Clinical trials

Children with medical complexity often receive a recommendation to see an outpatient clinician soon after leaving the hospital. It is uncertain whether every child needs this routine visit or whether follow-up can be guided by the family's concerns and the child's recovery. AFTERCARE is a multicenter pilot randomized trial comparing these two follow-up recommendations for children hospitalized with an acute infection. Families will be assigned by chance to receive either a recommendation to arrange follow-up with their usual outpatient clinician within seven days or a recommendation to seek follow-up if the child is not improving as expected or new concerns arise. Families in both groups may seek medical care whenever needed. The study will assess recruitment, follow-up data collection, delivery of the recommendations and acceptability. Caregiver surveys and medical record review will also describe outpatient visits and hospital returns. This pilot will inform the design of a larger trial, and is not designed to establish that the strategies are equally safe or effective.

The goal of this study is to conduct a clinical trial to understand effects of providing multilevel intervention to young people in the legal system. The multilevel intervention includes flexible care management using ACRA/ACC principles, family support, and overdose education. This study aims to test the implementation strategy, as well as the outcomes of the intervention with participants. The first question the study will address is how successful was implementation. This will be measured by the number of youth receiving case management, family support, naloxone supplies, and overdose prevention curricula, and adoption by the participating county court systems. The second question the study will address is whether the intervention impacted youth behavioral outcomes, such as overdose risk awareness, knowledge of how to use naloxone, and number of days per month using substances. Researchers will compare youth who received the intervention to youth who received usual treatment to see if the intervention increases overdose risk awareness and knowledge, and decreases frequency of substance use. Participants will receive the intervention (if assigned to) and complete study surveys. Participating counties will provide data on implementation, and site visits will also be performed to track each county's performance.

Although automatic follow-up is a nearly universal practice, research has shown that these visits are often unnecessary after hospitalizations caused by bronchiolitis. Despite endorsement by national pediatric authorities, robust evidence, and family enthusiasm for as-needed (PRN) follow-up, it remains substantially underutilized for children hospitalized for bronchiolitis. The goal of I-DECIDE is to compare the effects of two multi-component implementation strategies, both of which aim to (a) increase PRN follow-up prescribing by hospitalists (physicians who care for hospitalized children) and (b) decrease unnecessary follow-up visit attendance by families.

Brief Summary The aim of the FIBER study is to identify potential triggers associated with the later development of IBD (inflammatory bowel disease) based on baseline characteristics and biosamples (blood, urine, stool, saliva, and tissue). Aim 1: To identify clinical, demographic, and immunologic factors associated with an increased risk of developing IBD in family members of IBD patients. This aim will investigate the role of clinical factors (e.g., age of onset, gender, and family history), demographic factors (e.g., socioeconomic status, geographical location), and immune system markers (e.g., inflammatory cytokine profiles, immune cell populations) in predicting the likelihood of family members developing IBD over time. Aim 2: To examine dietary, environmental, and immunologic influences on the development of IBD in family members of IBD patients. This aim will explore how dietary habits (e.g., fiber, fats, processed foods), environmental exposures (e.g., smoking, pollution, and antibiotic use), and immune responses (e.g., changes in T-cell activation, inflammatory markers) contribute to the risk of IBD in family members. Aim 3: To analyze the multi-omic (metagenomic, transcriptomic, proteomic) cellular signatures of host and microbial signatures in family members of IBD patients and their association with IBD onset. This aim will investigate changes in the gut microbiome and immune-related gene expression profiles (transcriptomics) in family members. Specifically, the aim will focus on how shifts in microbial composition and host immune response genes (e.g., those involved in inflammation and epithelial barrier function) correlate with an increased risk of IBD development. Aim 4: To investigate the multi-omic (metabolomic, transcriptomic, proteomic) cellular signature of host and microbial signatures in family members to identify biomarkers predictive of IBD development. This aim will involve examining the metabolic, protein, and transcriptomic signatures (e.g., circulating cytokines, immune receptor expression) in blood, urine, or stool samples. The study will seek to identify early biomarkers from these profiles that can predict the onset of IBD, even in asymptomatic family members.

