Search Clinical Trials
Sponsor Condition of Interest |
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REgiStry Of the NAtural History of recurreNt periCarditis in pEdiatric and Adult Patients
Kiniksa Pharmaceuticals (UK), Ltd.
Recurrent Pericarditis
The registry will focus on furthering the understanding of the natural history of
recurrent pericarditis (RP), as well as document RP-related clinical, health-related
quality of life (HRQoL), and economic burden and will assist the medical community to
refine or develop data-driven recommendations... expand
The registry will focus on furthering the understanding of the natural history of recurrent pericarditis (RP), as well as document RP-related clinical, health-related quality of life (HRQoL), and economic burden and will assist the medical community to refine or develop data-driven recommendations for clinical management of RP patients to optimize clinical outcomes. It also aims to generate data in support of the impact of rilonacept on clinical outcomes in a real-world population. Type: Observational [Patient Registry] Start Date: Mar 2021 |
Pediatric Percutaneous Ultrasound Gastrostomy Technique
CoapTech
Gastrostomy
Gastrostomy Complications
Pediatric Disorder
Ultrasound
The purpose of this research study is to test a new device called the PUMA-G Pediatric
System. The research will measure if the device works well to safely aid doctors placing
gastrostomy feeding tubes in children. The PUMA-G Pediatric System is an investigational
device that uses ultrasound and magnets... expand
The purpose of this research study is to test a new device called the PUMA-G Pediatric System. The research will measure if the device works well to safely aid doctors placing gastrostomy feeding tubes in children. The PUMA-G Pediatric System is an investigational device that uses ultrasound and magnets to guide insertion of a feeding tube. Type: Interventional Start Date: Jul 2023 |
International Rare Brain Tumor Registry
Children's National Research Institute
Astroblastoma
BCOR ITD Sarcoma
CNS Sarcoma
Unclassified Tumor, Malignant
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. expand
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. Type: Observational [Patient Registry] Start Date: Jan 2023 |
Orphan Europe Carbaglu® Surveillance Protocol
Nicholas Ah Mew
N-acetylglutamate Synthase (NAGS) Deficiency
The purpose of this study is to conduct post-marketing surveillance of carglumic acid
(Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved
by the United States Food and Drug Administration (FDA) for treatment of acute
hyperammonemia due to N-acetylglutamate synthase... expand
The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved by the United States Food and Drug Administration (FDA) for treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency. Much of the FDA-required data is already collected through the Longitudinal Study of Urea Cycle Disorders (RDCRN Protocol #5101). This study will collect additional data on adverse events (interim events), adverse reactions, pregnancy, and fetal outcomes. Type: Observational [Patient Registry] Start Date: Apr 2012 |
The Myelin Disorders Biorepository Project
Children's Hospital of Philadelphia
Leukodystrophy
White Matter Disease
Leukoencephalopathies
4H Syndrome
Adrenoleukodystrophy
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical
data and biological samples from leukodystrophy patients worldwide to support ongoing and
future research projects. The MDBP is one of the world's largest leukodystrophy
biorepositories, having enrolled nearly... expand
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago. Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future. Type: Observational [Patient Registry] Start Date: Dec 2016 |
Azithromycin Treatment for Respiratory Syncytial Virus-induced Respiratory Failure in Children
University of Alabama at Birmingham
Respiratory Syncytial Virus Infections
The overarching hypothesis of the ARRC trial is that administration of Azithromycin (AZM)
during acute, Respiratory Syncytial Virus (RSV)-induced respiratory failure will be
beneficial, mediated through the matrix metalloproteinase (MMP)-9 pathway. expand
The overarching hypothesis of the ARRC trial is that administration of Azithromycin (AZM) during acute, Respiratory Syncytial Virus (RSV)-induced respiratory failure will be beneficial, mediated through the matrix metalloproteinase (MMP)-9 pathway. Type: Interventional Start Date: Feb 2022 |
A Feasibility Safety Study of Benign Centrally-Located Intracranial Tumors in Pediatric and Young Adult...
