Search Clinical Trials
Sponsor Condition of Interest |
---|
Safety of Sildenafil in Premature Infants With Severe Bronchopulmonary Dysplasia
Christoph Hornik
Bronchopulmonary Dysplasia of Newborn
This is a multicenter, randomized, placebo-controlled, sequential dose-escalating,
double-masked, safety study of sildenafil in premature infants (inpatient in Neonatal
Intensive Care Units (NICUs)) with severe bronchopulmonary dysplasia (BPD). expand
This is a multicenter, randomized, placebo-controlled, sequential dose-escalating, double-masked, safety study of sildenafil in premature infants (inpatient in Neonatal Intensive Care Units (NICUs)) with severe bronchopulmonary dysplasia (BPD). Type: Interventional Start Date: May 2021 |
Optune for Children With High-Grade Glioma or Ependymoma, and Optune With Radiation Therapy for Children...
Pediatric Brain Tumor Consortium
Malignant Glioma
Ependymoma
Diffuse Intrinsic Pontine Glioma
This is a multicenter trial of the Optune device to examine the feasibility and to
describe the device-related toxicity in children with supratentorial high grade glioma
(HGG) or ependymoma (Stratum 1) and to examine the feasibility and efficacy of concurrent
Optune and standard focal radiation therapy... expand
This is a multicenter trial of the Optune device to examine the feasibility and to describe the device-related toxicity in children with supratentorial high grade glioma (HGG) or ependymoma (Stratum 1) and to examine the feasibility and efficacy of concurrent Optune and standard focal radiation therapy (RT) in children with newly diagnosed diffuse intrinsic pontine glioma (DIPG) (Stratum 2). Type: Interventional Start Date: Apr 2017 |
Neuroimaging and Neuropsychological Outcomes in Urea Cycle Disorders
Children's National Research Institute
Urea Cycle Disorders
In proximal urea cycle disorders (UCD), particularly ornithine transcarbamylase
deficiency (OTCD), hyperammonemia (HA) causes increased brain glutamine (Gln) which
perturbation is thought to be at the core of the neurological injury. In contrast, in
distal UCD such as citrullinemia (argininosuccinate... expand
In proximal urea cycle disorders (UCD), particularly ornithine transcarbamylase deficiency (OTCD), hyperammonemia (HA) causes increased brain glutamine (Gln) which perturbation is thought to be at the core of the neurological injury. In contrast, in distal UCD such as citrullinemia (argininosuccinate synthetase deficiency; (ASSD) and argininosuccinic aciduria (argininosuccinate lyase deficiency); (ASLD) cognitive impairment and neuropsychiatric disease are common even in the absence of acute HA. As a consequence, both citrulline and argininosuccinate (ASA) or their metabolic products have been implicated as neurotoxic. In this project the investigators will use state-of- the-art neuroimaging and neuropsychological methods to investigate whether patients with OTCD have chronically elevated brain Gln and reduced myo-inositol (mI) levels that correlate with regional brain structural abnormalities and neurocognitive dysfunction. The researchers will further investigate whether during an acute episode of HA elevated brain Gln and decreased mI levels correlate with the magnitude of cytotoxic edema and whether a Gln/mI ratio threshold can be identified at which the cytotoxic edema is followed by cell loss. Finally, the researchers will investigate whether regions of brain damage in ASSD and/or ASLD are distinct from those in OTCD and compare brain Gln levels in ASSD and ASLD in the absence of HA to those in OTCD. The investigators will also seek to determine if brain citrulline and ASA can be identified in the brains of patients with distal UCD and whether they correlate with brain abnormalities seen in MRI and neuropsychological testing. This project will elucidate the chronology of brain pathology both in acute hyperammonemia and chronic UCD and whether, proximal and distal UCD differ in their pathophysiology of brain damage. Type: Observational Start Date: Aug 2016 |
Protocol CAUSE-03 / CHEETAH
National Institute of Allergy and Infectious Diseases (NIAID)
Asthma
This is a one-year longitudinal, observational study of 250 urban children and
adolescents with asthma and 60 without asthma, ages 6-17 years old.
Participants with asthma will require daily controller therapy with inhaled
corticosteroids ICS (at least Step 2 therapy). Those without asthma cannot... expand
This is a one-year longitudinal, observational study of 250 urban children and adolescents with asthma and 60 without asthma, ages 6-17 years old. Participants with asthma will require daily controller therapy with inhaled corticosteroids ICS (at least Step 2 therapy). Those without asthma cannot have used asthma medications in the year prior to enrollment and cannot demonstrate bronchodilator reversibility at baseline. Phenotypic characteristics will be established at baseline, and the participants will be seen at scheduled visits over 12 months. Each participant will be asked to monitor and self-report cold symptoms and will be asked to complete up to three cold visits Type: Observational Start Date: Apr 2024 |
Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults with Fragile X Syndrome
Zynerba Pharmaceuticals, Inc.
