Clinical trials

This is an open-label phase 1 safety and feasibility study evaluating a novel combination therapy for Diffuse Midline Glioma (DMG), an aggressive brain tumor with a very poor prognosis - an average one-year overall survival. This study combines blood-brain barrier (BBB) disruption (BBBD) using low-intensity focused ultrasound (LIFU) and microbubble treatment, and intravenous infusion of autologous, tumor multi-antigen associated specific cytotoxic T lymphocyte (TAA-T) therapy. The TAA-T cell investigational product in this protocol is manufactured to target Preferentially Expressed Antigen of Melanoma (PRAME), Wilms Tumor 1 (WT1), and survivin.

The goal of this clinical trial is to learn if a new school-based, Tier 2 (selective) cognitive-behavioral intervention called Unstuck and On Target for Early Childhood (Unstuck-EC) can treat executive function deficits in young children (ages 4-5). Executive function is a critical set of cognitive abilities that enable individuals to demonstrate skills, effectively complete tsks, and navigate social interactions through flexible regulation of their thinking, behavior, and emotions. All children will be receiving special education services and be identified by their classroom teachers as having problems with flexibility and/or emotion regulation. Unstuck-EC consists of 16 sessions, each lasting about 20 minutes, delivered 1-2 times per week. Unstuck-EC will be adapted in this trial to be delivered by classroom teachers. The main questions it aims to answer are: 1. Do special education preschool teachers find Unstuck-EC to be feasible, appropriate, and acceptable for use in their classrooms? 2. Do special education preschool teachers trained in Unstuck-EC better understand and support executive function in the classroom? 3. Does Unstuck-EC improve children's executive function skills? Researchers will compare Unstuck-EC (N=45 participants) to a "services as usual" control (N=45 participants). This means that all children will receive usual services at school through their Individualized Education Plan and associated special education supports. Those in Unstuck-EC will also receive the intervention. Participants in both groups will be followed for the same period of time (minimum of 8 weeks). All child participants will complete a baseline assessment, which include language testing for eligibility, as well as a classroom observation and direct testing of cognitive flexibility. Additionally, parents and case managers will complete behavioral rating scales of children's executive function skills and classroom behavior. All measures will be collected again at the end of the intervention period, with the exception of language testing. Additional measures will also be completed by the teacher to examine knowledge of executive function and use of Unstuck-EC strategies and executive-function supporting strategies. Teachers will also report on their own stress and self-efficacy and will complete ratings of child expulsion risk. Participants will \[describe the main tasks participants will be asked to do, interventions they'll be given and use bullets if it is more than 2 items\].

The primary objective is to assess if an AI-generated plain language summary of an inpatient progress note can improve caregiver understanding of their child's hospitalization by measuring differences in Hospital Course Comprehension scores, based on their responses to a survey regarding their child's hospital course. Secondary objectives include exploring the caregiver's perspective on the use of AI in clinical care, as well as 7-day and 30-day readmission rates, 7-day and 30-day emergency department (ED) re-utilization rates, length of stay, and environmental impact measures.

This is an open-label phase 1 safety and feasibility study evaluating a novel combination therapy for progressive and relapsed diffuse midline glioma (DMG) and other progressive and relapsed high-grade brain tumors. This study combines intravenous nivolumab therapy infused following transient blood-brain barrier opening (BBBO) using low-intensity focused ultrasound with microbubble (LIFU-MB) treatment using NeuroNavigation-Guided Focused Ultrasound (NgFUS). There are two groups in this study: * Group A: Patients with relapsed or progressive diffuse midline glioma in the brainstem * Group B: Patients with relapsed or progressive high grade brain tumor that clinically require surgical resection The primary outcome is to evaluate the safety and feasibility of 3 cycles of nivolumab with BBB disruption using NgFUS with microbubbles in pediatric patients with progressive or relapsed brainstem DMG or with high grade brain tumors after surgery. Secondary outcomes include preliminary efficacy and immunological effects.

This is a research study to find out if treatment with transdermal estradiol patches started between 8 to 11.5 years is safe in girls with Turner syndrome who have ovarian failure and to see whether it may improve performance on two tests of working speed and short-term memory.

This is a phase I dose-escalation study to determine the safety and feasibility of autologous CAR-TA T cells (B7-H3 CAR+ T cells administered with DNR-PRAME Tumor Antigen-specific T cells) following lymphodepleting chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor. Patients will be enrolled to one of three planned dose levels with B7-H3 CAR T cell dose determined based on the percentage of B7-H3 transduced cells (B7-H3+ population of cells), and dTBRII-transduced PRAME TA-specific T cell dose based on the total cell population. Both doses will be based on the recipient's body weight and combined in a 1:1 ratio. The safety of the CAR-TA T cell product will be evaluated and the maximum tolerated dose (MTD) will be determined. The safety endpoint will be assessed by monitoring for dose limiting toxicities for 28 days following CAR-TA T cell administration.