Children with medical complexity (CMC) live full meaningful lives that are often short. CMC and their families experience substantial distress, poor mental health, and low emotional well-being. Existing research and our prior studies suggest that legacy, defined as the summation of qualities and experiences that form the child's enduring presence, can be a resource for actionable appraisal of and coping with stressful life experiences. Photo-narratives are a novel intervention that facilitate family sharing of images to tell stories about everyday experiences related to their child's quality-of-life and personhood. In our prior studies, families reported that photo-narratives were a simple, easy way to meaningfully connect and had the potential to be an important part of their child's legacy. What remains poorly understood, however, is how photo-narratives might impact family-centered outcomes. This study aims to and evaluate the feasibility, acceptability, and potential impact of photo-narratives in a pilot randomized controlled trial.

The goal of this pilot trial is to examine whether a mobile app version of the Promoting Resilience in Stress Management (PRISM) intervention is acceptable, easy to use, and helps improve quality of life and mental health symptoms.

Severe and disabling abdominal pain is common in individuals with chronic pancreatitis. Although pain is associated with reduced quality of life and high economic and societal costs, there are limited effective options for pain management in this population. This study proposes an evidence-based psychological intervention approach using an internet-delivered pain self-management program to minimize the impact of pain and improve quality of life. The ultimate goal is to maximize the public health impact of the intervention with successful implementation and dissemination to pancreas clinics and to the community.

Subjects exposed to gene therapy products (GTPs) may be at risk for delayed or long-term adverse events. This is a long-term follow-up (LTFU) protocol designed to evaluate the long-term safety of Seattle Children's Therapeutics (SCTx) GTPs and incorporates in monitoring guidance from the U.S. Food and Drug Administration (FDA). Subjects will be followed for up to 15 years starting from the date of the most recent infusion of an SCTx GTP. Subjects planning to receive, or who have received, at least one infusion of an SCTx GTP will be offered participation in this LTFU study. Subjects enrolled in this study will have safety assessments and laboratory evaluations performed at scheduled intervals for each unique SCTx GTP received. No treatment is administered in this LTFU study.

Cystic Fibrosis (CF) is a devastating chronic pulmonary disease that continues to cause significant morbidity and mortality despite great advances in therapies. Hispanic children with CF have worse outcomes, including higher mortality and more severe pulmonary disease, than non-Hispanic white children with CF. It is not known why Hispanic children with CF have more severe disease as it is not explained by CFTR genetic severity, diagnosis age, or socioeconomic status. The health disparities have worsened, not improved, for Hispanic children with CF since the development of new disease-altering therapeutics, CFTR modulators. It is critical to determine what is contributing to lung disease severity in Hispanic children with CF. Non-genetic factors, including environmental exposures, are estimated to account for 50% of lung disease severity variability in CF. Air pollution exposure during early childhood is associated with lower pulmonary function in healthy children and severe lung disease in children with asthma. However, air pollution exposure is vastly understudied in other chronic pulmonary diseases of childhood, such as CF. Investigating air pollution exposure in CF may provide vital information about the drivers of health disparities in Hispanic children with CF and about the environmental exposures influencing lung disease severity across all children with CF. To investigate air pollution exposure in children with CF, the investigators have assembled a multidisciplinary team of international experts in air pollution exposure, CF lung disease, health disparities, and pulmonary microbiome. The investigators will use two phenomenally rich databases, the CF Foundation Patient Registry and the University of Washington Spatiotemporal Air Pollution Exposure Model, to investigate the first aim: 1A) To determine whether neighborhood-level ambient air pollution exposure during childhood differs between 1500 Hispanic and 8500 non-Hispanic white cwCF in the CF Foundation Patient Registry, and 1B) To determine if neighborhood-level ambient air pollution exposure is associated with lung disease severity in Hispanic and non-Hispanic white cwCF. Across six geographically diverse clinical research CF centers, the investigators will enroll 100 Hispanic and 100 non-Hispanic children with CF to investigate the following aims: 2) To assess differences in residential indoor and ambient air pollution exposures by ethnicity in 200 cwCF, as well as the association between such exposure and pulmonary function by ethnicity, 3) To investigate the association of indoor and ambient air pollution exposure on airway inflammation and microbiome diversity and composition in Hispanic and non-Hispanic white cwCF using metatranscriptomic RNA sequencing. The HEROIC-CF Study is poised to advance the knowledge of the effect of air pollution exposure on not only CF lung disease severity, but may be a model to understand environmental exposures on disease severity in other chronic pulmonary diseases of childhood.