InSightec
Benign Centrally-Located Intracranial Tumors
The goal of this prospective, non-randomized, single-arm, feasibility study is to develop
data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign
intracranial tumors which require clinical intervention in pediatric and young adult
subjects.
Indication of Use: Ablation of... expand
The goal of this prospective, non-randomized, single-arm, feasibility study is to develop data to evaluate the safety and feasibility of ExAblate 4000 treatment of benign intracranial tumors which require clinical intervention in pediatric and young adult subjects. Indication of Use: Ablation of benign intracranial tumors in children and young adults which are ExAblate accessible. Type: Interventional Start Date: Feb 2017 |
The Pediatric Anesthesia Quality Improvement Project
The Society for Pediatric Anesthesia
Surgery
Anesthesia
Children
The Study is designed to collect information about adverse events that occur in children
undergoing anesthesia in participating hospitals. Demographic information will be
collected on all anesthetics. An analysis of each adverse event will be performed and
entered into the database. From this information... expand
The Study is designed to collect information about adverse events that occur in children undergoing anesthesia in participating hospitals. Demographic information will be collected on all anesthetics. An analysis of each adverse event will be performed and entered into the database. From this information we will devise strategies to prevent these adverse events. Type: Observational [Patient Registry] Start Date: Feb 2008 |
Evaluation of The Food Allergy Mastery Program
Children's National Research Institute
Food Allergy in Children
The proposed research project will evaluate a novel behavioral intervention that promotes
early adolescent food allergy self-management and adjustment through 1) food allergy
education, 2) problem-solving, communication, assertiveness, and anxiety management skill
building, and 3) peer support. expand
The proposed research project will evaluate a novel behavioral intervention that promotes early adolescent food allergy self-management and adjustment through 1) food allergy education, 2) problem-solving, communication, assertiveness, and anxiety management skill building, and 3) peer support. Type: Interventional Start Date: Jun 2023 |
Multi-antigen T Cell Infusion Against Neuro-oncologic Disease
Catherine Bollard
Brain Tumor
This Phase I dose-escalation trial is designed to determine the safety and feasibility of
rapidly generated tumor multi-antigen associated specific cytotoxic T lymphocytes (TAA-T)
in patients with newly diagnosed diffuse intrinsic pontine gliomas DIPGs (Group A) or
recurrent, progressive, or refractory... expand
This Phase I dose-escalation trial is designed to determine the safety and feasibility of rapidly generated tumor multi-antigen associated specific cytotoxic T lymphocytes (TAA-T) in patients with newly diagnosed diffuse intrinsic pontine gliomas DIPGs (Group A) or recurrent, progressive, or refractory non-brainstem CNS malignancies (Group B). Pediatric and adult patients who have high-risk CNS tumors known to typically have positivity for one or more Tumor Antigen Associated (TAA) (WT1, PRAME and/or Survivin) will be eligible. TAA-T will all be generated from patient peripheral blood mononuclear cells (PBMC). Group A patients (DIPG): The first TAA-T dose will be infused any time 14 days or more after completion of radiotherapy. Group B patients (other recurrent/progressive/refractory CNS tumors): The first TAA-T dose will be infused any time 14 days or more after completing most recent course of conventional (non-investigational) therapy for their disease AND after appropriate washout periods as detailed in eligibility criteria. Type: Interventional Start Date: Dec 2018 |
A Pilot Study of Thermodox and MR-HIFU for Treatment of Relapsed Solid Tumors
Children's National Research Institute
Solid Tumors
Soft Tissue Sarcoma
Ewing Sarcoma
Malignant Epithelial Neoplasm
Rhabdomyosarcoma
This is a pilot study of LTLD with MR-HIFU hyperthermia followed by ablation in subjects
with refractory/relapsed solid tumors. expand
This is a pilot study of LTLD with MR-HIFU hyperthermia followed by ablation in subjects with refractory/relapsed solid tumors. Type: Interventional Start Date: Nov 2022 |
Central Blood Pressure and Pulse Wave Velocity in Kidney Transplant Recipients
Children's National Research Institute
End Stage Renal Disease
An observational cohort study will be conducted to non-invasively investigate central
blood pressure and pulse wave velocity in children with kidney disease and controls.