The objective of the International Rare Brain Tumor Registry (IRBTR) is to better understand rare brain tumors through the collection of biospecimens and matched clinical data of children, adolescents, and young adult patients diagnosed with rare brain tumors.

This is an open-label phase I dose-escalation study to evaluate the safety of partially human leukocyte antigen (HLA)-matched multi tumor-associated antigen-specific T cell (TAA-T) therapy following lymphodepleting conditioning with or without local tumor ablation for pediatric and adult patients with high-risk solid tumors due to the presence of refractory, relapsed and/or minimal residual detectable disease following conventional therapy (e.g., chemotherapy, surgery, radiation, autologous stem cell transplant, or targeted therapy).

This a single site, observational study that will examine how the pupil responds to neurostimulation under different conditions in healthy adults. The objective is to measure pupillary dilation reflex (PRD) in response to increasing neurostimulation intensities using the AlgometRx device.

The overarching goal of this study is the development of a physiologic endpoint of pain and treatment effect in three distinct rheumatology populations. This would enable objective assessment of pain and treatment in these populations and enable a much more precise approach to treatment. Such an endpoint stands to significantly improve outcomes in these patients by eliminating the need for a trial-and-error approach to treatment. This is a single site observational study that aims to collect initial pilot data in three distinct patient groups. As this is observational, there is no randomization or blinding in the study. Patients will be followed for a period of one year after enrollment. Baseline measurements will be taken at the time of enrollment, and at each subsequent standard of care clinic visit as feasible, for a period of one year. As this is an observational study, there will be no change to the treatment for any patient due to research activities. The primary objective of this study is the characterization of the nociceptive index in three pediatric rheumatology populations. The secondary objective is the characterization of the nociceptive index in these populations in response to standard of care interventions. This is necessary to demonstrate the ability of this approach to serve as an endpoint of treatment effect.

The purpose of this study is the development of a physiologic endpoint using a novel technology that would provide an objective, easy to use and more sensitive assessment of VIPN in children and adolescents. The ability to more easily detect and monitor VIPN, even before it is clinically evident, would facilitate optimizing the dosing of vincristine for maximal disease response while minimizing the risk of lifelong functional deficits affecting quality of life. This approach would also enable the development of specific therapies to minimize or eliminate the occurrence of VIPN in children and adolescents. This is a single site study that aims to develop a novel device to evaluate and characterize vincristine-induced neuropathic pain. The investigators will enroll patients with ALL following the Delayed Intensification (DI) phase of treatment. At each study visit, the investigators will evaluate the nPRD as well as the TNS-PV. The nPRD will inform the neuropathy index which will be used to compare to the TNS-PV. We anticipate a correlation between the two.

This is a mixed methods study exploring the experiences of pregnancy and parenting among participants with Lyme disease. Eligible participants will have been diagnosed with Lyme disease (LD), post-treatment Lyme disease syndrome (PTLDS), and/or chronic Lyme (CL) either during or before a prior pregnancy. Participants will complete quantitative surveys on topics such as their medical history, their child(ren)'s development, and demographic information. They will then participate in a qualitative interview where they will be asked about their experiences with pregnancy and with parenting their child(ren) in the context of their condition.

This is an open-label phase 1 safety and feasibility study that will employ multi-tumor antigen specific cytotoxic T lymphocytes (TSA-T) directed against proteogenomically determined personalized tumor-specific antigens (TSA) derived from a patient's primary brain tumor tissues. Young patients with embryonal central nervous system (CNS) malignancies typically are unable to receive irradiation due to significant adverse effects and are treated with intensive chemotherapy followed by autologous stem cell rescue; however, despite intensive therapy, many of these patients relapse. In this study, individualized TSA-T cells will be generated against proteogenomically determined tumor-specific antigens after standard of care treatment in children less than 5 years of age with embryonal brain tumors. Correlative biological studies will measure clinical anti-tumor, immunological and biomarker effects.

The purpose of this pilot study is to assess the feasibility of longitudinal neurodevelopmental evaluation of fetuses and infants exposed to Lyme disease in utero. Participants with Lyme disease or Post-Treatment Lyme Disease Syndrome (PTLDS) will be recruited during pregnancy. Pregnancies will be monitored and infant development will be assessed from birth until age 18 months.