The goal of this clinical trial is to definitively determine whether copper intrauterine device (IUD) or hormonal intrauterine system (IUS) results in greater vaginal microbial diversity after 1 year in women (n= approximately 120) aged 18-40 years, who desire to use a copper IUD or hormonal IUS as contraception, are HIV-negative and could benefit from STI prevention. The main questions it aims to answer are: 1. Whether women assigned to copper IUD vs hormonal IUS have differences in vaginal microbial diversity after 1 year of use 2. Whether women randomized to Copper IUD have reduced genital mucosal barrier integrity as indicated by proteomic signatures 3. Whether women randomized to Copper IUD have greater incidence of high-risk HPV or curable STIs (Ct, Ng, Tv) Participants will be assigned to have either the copper IUD or the hormonal IUS inserted as contraception. After that, they will have blood and vaginal fluids collected every 3 months for one year to look at the bacteria in their vagina, test for sexually transmitted infections, and examine markers of vaginal health. After enrolment, each participant will be followed for 12 months, and at the end of the trial, the participant can continue to use the IUD/IUS or the study clinician can remove it.

Parent-focused Redesign for Encounters, Newborns to Toddlers (PARENT) is a team-based approach to care that utilizes a community health worker in a health educator role ("Parent's Coach") to provide many of the Well-Child Care (WCC) services that children and families should receive, addresses specific needs faced by families in low-income communities, and decreases reliance on the clinician as the primary provider of WCC services. The model was developed in partnership with clinics and parents in low-income communities and previously tested among largely Latino, Medicaid-insured populations. The aims of this study are to (1) Adapt the PARENT intervention to meet the needs of a diverse, largely Black population of underserved families, (2) Determine the effect of adapted PARENT on receipt of nationally recommended preventive care services, emergency department utilization, and parent experiences of care, (3) Determine whether the effectiveness of adapted PARENT differs by family-level factors, (4) Explore parents' experiences in receiving adapted PARENT, (5) Examine the economic impact of adapted PARENT from the parent stakeholder perspective, (6) Examine the economic impact of adapted PARENT from the pediatric provider and clinic stakeholder perspective, and (7) Examine the economic impact of adapted PARENT on healthcare utilization, from the perspectives of parents and families. This study will evaluate the effectiveness of the adapted PARENT model as compared to traditional guideline-based WCC and assess the patient-centered economic outcomes of the adapted PARENT model.

This study will examine a novel stakeholder-informed intervention to identify vaccine-eligible children and promote evidence-based clinician vaccine communication with families with the goal of increasing vaccine uptake during hospitalization.

This study explores the use of an exclusive human milk diet versus standard feeding practices to compare the influence on feeding outcomes and the gut bacteria in infants with intestinal differences.

Investigators will conduct a pilot randomized controlled trial assessing the efficacy and feasibility of the newly developed Family Media Check-In (FMC).

This is a prospective observational study collecting long-term clinical data and samples for research in pediatric inflammatory bowel disease (IBD) patients with gut inflammation and a control cohort of pediatric patients with disorders of the brain-gut interactions (DBGI) with no detectable gut inflammation.

Investigators will conduct a randomized controlled trial assessing effectiveness of the Family Safety Check-In website on firearm storage practices. Prior to conducting the RCT, investigators will engage in a robust, participatory process of language adaptation to ensure the intervention meets the expressed needs of the large and growing proportion of parents in the United States who identify as Hispanic and speak primarily Spanish.

