Children's National

The objective of this study is to assess the safety and efficacy of mecbotamab vedotin (BA3011) in solid tumors.

Type: Interventional

Start Date: Feb 2018

open study

This clinical trial keeps track of and collects follow-up information from patients who are currently enrolled on or have participated in a Children's Oncology Group study. Developing a way to keep track of patients who have participated in Children's Oncology Group studies may allow doctors learn more about the long-term effects of cancer treatment and help them reduce problems related to treatment and improve patient quality of life.

Type: Observational

Start Date: Jul 2008

open study

The NSR-GENE study is a longitudinal cohort study of approximately 300 parent-child trios from the Neonatal Seizure Registry and participating site outpatient clinics that aims to evaluate whether and how genes alter the risk of post-neonatal epilepsy among children with acute provoked neonatal seizures. The researchers aim to develop prediction rules to stratify neonates into low, medium, and high risk for post-neonatal epilepsy based on clinical, electroencephalogram (EEG), magnetic resonance imaging (MRI), and genetic risk factors.

Type: Observational [Patient Registry]

Start Date: Aug 2022

open study

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

Type: Interventional

Start Date: Jan 2024

open study

In this study, the investigator plans a randomized trial of melatonin versus placebo post acute pediatric concussion. The investigator hypothesizes that patients with acute concussions managed with melatonin will have improved sleep, decreased depressive symptoms, decreased risk of prolonged concussion symptoms and faster resolution of concussion symptoms.

Type: Interventional

Start Date: Mar 2023

open study

Urea cycle disorders (UCD) are a group of rare inherited metabolism disorders. Infants and children with UCD commonly experience episodes of vomiting, lethargy, and coma. The purpose of this study is to perform a long-term analysis of a large group of individuals with various UCDs. The study will focus on the natural history, disease progression, treatment, and outcome of individuals with UCD.

Type: Observational

Start Date: Feb 2006

open study

To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.

Type: Observational

Start Date: Jun 2022

open study

FIREFLY-1 is an ongoing, Phase 2, multi center, open-label study to evaluate the safety and efficacy of oral pan-RAF inhibitor DAY101 in pediatric, adolescent, and young adult patients with recurrent or progressive low-grade glioma or an advanced solid tumor harboring a known RAF alteration.

Type: Interventional

Start Date: Apr 2021

open study

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.

Type: Interventional

Start Date: Mar 2023

open study

This multisite prospective study seeks to determine if HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus (Sickle transplant Using a Nonmyeloablative approach, "SUN") can decrease the toxicity of transplant while achieving a high cure rate for children with sickle cell disease (SCD).

Type: Interventional

Start Date: Apr 2018

open study

The purpose of this study is to evaluate the safety and efficacy of targeted blood brain barrier disruption with Exablate Model 4000 Type2.0/2.1 in combination with Doxorubicin therapy for the treatment of DIPG in pediatric patients

Type: Interventional

Start Date: Jan 2023

open study

This is a 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 for patients with high-risk solid tumors due to the presence of refractory, relapsed and/or minimal residual detectable disease following conventional therapy. Conventional therapy may include chemotherapy, surgery, radiation, autologous stem cell transplant, or targeted therapy.

Type: Interventional

Start Date: Nov 2021

open study

The investigators want to study if lower doses of chemotherapy will help babies with SCID to achieve good immunity with less short and long-term risks of complications after transplantation. This trial identifies babies with types of immune deficiencies that are most likely to succeed with this approach and offers them transplant early in life before they get severe infections or later if their infections are under control. It includes only patients receiving unrelated or mismatched related donor transplants. The study will test if patients receiving transplant using either a low dose busulfan or a medium dose busulfan will have immune recovery of both T and B cells, measured by the ability to respond to immunizations after transplant. The exact regimen depends on the subtype of SCID the patient has. Donors used for transplant must be unrelated or half-matched related (haploidentical) donors, and peripheral blood stem cells must be used. To minimize the chance of graft-versus-host disease (GVHD), the stem cells will have most, but not all, of the T cells removed, using a newer, experimental approach of a well-established technology. Once the stem cell transplant is completed, patients will be followed for 3 years. Approximately 9-18 months after the transplant, vaccinations will be administered, and a blood test measuring whether your child's body has responded to the vaccine will be collected.