Using an oscillometric monitor, the investigators aim to non-invasively obtain the
central blood pressure and pulse wave velocity... expand
An observational cohort study will be conducted to non-invasively investigate central blood pressure and pulse wave velocity in children with kidney disease and controls. Using an oscillometric monitor, the investigators aim to non-invasively obtain the central blood pressure and pulse wave velocity (PWV), or arterial stiffness, of children with kidney disease. The investigators will also enroll age- and race-matched healthy controls and measure the same parameters for comparison. In addition, the investigators will measure PWV by standard arterial tonometry method in a subset of patients. Type: Observational Start Date: Jul 2021 |
Feasibility and Efficacy of Attentional-Control Training in Sickle Cell Disease
Children's National Research Institute
Sickle Cell Disease
Attention Deficit
Cognitive Deficit in Attention
Children with sickle cell disease (SCD) exhibit significantly reduced cognitive
functioning (often difficulties with attention) compared to peers and siblings without
SCD. EndeavorRx (Akili Interactive Labs: Boston, MA) is an FDA-approved home-based,
electronic attentional-control training program... expand
Children with sickle cell disease (SCD) exhibit significantly reduced cognitive functioning (often difficulties with attention) compared to peers and siblings without SCD. EndeavorRx (Akili Interactive Labs: Boston, MA) is an FDA-approved home-based, electronic attentional-control training program designed to treat attention problems in youth. Users access EndeavorRx on a tablet device for 25-30 minutes each day, 5 days per week, for 4 weeks. The program involves training in a game-like environment that repeatedly challenges attentional-control abilities and adapts to user performance, becoming more difficult over time as performance improves. This pilot study is examining the feasibility, acceptability, and preliminary efficacy of EndeavorRx in a sample of 20 children with SCD ages 8-16 who are being treated with chronic blood transfusion therapy. Type: Interventional Start Date: Dec 2022 |
PedBot Ankle Rehablitation
Children's National Research Institute
Cerebral Palsy
The primary objective of the study is to determine engagement with use and adherence to a
home exercise program with our PedBotHome ankle rehabilitation device. We will place the
device in the home for 90 days and consider the study a success if used for at least 30
minutes a day for at least 3 days... expand
The primary objective of the study is to determine engagement with use and adherence to a home exercise program with our PedBotHome ankle rehabilitation device. We will place the device in the home for 90 days and consider the study a success if used for at least 30 minutes a day for at least 3 days a week during this period. Type: Observational Start Date: Jul 2022 |
NIAID Centralized Sequencing Protocol
National Institute of Allergy and Infectious Diseases (NIAID)
Atopy
Primary Immunodeficiency
Autoimmunity
Autoinflammation
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... expand
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 |
Fontan Udenafil Exercise Longitudinal Assessment Trial - 2
Mezzion Pharma Co. Ltd
Single Ventricle Heart Disease
This study will evaluate the clinical efficacy and safety of udenafil, an orally
administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of
adolescent who have had the Fontan procedure. expand
This study will evaluate the clinical efficacy and safety of udenafil, an orally administered, potent and selective inhibitor of PDE5, versus placebo for the treatment of adolescent who have had the Fontan procedure. Type: Interventional Start Date: Oct 2023 |
A Study to Compare Standard Therapy to Treat Hodgkin Lymphoma to the Use of Two Drugs, Brentuximab Vedotin...