This is a Phase 1 study of central nervous system (CNS) locoregional adoptive therapy with autologous CD4+ and CD8+ T cells lentivirally transduced to express a B7H3-specific chimeric antigen receptor (CAR) and EGFRt. CAR T cells are delivered via an indwelling catheter into the tumor resection cavity or ventricular system in children and young adults with diffuse intrinsic pontine glioma (DIPG), diffuse midline glioma (DMG), and recurrent or refractory CNS tumors. A child or young adult meeting all eligibility criteria, including having a CNS catheter placed into the tumor resection cavity or into their ventricular system, and meeting none of the exclusion criteria, will have their T cells collected. The T cells will then be bioengineered into a second-generation CAR T cell that targets B7H3-expressing tumor cells. Patients will be assigned to one of 3 treatment arms based on location or type of their tumor. Patients with supratentorial tumors will be assigned to Arm A, and will receive their treatment into the tumor cavity. Patients with either infratentorial or metastatic/leptomeningeal tumors will be assigned to Arm B, and will have their treatment delivered into the ventricular system. The first 3 patients enrolled onto the study must be at least 15 years of age and assigned to Arm A or Arm B. Patients with DIPG will be assigned to Arm C and have their treatment delivered into the ventricular system. The patient's newly engineered T cells will be administered via the indwelling catheter for two courses. In the first course patients in Arms A and B will receive a weekly dose of CAR T cells for three weeks, followed by a week off, an examination period, and then another course of weekly doses for three weeks. Patients in Arm C will receive a dose of CAR T cells every other week for 3 weeks, followed by a week off, an examination period, and then dosing every other week for 3 weeks. Following the two courses, patients in all Arms will undergo a series of studies including MRI to evaluate the effect of the CAR T cells and may have the opportunity to continue receiving additional courses of CAR T cells if the patient has not had adverse effects and if more of their T cells are available. The hypothesis is that an adequate amount of B7H3-specific CAR T cells can be manufactured to complete two courses of treatment with 3 or 2 doses given on a weekly schedule followed by one week off in each course. The other hypothesis is that B7H3-specific CAR T cells can safely be administered through an indwelling CNS catheter or delivered directly into the brain via indwelling catheter to allow the T cells to directly interact with the tumor cells for each patient enrolled on the study. Secondary aims of the study will include evaluating CAR T cell distribution with the cerebrospinal fluid (CSF), the extent to which CAR T cells egress or traffic into the peripheral circulation or blood stream, and, if tissues samples from multiple timepoints are available, also evaluate disease response to B7-H3 CAR T cell locoregional therapy.

Investigators will conduct a randomized controlled trial assessing the effectiveness and implementations of Pre-Game Safety Huddles (Huddles) in youth soccer.

This Phase 1, open-label, non-randomized study will enroll pediatric and young adult subjects with relapsed or refractory non-central nervous system (CNS) malignant solid tumors expressing glypican-3 (GPC3) to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to co-express a GPC3-specific chimeric antigen receptor (CAR), interleukin (IL)-15 and IL-21 as well as the inducible caspase 9 (iC9) suicide gene (SC-CAR.GPC3xIL15.21 T cells). A child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have a blood sample collected, which will be used to bioengineer the CAR T cells targeting their tumor.

The objective of this study is to conduct a pilot randomized controlled trial (RCT) of a photo-narrative communication intervention developed by our study team with patients/parents of children with severe neurological impairment (SNI) and their pediatric intensive care unit (PICU) clinicians to assess feasibility, acceptability, and early efficacy.

The goal of this clinical trial is to test efficacy of the REACH program in parents with irritable bowel syndrome (IBS) and their young children. The main question it aims to answer is: -How can parents with IBS help their young kids develop healthy habits? Participants will be asked to complete online surveys and to use a website. Researchers will compare results from parents who use one of two websites chosen by chance, like flipping a coin. One website focuses on child health and safety behaviors. The other website focuses on strategies to promote child wellness behaviors.

The purpose of this trial is to study if priming the pump used during cardiac surgery with non-blood fluids instead of donated blood products reduces the inflammation that occurs after heart surgery. The study will focused on pediatric participants who require open heart surgery to repair certain types holes in the heart. Typically for pediatric patients, the cardiopulmonary bypass pump is "primed" (filled) with donated blood products. This project is going to test if the exposure to these blood products causes inflammation. Patients experience significant inflammation (swelling) after undergoing cardiopulmonary bypass. This inflammation can interfere and slow down the patient's recovery from cardiac surgery. With this project, the investigator are studying if filling the bypass pump with non-blood products reduces the bypass-associated inflammation. The investigators are also studying if using non-blood fluids to fill the bypass pump reduces bypass associated side effects. The investigators are also trying to understand how the inflammation starts. The investigators also want to study genetic material called DNA that is collected from a person's blood. Instructions for the body are contained in parts of DNA called genes. Genes determine things like hair and eye color. The investigator hope by studying genes the investigator can learn more about the inflammation that occurs after heart surgery, but the investigators might use participant's genetic information to study other diseases or conditions other the inflammation that occurs after heart surgery. The investigators will be studying the recovery of 60 participants between 1 month to 18 months of age who require open heart surgery to repair ventricular septal defects (VSDs), a congenital heart defect where there a hole between the lower chambers of the heart. Participants will: Allow for information about how the participants recover from surgery to be collected. Allow blood samples during and after surgery to be collected to understand how the markers of inflammation change between the two groups (blood versus non-blood priming).