The primary objective of the proposed study is to evaluate the safety of ExAblate Transcranial MRgFUS as a tool for creating bilateral or unilateral lesions in the globus pallidus (GPi) in patients with treatment-refractory secondary dystonia due to dyskinetic cerebral palsy

This clinical trial is studying a drug called SGT-53 along with radiation and another drug called Nivolumab. It's for children with brain tumors that have come back, gotten worse, or didn't get better with earlier treatments. The main questions it aims to answer are: What is the right dose of SGT-53 that children can safely receive when it is used with radiation and Nivolumab? This dose will be used in the second phase of the trial. What side effects are there of SGT-53 when it is used with radiation and Nivolumab? How does SGT-53 move through the body when given with radiation and Nivolumab? How much of the SGT-53 drug is found in the tumor tissue? This will be tested in a small group of patients? Participants will: For the first treatment cycle: Get SGT-53 twice per week Get Nivolumab every 2 weeks Receive radiation therapy during week 2 For Cycles 2-6: Get SGT-53 once per week during even cycles and twice per week during odd cycles Get Nivolumab every 2 weeks For Cycles 7+ Get both SGT-53 and Nivolumab every 2 weeks In the phase 0 part of the study, 4 participants will have genetic testing performed on their tumor tissue after receiving SGT-53. These samples will be compared to another sample taken from the skin.

The purpose of this clinical study is the development of physiologic endpoint of inflammation in pediatric patients diagnosed with inflammatory bowel disease (IBD), specifically subtypes Crohn's disease (CD) and ulcerative colitis (UC). The novel medical device evaluates the patient's sensory response to each of the three sensory nerve fiber types. Data from the device provides an assessment of disease activity and a more precise approach to treatment.

We will compare differences in sexually transmitted infection (STI) detection rates between sexual health survey (SHS)-derived electronic clinical decision support (CDS) versus usual care (e.g. no provision of CDS) using an interrupted time series design. We hypothesize that population-based STI detection rates will be higher when SHS-derived electronic CDS is provided compared to usual care. Secondary analysis will include a comparison of STI detection rates by sexual risk strata (high risk vs. at risk) and race/ethnicity.

This is an open label, phase I dose-escalation study to evaluate the safety of coronavirus-specific T cell (CST) therapy for prevention of SARS-CoV-2 infection in immunocompromised patients following hematopoietic stem cell transplantation (HSCT). Participants will receive donor-derived CSTs for prevention of SARS-CoV-2 infection after HSCT (≥28 days and \<4 months after HSCT). In this dose escalation trial, three doses (1x107/m2, 2x107/m2, and 4x107/m2) will be tested for safety, with study arms for adult (≥18 years of age and \<80 years) HSCT recipients (Arm A) and two arms for pediatric (≥12 years of age and \<18 years; ≥2 years and \<12 years) HSCT recipients (Arm B and Arm C, respectively), and defined dose escalations in each study arm. The study agent will be assessed for safety (stopping rules defined) and antiviral activity.

The goal of this clinical trial is to test the effectiveness and implementation of delivering Enhanced Brief Interpersonal Psychotherapy (IPT-B), an evidence-based maternal depression treatment, to mothers of children under the age of 18 in an urban pediatric asthma clinic. Researchers will compare Enhanced IPT-B and supplemented usual care (brief care coordination). The main questions the trial aims to answer are: 1. Does Enhanced IPT-B decrease maternal depressive symptoms? 2. Does Enhanced IPT-B improve child asthma management and health outcomes (exacerbations, symptoms, control)? 3. What are the preliminary implementation outcomes of delivering Enhanced IPT-B in an urban pediatric asthma clinic?

This is a Phase I dose-escalation study to evaluate the safety of norovirus -specific T-cell (NST) therapy for chronic norovirus infection in participants following hematopoietic stem cell transplantation (HSCT) or who are immunocompromised due to PID and have not undergone HSCT, or Solid Organ Transplant (SOT) recipients.

This study aims to better understand how acute pain and responses to pain treatment can be measured in children and adolescents using a non-invasive device. Pain is usually assessed using self-reported or observational scales, which can be difficult to interpret, especially in pediatric patients. This study will evaluate whether a novel device, the AlgometRx Nociometer, can provide an objective measure of nociceptive pain. Participants ages 6 to 21 years who are undergoing elective surgery or bone marrow transplantation will be enrolled at Children's National Hospital. The device measures changes in pupil size in response to gentle electrical stimulation, which reflects activity in pain-related nerve pathways. Measurements will be taken before and after procedures and during routine hospital care, alongside standard pain assessments. This is an observational study. Participation will not change or guide any medical treatment, and all pain management will follow standard clinical care. The information collected may help improve future pain assessment and treatment for pediatric patients.

This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies.

The purpose of this study is to determine feasibility and safety of using an augmented reality system in patients undergoing MRI-Guided needle procedures.

This is a single-center prospective study evaluating the efficacy of a prototype gastrostomy anchor dressing for use with button gastrostomy tubes. The anchor dressing has been previously evaluated for usability by parents, patients, and nurses. This study compares several outcomes related to complications from gastrostomy tube placement using a prospective cohort of patients and a retrospective analysis of complications associated with gastrostomy tubes. Prospective patients will initially be monitored until they have completed their last dressing change or for a maximum of 50 days. They will then complete their final survey. Their charts will be reviewed for up to one year from their initial gastrostomy placement. The study's primary objective is to determine if the novel gastrostomy tube anchor dressing reduces rates of gastrostomy tube dislodgement and ED/clinic visits for gastrostomy tube complications.