Fragile X Syndrome
This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess
the efficacy and safety of Cannabidiol administered as ZYN002 for the treatment of
children, adolescent, and young adult patients with Fragile X Syndrome (FXS). Eligible
participants will participate in up to... expand
This is a randomized, double-blind, placebo-controlled, multiple-center study, to assess the efficacy and safety of Cannabidiol administered as ZYN002 for the treatment of children, adolescent, and young adult patients with Fragile X Syndrome (FXS). Eligible participants will participate in up to an 18-week treatment period, where all participants will receive placebo or active study drug. Patients ages 3 to < 30 years will be eligible to participate. Type: Interventional Start Date: Sep 2021 |
Evaluation of Immunologic Response Following COVID-19 Vaccination in Children, Adolescents, and Young...
Children's Oncology Group
COVID-19 Infection
Hematopoietic and Lymphatic System Neoplasm
Malignant Solid Neoplasm
This study evaluates immunologic response following COVID-19 vaccination in children,
adolescents, and young adults with cancer. Vaccines work by stimulating the body's immune
cells to respond against a specific disease. The immune response produces protection from
that disease. Effects from cancer... expand
This study evaluates immunologic response following COVID-19 vaccination in children, adolescents, and young adults with cancer. Vaccines work by stimulating the body's immune cells to respond against a specific disease. The immune response produces protection from that disease. Effects from cancer and from treatments for cancer can reduce the body's natural disease fighting ability (called immunity). Factors such as vaccine type, timing of vaccine dosing related to treatment for cancer and number of vaccine doses or "boosts" (extra vaccine shots) may strengthen or diminish the body's protective immune response. This study may help researchers learn more about how the body's immune system responds to the COVID-19 vaccine when the vaccination is given during or after cancer treatment. Type: Observational Start Date: Apr 2022 |
Pediatric Dose Optimization for Seizures in Emergency Medical Services
Stanford University
Seizures
The Pediatric Dose Optimization for Seizures in Emergency Medical Services (PediDOSE)
study is designed to improve how paramedics treat seizures in children on ambulances.
Seizures are one of the most common reasons why people call an ambulance for a child, and
paramedics typically administer midazolam... expand
The Pediatric Dose Optimization for Seizures in Emergency Medical Services (PediDOSE) study is designed to improve how paramedics treat seizures in children on ambulances. Seizures are one of the most common reasons why people call an ambulance for a child, and paramedics typically administer midazolam to stop the seizure. One-third of children with active seizures on ambulances arrive at emergency departments still seizing. Prior research suggests that seizures on ambulances continue due to under-dosing and delayed delivery of medication. Under-dosing happens when calculation errors occur, and delayed medication delivery occurs due to the time required for dose calculation and placement of an intravenous line to give the medication. Seizures stop quickly when standardized medication doses are given as a muscular injection or a nasal spray. This research has primarily been done in adults, and evidence is needed to determine if this is effective and safe in children. PediDOSE optimizes how paramedics choose the midazolam dose by eliminating calculations and making the dose age-based. This study involves changing the seizure treatment protocols for ambulance services in 20 different cities, in a staggered and randomly-assigned manner. One aim of PediDOSE is to determine if using age to select one of four standardized doses of midazolam and giving it as a muscular injection or nasal spray is more effective than the current calculation-based method, as measured by the number of children arriving at emergency departments still seizing. The investigators believe that a standardized seizure protocol with age-based doses is more effective than current practice. Another aim of PediDOSE is to determine if a standardized seizure protocol with age-based doses is just as safe as current practice, since either ongoing seizures or receiving too much midazolam can interfere with breathing. The investigators believe that a standardized seizure protocol with age-based doses is just as safe as current practice, since the seizures may stop faster and these doses are safely used in children in other healthcare settings. If this study demonstrates that standardized, age-based midazolam dosing is equally safe and more effective in comparison to current practice, the potential impact of this study is a shift in the treatment of pediatric seizures that can be easily implemented in ambulance services across the United States and in other parts of the world. Type: Interventional Start Date: Aug 2022 |
Trametinib and Everolimus for Treatment of Pediatric and Young Adult Patients With Recurrent Gliomas...
University of California, San Francisco
Recurrent World Health Organization (WHO) Grade II Glioma
Low-grade Glioma
High Grade Glioma
This phase I trial studies the side effects and best dose of trametinib and everolimus in
treating pediatric and young adult patients with gliomas that have come back (recurrent).
Trametinib acts by targeting a protein in cells called MEK and disrupting tumor growth.