National Cancer Institute (NCI)
Lugano Classification Limited Stage Hodgkin Lymphoma AJCC v8
This phase III trial compares the effect of adding immunotherapy (brentuximab vedotin and
nivolumab) to standard treatment (chemotherapy with or without radiation) to the standard
treatment alone in improving survival in patients with stage I and II classical Hodgkin
lymphoma. Brentuximab vedotin... expand
This phase III trial compares the effect of adding immunotherapy (brentuximab vedotin and nivolumab) to standard treatment (chemotherapy with or without radiation) to the standard treatment alone in improving survival in patients with stage I and II classical Hodgkin lymphoma. Brentuximab vedotin is in a class of medications called antibody-drug conjugates. It is made of a monoclonal antibody called brentuximab that is linked to a cytotoxic agent called vedotin. Brentuximab attaches to CD30 positive lymphoma cells in a targeted way and delivers vedotin to kill them. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Chemotherapy drugs such as doxorubicin hydrochloride, bleomycin sulfate, vinblastine sulfate, dacarbazine, and procarbazine hydrochloride work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid (DNA) and may kill cancer cells. It may also lower the body's immune response. Etoposide is in a class of medications known as podophyllotoxin derivatives. It blocks a certain enzyme needed for cell division and DNA repair and may kill cancer cells. Vincristine is in a class of medications called vinca alkaloids. It works by stopping cancer cells from growing and dividing and may kill them. Prednisone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Radiation therapy uses high energy x-rays to kill tumor cells and shrink tumors. Adding immunotherapy to the standard treatment of chemotherapy with or without radiation may increase survival and/or fewer short-term or long-term side effects in patients with classical Hodgkin lymphoma compared to the standard treatment alone. Type: Interventional Start Date: May 2023 |
Vincristine Pharmacokinetics in Infants
Children's Oncology Group
Hematopoietic and Lymphoid Cell Neoplasm
Malignant Solid Neoplasm
This pilot trial compares drug exposure levels using a new method for dosing vincristine
in infants and young children compared to the standard dosing method based on body
surface area (BSA) in older children. Vincristine is an anticancer drug used to a variety
of childhood cancers. The doses anticancer... expand
This pilot trial compares drug exposure levels using a new method for dosing vincristine in infants and young children compared to the standard dosing method based on body surface area (BSA) in older children. Vincristine is an anticancer drug used to a variety of childhood cancers. The doses anticancer drugs in children must be adjusted based on the size of the child because children vary significantly in size (height, weight, and BSA) and ability to metabolize drugs from infancy to adolescence. The dose of most anticancer drugs is adjusted to BSA, which is calculated from a patient's weight and height. However, infants and young children have more severe side effects if the BSA is used to calculate their dose, so new dosing models have to be made to safely give anticancer drugs to the youngest patients. This new method uses a BSA-banded approach to determine the dose. Collecting blood samples before and after a dose of the drug will help researchers determine whether this new vincristine dosing method results in equivalent drug levels in the blood over time in infants and young children compared to older children. Type: Observational Start Date: Nov 2022 |
A Study Investigating Oral Ozanimod (RPC1063) in Pediatric Participants With Moderate to Severe Active...
Bristol-Myers Squibb
Colitis, Ulcerative
The purpose of this study is to evaluate the effectiveness and safety of ozanimod
(RPC1063) in achieving and maintaining clinical remission. Ozanimod will be administered
orally to pediatric participants with moderate to severe active ulcerative colitis (UC)
who have had an inadequate response to... expand
The purpose of this study is to evaluate the effectiveness and safety of ozanimod (RPC1063) in achieving and maintaining clinical remission. Ozanimod will be administered orally to pediatric participants with moderate to severe active ulcerative colitis (UC) who have had an inadequate response to conventional therapy. Type: Interventional Start Date: May 2022 |
Immunotherapy Before and After Surgery for Treatment of Recurrent or Progressive High Grade Glioma in...
Sabine Mueller, MD, PhD
Glioblastoma
Malignant Glioma
Recurrent Glioblastoma
Recurrent Malignant Glioma
Recurrent Grade III Glioma
This phase I trial studies the side effects of nivolumab before and after surgery in
treating children and young adults with high grade glioma that has come back (recurrent)
or is increasing in scope or severity (progressive). Immunotherapy with monoclonal
antibodies, such as nivolumab, may help the... expand
This phase I trial studies the side effects of nivolumab before and after surgery in treating children and young adults with high grade glioma that has come back (recurrent) or is increasing in scope or severity (progressive). Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Type: Interventional Start Date: Oct 2020 |
Advancing Transplantation Outcomes in Children
National Institute of Allergy and Infectious Diseases (NIAID)
Kidney Transplant
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.... expand
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 |
Tiragolumab and Atezolizumab for the Treatment of Relapsed or Refractory SMARCB1 or SMARCA4 Deficient...
