice. Participants will be treated for a total of 16 weeks. Neuroendocrine rhythms pre- and post-treatment will be compared.

The Diabetes \& Equine Empowerment Program (DEEP) is a research study evaluating a novel diabetes education program that combines hands-on interactions with miniature horses and evidence-based diabetes self-management education for youth ages 8-20 years with diabetes. Participants and their caregivers attend four structured educational sessions led by a diabetes specialist and a certified equine specialist. Topics include diabetes self-management, coping skills, problem-solving, self-advocacy, and transition readiness. Researchers will evaluate whether the program is feasible to deliver, acceptable to participants and families, and helpful in supporting engagement in diabetes care. Participants will be followed for up to six months after the program, and study outcomes include satisfaction, diabetes-related distress, self-care behaviors, clinic attendance, and diabetes technology use. In simple terms, this study is exploring whether learning about diabetes through guided activities with miniature horses can help children, adolescents, and young adults feel more confident in managing their diabetes, improve self-care skills, and become more engaged in their health care. The goal is to determine whether this innovative educational approach is practical to offer and valued by patients, families, and health care providers.

Preterm infants often have difficulty learning how to feed by mouth because the coordination of sucking, swallowing, and breathing develops later in pregnancy. Delays in achieving full oral feeding can prolong hospitalization and increase the need for tube feeding. This study evaluated whether a Pacifier-Activated Lullaby (PAL) system could improve oral feeding performance in preterm infants. The PAL device plays a recorded maternal or caregiver voice when an infant generates an adequate non-nutritive suck on a pacifier, providing positive reinforcement during feeding development. In this randomized controlled trial conducted at Nemours Children's Hospital in Orlando, Florida, preterm infants who were transitioning from tube feeding to oral feeding were assigned either to receive PAL therapy in addition to standard feeding care or to receive standard feeding care alone. Researchers compared measures of oral feeding performance, feeding efficiency, growth, and progression toward independent oral feeding between the two groups. The goal of the study was to determine whether reinforcing non-nutritive sucking with auditory feedback could enhance feeding development and improve clinical outcomes in preterm infants. The information gained from this study may help clinicians better understand the role of music-assisted feeding interventions in neonatal care and identify which infants are most likely to benefit from this approach.

The purpose of this study is to identify the independent and combined effects of two types of self-monitoring and two types of micro-interventions when combined with standard cognitive behavioral treatment for bulimia nervosa (BN) and binge eating disorder (BED). The primary aims of this study are (1) to evaluate the optimal complexity of Self-Monitoring and Micro-Interventions on eating pathology (at post-treatment and at 6 and 12-month follow-ups and (2) to test the hypotheses that the optimal complexity level of each component is moderated by baseline deficits in self-regulation. The secondary aim will be to test target engagement for each level of complexity for each component, i.e., to test whether higher complexity of each technological components is associated with better rates of therapeutic skill use and acquisition and that improvements in skill use and acquisition are associated with improvements in outcomes. A final exploratory aim will be to quantify the component interaction effects, which may be partially additive (because components overlap and/or there is diminishing return), fully additive, or synergistic (in that component complexities may partially depend on each other).

This R01 is in response to RFA-DK-19-021, Treating Diabetes Distress to Improve Glycemic Outcomes in Type 1 Diabetes. The objective is to test the feasibility and acceptability of a novel, practical, and potentially scalable screen to treat program for diabetes distress in families of school-age children with T1D (called Remedy to Diabetes Distress \[R2D2\]) and to test the initial efficacy of R2D2 to reduce diabetes distress to improve children's glycemic control.

The goal of this study is to learn if toolkits of implementation strategies (e.g., provider and staff education, clear plan for how to refer and bill) help pediatric type 1 diabetes medical and psychology providers deliver a behavioral intervention (Diabetes Coaching) to more families. The main questions this study aims to answer are: * Is it possible to put these toolkits of implementation strategies into place in one pediatric hospital? * Do providers and staff find these toolkits of implementation strategies acceptable? Participants will be type 1 diabetes professionals (endocrinologists, nurse practitioners, psychologists, scheduling and billing staff) at Nemours Children's Hospital Delaware. Participants will: * Attend educational and/or planning meetings * Receive email updates on referral data * Complete surveys * Complete an interview

The goal of this registry is to collect medical information on individuals with rhizomelic chondrodysplasia punctata and closely related conditions. The study team hopes to learn more about these conditions and improve the care of people with it by establishing this registry.