Traumatic brain injury (TBI) is a significant problem for U.S. Hispanic children. Compared to non-Hispanic children, Hispanic children have higher long-term disability and lower health related quality of life, even though differences are not present at hospital discharge. Rehabilitation decreases disability, but needs timely initiation, and long treatments in hospitals, community healthcare facilities and schools. Parents play a key role in their child's recovery. Hispanic parents face additional barriers to initiate and maintain outpatient treatments. They report knowledge gaps in TBI-education, community, and school support systems; language and health literacy barriers. The investigators developed, a bilingual bicultural theory-based program for Hispanic families consisting of Brain Injury Education and outpatient care Navigation (1st BIEN). It integrates in-person education enriched by video content delivered through mobile phones, with navigation during transitions to outpatient care and school return. The pilot established feasibility and acceptability of the program. This randomized control trial will determine efficacy to maintain long-term adherence to rehabilitation and reduce disability. It will enroll 150 parent-child dyads: children (6-17 y), with mild-complicated, moderate-severe TBI in 5 centers in Washington, Texas, Dallas, Utah and Oregon and their parents. Intervention group parents receive: One in-person education session, plus bi-weekly videos tailored to the child's TBI and therapies; and, 3-months of bilingual outpatient care navigation. Attention control parents receive one in person-education session, monthly well-child texts and usual institutional follow up care. Primary outcome is treatment adherence at 6 months post-discharge measured by percentage of follow-up appointments attended during the prescribed time at hospitals, and community care facilities. Secondary outcomes are functional status of the child using PROMIS parental report measures; and parental health literacy, self-efficacy, and mental health at 3, 6, and 12 months after discharge. Child's academic performance will be assessed using school records. The study evaluates a flexible and scalable intervention using mobile phones to aid transitions of care, improve treatment adherence and TBI outcomes. It addresses the needs of an understudied population and can serve as a model for TBI family centered care for at risk groups.

A phase 1, open-label, non-randomized study enrolling pediatric and young adult patients with relapsed or refractory CD33+ leukemia with and without prior history of allogeneic hematopoietic cell transplantation, to examine the safety and feasibility of administering an autologous T cell product that has been genetically modified to express a Dimerizing Agent Regulated Immunoreceptor Complex (DARIC).

This randomized control trial comparing Organizational Skills Training (OST) and Mindfulness-Based Intervention (MBI) among adolescents with a pre-existing ADHD diagnosis presenting to the Duke ADHD Program. Both treatments are eight 90 minute sessions. The research component will involve a pre-treatment assessment and post-treatment assessment. Both assessments will involve adolescents and one caregiver to complete questionnaires over REDCap. Rating scales will include ADHD symptom severity (Conners 3: self and parent report), functional impairment (IRS: self and parent report), executive functioning (BRIEF-2: parent report), emotion dysregulation (DERS: self and parent report), trait mindfulness (FFMQ: self report), organizational skills (BRIEF-2: parent report), treatment satisfaction (self report and parent report) and credibility (self report and parent report). Post-treatment assessments for feasibility will include attendance (measured over the course of treatment) and homework completion rates on a scale of 1 to 5 in which 5 indicates higher homework completion. We will also assess acceptability via individual items on a Likert scale (self report): overall satisfaction, how much was learned about ADHD, usefulness of information learned, content relevance to individual experience, comprehension of strategies, confidence about using strategies, likelihood of using strategies, helpfulness to share with the group, benefits from hearing from other group members, willingness to recommend the same treatment to others, and whether or not treatment was beneficial.