Type: Interventional

Start Date: Oct 2018

open study

Using stakeholder feedback (i.e., behavioral health providers & caregivers), the goal of this study is to develop a behavioral parenting program that focuses on both parent mental health and parenting for parents of toddlers (12-35 months old). Using a deployment focused intervention model, this study will : (1) develop an early parenting intervention for parents of at-risk toddlers which integrates a focus on parent mental health with evidence-based behavioral parenting strategies, and (2) examine context-specific factors related to the intervention, including feasibility and acceptability to design a more practice-ready intervention.

Type: Interventional

Start Date: Apr 2021

open study

OBOE is a prospective, pilot, parallel group RCT with the overall aim of examining the effect of a single dose of anti-IgE (omalizumab) vs. placebo administered at the onset of URIs in the fall season among highly exacerbation-prone, urban, and atopic youth aged 6-17 years with persistent asthma. OBOE will recruit and randomize participants over 3 years (3 annual cohorts of participants). Recruitment for each of the yearly cohorts of OBOE will begin in February. Each cohort will be followed for a 2-6-month run-in period with the objective to gain control of each participant's asthma and to stabilize the required controller medication step level. Participants will receive routine asthma care every 1-2 months (a total of 2-4 times) during run-in using a previously described algorithm developed by the Inner-city Asthma Consortium and successfully employed in the PROSE study. The primary outcome is the change in the amount of nasal IFN-α recovered by nasal fluid absorption between two time points, within 72 hours of onset of a URI as defined by onset of (or substantial worsening of) rhinorrhea, nasal congestion or sneezing (single or multiple symptoms) and 3-6 days after study drug injection.

Type: Interventional

Start Date: May 2022

open study

The LCH-IV is an international, multicenter, prospective clinical study for pediatric Langerhans Cell Histiocytosis LCH (age < 18 years).

Type: Interventional

Start Date: Nov 2016

open study

COMPASS is a prospective multicenter randomized interventional trial. Participants with ductal-dependent pulmonary blood flow will be randomized to receive either a systemic-to-pulmonary artery shunt or ductal artery stent. Block randomization will be performed by center and by single vs. two ventricle status. Participants will be followed through the first year of life.

Type: Interventional

Start Date: Jun 2022

open study

The GRACE-2 study is a prospective, multi-center, double-blind, randomized controlled trial of the drug GM-CSF vs placebo in children with sepsis-induced multiple organ dysfunction syndrome (MODS) who have immunoparalysis with mild to moderate inflammation.

Type: Interventional

Start Date: Jun 2022

open study

This study develops and refines an online platform that will support clinician-directed behavioral and organizational skills intervention for adolescents with Attention-Deficit/Hyperactivity Disorder (ADHD) with input guided from key stakeholders during focus groups and interviews (phase 1), extended usability testing (phase 2), and a pilot randomized trial (phase 3) of the online tool used in conjunction with an organizational skills intervention.

Type: Interventional

Start Date: Feb 2021

open study

In this study, the investigators propose a novel method to detect Down syndrome using photography for facial dysmorphology, a tool called computer-aided diagnosis (CAD). After validating the method, this technology will be expanded to perform similar functions to assist in the detection of other dysmorphic syndromes. By using photography and image analysis this automated assessment tool would have the potential to improve the diagnosis rate and allow for remote, non-invasive diagnostic evaluation for dysmorphologists in a timely manner.