The Investigators are investigating a continuous passive motion device to exercise the knee of young children with arthrogryposis after they have had knee surgery.

This study is being performed to understand the effects of a lifestyle modification and sleep intervention on weight loss outcomes in adolescence. The main question the study aims to answer is: \- What are the initial effects of a lifestyle modification and sleep intervention, including effects on weight, eating behaviors and dietary intake, and sleep? Participants will: * Participate in remote, group-based, weekly weight loss and sleep coaching for 16 weeks. * Complete study assessments at baseline (pre-intervention), mid-intervention, and post-intervention. * Complete daily sleep diaries for three 7-day periods at each assessment point.

The goal of this clinical trial is to learn about cardiovascular disease risk among adolescent girls with overweight/obesity. The main questions this trial aims to answer are: 1. Does an acceptance-based healthy lifestyle intervention demonstrate more weight loss than a health education intervention? 2. Does an acceptance-based healthy lifestyle intervention show more improvements in participants' physiological, health-related, and psychological factors compared to a health education intervention? Participants in this trial will: 1. Engage in treatment for 6-months, comprising of a mixture of in-person and remote group sessions, occurring at Drexel University or over a video-conferencing platform. 2. Complete various assessments. These include self-reported questionnaires, measures of weight, height, body composition, blood pressure, and blood lipids, and wearing of smart watches to measure sleep and physical activity. Researchers will compare the acceptance-based and health education intervention groups to see if the acceptance-based treatment will indeed be effective in improving participants' health outcomes.

50 children/adolescents (ages 6 to \<18yrs) with T1D in suboptimal control (HbA1c≥8.0%) and lower SES (below 200% poverty line) on insulin therapy (either injections or open-loop pumps) will be recruited at Nemours \~ 1/3 each AA, Hispanic/Latino, non-Hispanic whites. All families that qualify and agree to transition to closed-loop technologies will be recruited to allow data to be gathered before and after use of devices. They will go through the process of approval with the assistance of an insurance navigator in clinic. Those not a CGM will be prescribed one as well. Diabetes care will be 'real life', devices will be prescribed, and care per clinic routine with periodic device downloads. Principal outcome, time-in-range, will be analyzed at 3-months compared to baseline, each participant their own control. Secondary outcomes including HbA1c, other glucose metrics and questionnaires related to use of technology and diabetes distress will be also analyzed. All outcomes will also be collected at 6-months. Results could have important and fast applicability to the field and help better inform decision makers, including payers, clinicians, and patients and families and could serve to decrease health care disparities in this needy population.

It is critical to provide accessible evidence-based psychosocial support to parents and caregivers of children with cancer (PCCC) in order to mitigate individual and family-level psychosocial risks. This effectiveness trial evaluates an eHealth intervention for English- and Spanish-speaking (PCCC) with study endpoints focused on decreasing negative psychosocial sequelae (acute distress, posttraumatic stress, and anxiety) and improving coping abilities (coping self-efficacy, cognitive coping strategies). The long-term goal of this research program is to sustain and disseminate an effective, scalable, high-reach, and cost-effective intervention to provide crucial support to PCCC across the pediatric cancer trajectory.

The goal of this multicenter, cluster-randomized, crossover trial is to determine if umbilical cord milking compared to early cord clamping will reduce in-hospital mortality in non-vigorous preterm infants born between 30 weeks and 34 weeks of gestation.

This study is a prospective, randomized, double-blind clinical trial evaluating whether intraoperative intravenous lidocaine infusion (IVLI) reduces opioid requirements and improves postoperative pain control in children undergoing surgical fixation of upper extremity long bone fractures under general anesthesia. Ninety pediatric patients, ages 3-18 will be enrolled and randomized 1:1 to receive either IV lidocaine or placebo (normal saline) during surgery. All participants will receive standardized anesthesia care and postoperative pain management. Opioid consumption and pain scores will be measured intraoperatively and throughout the postoperative recovery, with the primary outcome focused on total opioid use 60 minutes after arrival to the post-anesthesia care unit (PACU). Secondary outcomes include opioid use at additional time points, postoperative pain scores, PACU length of stay, rescue antiemetic use and the relationship between infusion duration and outcomes. Patients will be closely monitored for signs of local anesthetic systemic toxicity and other adverse events. The goal of this study is to determine whether IV lidocaine is an effective opioid-sparing adjunct in pediatric orthopedic surgery and to support safer, multimodal analgesia strategies.