National Cancer Institute (NCI)
Atypical Teratoid/Rhabdoid Tumor
Epithelioid Sarcoma
Kidney Medullary Carcinoma
Malignant Solid Neoplasm
Poorly Differentiated Chordoma
This phase I/II trial studies how well tiragolumab and atezolizumab works when given to
children and adults with SMARCB1 or SMARCA4 deficient tumors that have either come back
(relapsed) or do not respond to therapy (refractory). SMARCB1 or SMARCA4 deficiency means
that tumor cells are missing the... expand
This phase I/II trial studies how well tiragolumab and atezolizumab works when given to children and adults with SMARCB1 or SMARCA4 deficient tumors that have either come back (relapsed) or do not respond to therapy (refractory). SMARCB1 or SMARCA4 deficiency means that tumor cells are missing the SMARCB1 and SMARCA4 genes, seen with some aggressive cancers that are typically hard to treat. Immunotherapy with monoclonal antibodies, such as tiragolumab and atezolizumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Type: Interventional Start Date: Nov 2022 |
Thoracotomy Versus Thoracoscopic Management of Pulmonary Metastases in Patients With Osteosarcoma
Children's Oncology Group
Metastatic Malignant Neoplasm in the Lung
Metastatic Osteosarcoma
Osteosarcoma
This phase III trial compares the effect of open thoracic surgery (thoracotomy) to
thoracoscopic surgery (video-assisted thoracoscopic surgery or VATS) in treating patients
with osteosarcoma that has spread to the lung (pulmonary metastases). Open thoracic
surgery is a type of surgery done through... expand
This phase III trial compares the effect of open thoracic surgery (thoracotomy) to thoracoscopic surgery (video-assisted thoracoscopic surgery or VATS) in treating patients with osteosarcoma that has spread to the lung (pulmonary metastases). Open thoracic surgery is a type of surgery done through a single larger incision (like a large cut) that goes between the ribs, opens up the chest, and removes the cancer. Thoracoscopy is a type of chest surgery where the doctor makes several small incisions and uses a small camera to help with removing the cancer. This trial is being done evaluate the two different surgery methods for patients with osteosarcoma that has spread to the lung to find out which is better. Type: Interventional Start Date: Apr 2022 |
Inspiring New Science In Guiding Healthcare in Turner Syndrome Registry
University of Colorado, Denver
Turner Syndrome
INSIGHTS is a registry research study that collects key information on medical history
for girls and women with Turner syndrome and the clinical care they receive. This
includes genetic tests, imaging, medications, and more for hundreds of patients seen at a
number of clinics across the US. In addition... expand
INSIGHTS is a registry research study that collects key information on medical history for girls and women with Turner syndrome and the clinical care they receive. This includes genetic tests, imaging, medications, and more for hundreds of patients seen at a number of clinics across the US. In addition to learning a lot about the current state of health for individuals with TS, INSIGHTS serves as an infrastructure to conduct future studies are meaningful to patients and their families. Type: Observational [Patient Registry] Start Date: May 2020 |
CBL0137 for the Treatment of Relapsed or Refractory Solid Tumors, Including CNS Tumors and Lymphoma
Children's Oncology Group
Diffuse Midline Glioma, H3 K27M-Mutant
Metastatic Malignant Neoplasm in the Central Nervous System
Recurrent Diffuse Intrinsic Pontine Glioma
Recurrent Lymphoma
Recurrent Malignant Solid Neoplasm
This phase I/II trial evaluates the best dose, side effects and possible benefit of
CBL0137 in treating patients with solid tumors, including central nervous system (CNS)
tumors or lymphoma that has come back (relapsed) or does not respond to treatment
(refractory). Drugs, such as CBL0137, block signals... expand
This phase I/II trial evaluates the best dose, side effects and possible benefit of CBL0137 in treating patients with solid tumors, including central nervous system (CNS) tumors or lymphoma that has come back (relapsed) or does not respond to treatment (refractory). Drugs, such as CBL0137, block signals passed from one molecule to another inside a cell. Blocking these signals can affect many functions of the cell, including cell division and cell death, and may kill cancer cells. Type: Interventional Start Date: Jan 2022 |
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