Type: Interventional

Start Date: Feb 2013

open study

The purpose of this study is to evaluate the device performance and monitor the safety and effectiveness of the Berlin Heart EXCOR Active Driving System while being used with the approved EXCOR Pediatric Ventricular Assist Device. EXCOR Active Driving System is intended for use with the approved EXCOR Pediatric VAD. The EXCOR Pediatric VAD is intended to provide mechanical circulatory support as a bridge to cardiac transplantation for pediatric patients. Pediatric candidates with severe isolated left ventricular or biventricular dysfunction who are candidates for cardiac transplant and require circulatory support may be treated using the EXCOR Pediatric. EXCOR Active is intended for use in a clinical setting. EXCOR Active can be used in any kind of hospital unit (e.g. OR, ICU, intermediate care unit or general care unit). The driving unit may be moved between clinical units using the caddy or baby buggy; however, a patient must always be accompanied by a person trained in the use of the manual pump and emergency procedures during transport in the event of an emergency. The driving unit can be transported during operation.

Type: Interventional

Start Date: Nov 2022

open study

Background: Genetic testing called "sequencing" helps researchers look at DNA. Genes are made of DNA and are the instructions for our bodies to function. We all have thousands of genes. DNA variants are differences in genes between two people. We all have lots of variants. Most are harmless and some cause differences like blue or brown eyes. A few variants can cause health problems. Objective: To understand the genetics of immune disorders various health conditions, as well as outcomes of clinical genomics and genetic counseling services performed under this protocol. Eligibility: Participants in other NIH human subjects research protocols - either at the NIH Clinical Center (CC) or at Children s National Health System (CNHS) - (aged 0-99 years), and, in select cases, their biological relatives Design: Researchers will study participant s DNA extracted from blood, saliva, or another tissue sample, including previously collected samples we may have stored at the NIH. Researchers will look at participant s DNA in great detail. We are looking for differences in the DNA sequence or structure between participants and other people. Participants will receive results that: - Are important to their health - Have been confirmed in a clinical lab - Suggest that they could be at risk for serious disease that may affect your current or future medical management. Some genetic information we return to participants may be of uncertain importance. If genetic test results are unrelated to the participant s NIH evaluations, then we will not typically report: - Normal variants - Information about progressive, fatal conditions that have no effective treatment - Carrier status (conditions you don t have but could pass on) The samples and data will be saved for future research. Personal data will be kept as private as possible. If future studies need new information, participants may be contacted....

Type: Observational

Start Date: Jul 2017

open study

Participants are being asked to be in the study if they are the parent or legal guardian of a child (>1 year or <18 years old) with a rare condition. The group based psychoeducational intervention is called Rare Group Problem Management Plus. Rare Group PM Plus may help adults with practical and emotional problems. It is a group program (there will be other men or women with similar problems) It happens once a week for 5 weeks (each session lasts 90 minutes) Participants will complete assessments before they start Rare Group PM+. Participants will also complete the same assessments within a few weeks of completing Rare Group PM+. Assessments should only take one hour. Study visits are by Telemedicine. Participants will need a smart phone or tablet. If they do not have a smart phone or tablet, the study team will help with this. Participants will not receive any materials or money or medication.

Type: Interventional

Start Date: Feb 2024

open study

This is a pediatric kidney transplant study comparing the safety and efficacy of an immunosuppressive regimen of belatacept and sirolimus to tacrolimus and Mycophenolate Mofetil (MMF). Two hundred participants will be randomized (1:1) to one of two groups within 24 hours following the transplant procedure. The duration of the study from time of transplant to the primary endpoint is 12-24 months.

Type: Interventional

Start Date: May 2024

open study

Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular treatment for patients with aplastic anemia who have a matched sibling (brother or sister), or family donor is a bone marrow transplant. Patients without a matched family donor normally are treated with immune suppressive therapy (IST). Match unrelated donor (URD) bone marrow transplant (BMT) is used as a secondary treatment in patients who did not get better with IST, had their disease come back, or a new worse disease replaced it (like leukemia). This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat SAA. The trial will also assess whether health-related quality of life and early markers of fertility differ between those randomized to URD BMT or IST, as well as assess the presence of marrow failure-related genes and presence of gene mutations associated with MDS or leukemia and the change in gene signatures after treatment in both study arms. This study treatment does not include any investigational drugs. The medicines and procedures in this study are standard for treatment of SAA.

Type: Interventional

Start Date: Jan 2023

open study