Everolimus is a drug that may... expand
This phase I trial studies the side effects and best dose of trametinib and everolimus in treating pediatric and young adult patients with gliomas that have come back (recurrent). Trametinib acts by targeting a protein in cells called MEK and disrupting tumor growth. Everolimus is a drug that may block another pathway in tumor cells that can help tumors grow. Giving trametinib and everolimus may work better to treat low and high grade gliomas compared to trametinib or everolimus alone. Type: Interventional Start Date: Dec 2020 |
Hospital-Based Cluster Trial: Magnetically Controlled Growing Rods Using Distraction Intervals
Pediatric Spine Foundation
Early-Onset Scoliosis Deformity of Spine
A hospital-based cluster stratified randomization control study will be conducted to
investigate spinal growth in Early Onset Scoliosis patients between 5 and 9 years of age.
Patients must have a major coronal curve measuring over 50 degrees and be undergoing
Magnetically Controlled Growing Rod treatment.... expand
A hospital-based cluster stratified randomization control study will be conducted to investigate spinal growth in Early Onset Scoliosis patients between 5 and 9 years of age. Patients must have a major coronal curve measuring over 50 degrees and be undergoing Magnetically Controlled Growing Rod treatment. We will be studying 6-week lengthening intervals compared to 16-week lengthening intervals on spinal growth within 3 years. Type: Interventional Start Date: Nov 2019 |
CAB-AXL-ADC Safety and Efficacy Study in Adult and Adolescent Patients With Sarcoma
BioAtla, Inc.
Undifferentiated Pleomorphic Sarcoma
Myxofibrosarcoma
The objective of this study is to assess the safety and efficacy of mecbotamab vedotin
(BA3011) in solid tumors. expand
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 |
Long-Term Follow-Up of Patients Who Have Participated in Children's Oncology Group Studies
Children's Oncology Group
Hematopoietic Cell Transplantation Recipient
Leukemia
Solid Tumor
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... expand
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 |
Neonatal Seizure Registry, GEnetics of Post-Neonatal Epilepsy
University of California, San Francisco
Neonatal Seizure
Hypoxic-Ischemic Encephalopathy
Stroke
Intracranial Hemorrhage
Epilepsy
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.... expand
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 |
Focused Ultrasound Pallidotomy for Dyskinetic Cerebral Palsy in Pediatric and Young Adult Subjects
Children's National Research Institute
Cerebral Palsy, Dyskinetic
Secondary Dystonia
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 expand
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 |
Acute Concussion and Melatonin
Children's National Research Institute
Concussion, Mild
Concussion, Brain
Pediatric ALL
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... expand
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 |
Longitudinal Study of Urea Cycle Disorders
Andrea Gropman
Brain Diseases, Metabolic, Inborn
Amino Acid Metabolism, Inborn Errors
Urea Cycle Disorders
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... expand
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 |
An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics
Recordati Rare Diseases
Hyperammonemia
Methylmalonic Acidemia
Propionic Acidemia
To obtain short-term and long-term clinical safety information, in pediatric and adult
patients with PA and MMA treated with Carbaglu®. expand
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 |
A Study to Evaluate DAY101 in Pediatric and Young Adult Patients With Relapsed or Progressive Low-Grade...
Day One Biopharmaceuticals, Inc.
Low-grade Glioma
Advanced Solid Tumor
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. expand
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 |
Health Information Technology to Reduce Disparities in Adolescent Health Outcomes: A Pragmatic Trial
Children's National Research Institute
Sexually Transmitted Diseases
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... expand
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 |
Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease
Robert Nickel
Sickle Cell Disease
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... expand
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 |
Blood Brain Barrier (BBB) Disruption Using Exablate Focused Ultrasound With Doxorubicin for Treatment...
InSightec
Brain Tumor
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 expand
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 |
Multi Tumor-Associated Antigen-Specific T Lymphocytes to Treat Patients With High Risk Solid Tumors
Children's National Research Institute
Solid Tumor
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... expand
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 |
Conditioning SCID Infants Diagnosed Early
Michael Pulsipher, MD
SCID
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... expand
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 |
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Cumberland Pharmaceuticals
Duchenne Muscular Dystrophy Cardiomyopathy
Cardiomyopathy, Dilated
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss
of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any
age, and inevitably premature death of affected young men in their late twenties. DMD is
the most common fatal genetic disorder... expand
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and inevitably premature death of affected young men in their late twenties. DMD is the most common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every 3,500 live male births across all races and cultures, and results in 20,000 new cases each year worldwide.Significant advances in respiratory care have unmasked CM as the leading cause of death. As there are yet no specific cardiac treatments to extend life, the current study aims to address this unmet medical need using a new therapeutic strategy for patients with DMD. Funding Source - FDA OOPD Type: Interventional Start Date: Oct 2020 |
Helping Toddlers and Parents Together
University of Maryland, College Park
ADHD
Parenting
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... expand
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 |
Omalizumab Before Onset of Exacerbations
Stephen J. Teach, MD, MPH
Asthma in Children
Atopy
Viral Upper Respiratory Infection
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.... expand
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 |
- Previous
- Next