The incidence of type 1 diabetes (T1D) is increasing globally with the most substantial increases occurring in the youngest age groups. A growing number of youths with T1D must therefore transition their healthcare from pediatric to adult T1D care settings as they approach young adulthood. This healthcare transition introduces many challenges because it coincides with a developmental period that is fraught with social, financial, residential, school/work and other changes/demands. Thus, it is not surprising that young adults (YA) with T1D are at risk for suboptimal glycemic levels, the development of diabetes-related complications, and psychosocial issues such as depression, anxiety, and disordered eating. Yet, few evidence-based interventions to assist YA with T1D with this complex healthcare transition exist. In this study, we are testing the feasibility, acceptability, and initial efficacy of a novel transdisciplinary model of care (Transdisciplinary Care for Transition; TCT) in which a diabetes nurse educator, psychologist, and transition navigator (case manager) co-deliver transitional care. TCT addresses the psychological and systems barriers to transition and aims to improve outcomes via better assessment of YA needs and resources, better cross-discipline and YA-provider communication, and better collaboration with YA to resolve problems that cross pediatric and adult healthcare settings. We will recruit 80 YA with T1D during their final visit in pediatric T1D specialty care to participate in a pilot randomized controlled trial during which we will randomize YA to a standard care control group (SCC) or to receive three TCT visits during the 6 months post discharge from pediatric T1D care. In SCC, participants will transition to adult T1D care as usual per their clinic's standard transition procedures. All study procedures can be completed remotely. This includes enrollment, study evaluations, and TCT visits for those randomized to TCT. Thus, we are recruiting individuals from across the United States. Aim 1: Examine the feasibility, acceptability, and fidelity of TCT in YA with T1D. Aim 2: Examine the preliminary efficacy of TCT versus SCC on YA HbA1c, transition readiness and success, and continuity of care and explore for an effect on diabetes distress and acute care utilization.

The goal of this clinical trial is to help adolescents and young adults between the ages of 13-25 with Cystic Fibrosis (CF), medically stable, able to speak and read English, and are not experiencing a CF - related exacerbation, who are already active to remain, or gradually encourage them to increase their levels of physical activity Participants will be asked to utilize a smartphone program, called NUDGE that we have developed. NUDGE is a chatbot with evidence-based features known to help teens make progress toward health goal: * Set and review goals * Self-monitor progress * Provide feedback on goal attainment * Revise future goals

This study will evaluate the acceptability, feasibility, and preliminary efficacy of a shared decision making intervention for adolescents and young adults (AYAs) with sickle cell disease (SCD). 60 AYAs with SCD ages 15-25 and their caregivers and 8 SCD providers will participate in the pilot pragmatic trial. AYAs, caregivers, and providers will be recruited from Nemours Children's Hospital, Delaware (NCH-DE), Nemours Children's Hospital in Orlando, FL (NCH-ORL), and Nemours Children's Health at Wolfson Children's Hospital in Jacksonville, FL (NCH-JAX). NCH-DE participants (n=30) will receive the SDM intervention including a virtual reality patient health education component, whereas NCH-ORL and NCH-JAX participants (n=30) will receive the SDM intervention with standard patient education materials (print, video). SCD providers will be trained to use the toolkit components and will introduce decision aids during an outpatient clinic visit for AYAs who are candidates for one or more disease-modifying therapies.

Large for Gestational Age (LGA) infants have excess fat-mass (FM) proportion secondary to prolonged in utero exposure to an energy-rich environment. Our preliminary data suggest that excess FM proportion can be associated with oral feeding delay and a potentially modifiable therapeutic target to improve oral feeding outcomes. The objective of this study is to determine the impact of a short-term Fat-free mass (FFM)-indexed feeding on the oral intake volumes in LGA infants with oral feeding difficulties.

Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment that can be applicable to all ages of patients to better understand the progress of their disease and the most serious clinical problems (cervical instability and stenosis, tracheal obstruction, hyperlaxity of joints, hip dysplasia, and small lung capacity). The outcome of this project will lead to a more precise understanding of the skeletal/pulmonary compromise and defining clinical endpoints in this disease for future clinical trials of current or developing therapies.

Bronchiolitis is the leading cause of pediatric morbidity and healthcare costs. Despite the commonplace use of bronchodilator treatments, like albuterol, in conditions like asthma, their efficacy in bronchiolitis remains controversial due to the heterogeneity in patient response. Although studies indicate that bronchodilators do not enhance outcomes in bronchiolitis, meta-analyses can obscure the heterogeneity of treatment effects. While bronchodilator response genetics have not been explored in bronchiolitis, treatment effectiveness variations often depend on genomic factors. Genome-wide association studies (GWAS) have linked genetic variants with bronchodilator response and outcomes in childhood asthma, suggesting a bronchodilator-responsive genotype. This proposal aims to extend this paradigm to bronchiolitis, addressing the gap in knowledge where GWAS and clinical characteristics intersect. The proposed study's objective is to characterize phenotypic and genotypic variations of children with bronchiolitis and their association with bronchodilator response. We hypothesize that children with bronchiolitis who exhibit clinical and historical characteristics associated with atopy and specific physical findings have genetic variants linked to bronchodilator response. To achieve this, we propose to (Aim 1) define airway responsiveness to bronchodilator treatment in children with bronchiolitis using the change in respiratory score, (Aim 2a) identify the associations between candidate genetic variants and bronchodilator response among children with bronchiolitis, and (Aim 2b) determine the associations between candidate genetic variants and clinical patient data to identify bronchodilator-responsive children with bronchiolitis. A prospective, double-blind, randomized, placebo-controlled trial of a single albuterol dose in children aged 3 to 24 months presenting with bronchiolitis to the emergency department will be conducted to achieve these aims. Patient information and respiratory assessment outcomes will be collected before and after intervention. Blood, urine, DNA buccal swabs, and nasopharyngeal swabs will also be collected. Completion of these aims will result in a novel clinical prediction model for bronchodilator response determination in bronchiolitis, integrating clinical, physical, and genetic data. Furthermore, this research supports the candidates' career development goals of advancing training in clinical trial research design and execution and becoming an expert in clinical and translational methods to enhance pediatric emergency department health and outcomes. Ultimately, this work will inform an R01 application to validate an evidence-based prediction rule for identifying bronchodilator-responsive children with bronchiolitis through a multi-center emergency medicine research network, optimizing therapeutic approaches, and reducing resource use in those with a low likelihood of bronchodilator response.

The goal of this prospective, observational study is to evaluate for the presence of vocal fold motion impairment (VFMI) in the children admitted to the pediatric intensive care unit on noninvasive positive pressure ventilation (NIV PPV). Participants will have two ultrasounds of their vocal folds performed, once while on NIV PPV and once after weaned off of the NIV PPV. This results of these scans will be reviewed against one another and against the gold standard, fiberoptic nasolaryngoscopy (FNL). The main question this study aims to answer is: Can POCUS be used to reliably detect VFMI while pediatric patients on supported with NIV PPV?

The goal of this intervention trial is to learn if a ultrasound diagnosis of elbow fractures can improve care in pediatric patients seen in the emergency room. The main questions it aims to answer are: The primary outcomes measured are cost of the emergency visit, radiation exposure, time spent in the emergency department and patient satisfaction. Patients who are evaluated with ultrasounds of the elbow will be compared to patients who have x-rays of the elbow. Participants will be assigned either to the ultrasound group or x-rays group when they present with elbow pain.

This study aims to improve surgical outcomes for children and young adults with ear differences severe secondary to congenital conditions (like microtia, a condition where the ear is underdeveloped), trauma (such as dog bites), or surgical resections secondary to skin cancer. The investigators have developed a novel medical device to shape cartilage into pre-determined shapes that, once assembled, assists the surgeon in creating an anatomically accurate cartilaginous ear framework, reduce surgery time, and minimize wire need. Other factors that might affect the quality of surgical outcomes will be examined in clinical trial participants.

Pilot randomized trial assessing the effectiveness of laser hair depilation on pilonidal disease recurrence in patients with darker skin color