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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®.
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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... 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... 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... 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
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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 |
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... 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 < 23 years will be eligible to participate. Type: Interventional Start Date: Sep 2021 |
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... 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 |
Highest Dose of Uproleselan in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid...
National Cancer Institute (NCI)
Acute Myeloid Leukemia Post Cytotoxic Therapy
Down Syndrome
Myelodysplastic Syndrome Post Cytotoxic Therapy
Recurrent Acute Myeloid Leukemia
Recurrent Mixed Phenotype Acute Leukemia
This phase I trial tests the safety, side effects, and best dose of uproleselan in
combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia,
myelodysplastic syndrome or mixed phenotype acute leukemia that has come back (relapsed) or
does... expand
This phase I trial tests the safety, side effects, and best dose of uproleselan in combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that has come back (relapsed) or does not respond to treatment (refractory) and that expresses E-selectin ligand on the cell membrane. Uproleselan binds to E-selectin expressed on endothelial cells of the bone marrow and prevents their interaction with selectin-E ligand-expressing cancer cells. This may prevent leukemia cells from being sequestered in the bone marrow niche and escaping the effect of chemotherapy. Chemotherapy drugs, such as fludarabine and cytarabine, 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. Giving uproleselan in combination with fludarabine and cytarabine may enhance their activity. Type: Interventional Start Date: Dec 2023 |
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... 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... 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 |
Feasibility/Acceptability of Attentional-Control Training in Survivors
Children's National Research Institute
Pediatric Cancer
Pediatric ALL
Pediatric Brain Tumor
Attention Difficulties
Cognitive Deficit in Attention
This is a multicenter pilot randomized controlled trial, with an active control condition, of
the feasibility, acceptability, and preliminary efficacy of EndeavorRx in a cohort of
survivors of acute lymphoblastic leukemia or brain tumor ages 8-16 who are > 1 year from the... expand
This is a multicenter pilot randomized controlled trial, with an active control condition, of the feasibility, acceptability, and preliminary efficacy of EndeavorRx in a cohort of survivors of acute lymphoblastic leukemia or brain tumor ages 8-16 who are > 1 year from the end of therapy. Type: Interventional Start Date: Jun 2023 |
Registry of Asthma Characterization and Recruitment 3 (RACR3)
National Institute of Allergy and Infectious Diseases (NIAID)
Asthma
This is a multi-center, non-interventional registry to create and maintain a database of
participants to serve as a recruitment source for current and future DAIT NIAID-sponsored
Childhood Asthma in Urban Settings (CAUSE) studies.
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This is a multi-center, non-interventional registry to create and maintain a database of participants to serve as a recruitment source for current and future DAIT NIAID-sponsored Childhood Asthma in Urban Settings (CAUSE) studies. Type: Observational [Patient Registry] Start Date: Apr 2022 |
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).
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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 |
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... 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... 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 |
Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders
Carl Allen
Langerhan's Cell Histiocytosis
Juvenile Xanthogranuloma
Erdheim-Chester Disease
Rosai Dorfman Disease
Neuro-Degenerative Disease
This is a research study of a drug called cobimetinib in children and adults diagnosed with
Langerhans cell histiocytosis (LCH), and other histiocytic disorders that has returned or
does not respond to treatment. Cobimetinib blocks activation of a protein called
Mitogen-activated... expand
This is a research study of a drug called cobimetinib in children and adults diagnosed with Langerhans cell histiocytosis (LCH), and other histiocytic disorders that has returned or does not respond to treatment. Cobimetinib blocks activation of a protein called Mitogen-activated protein kinase (MEK) that is part of incorrect growth signals in histiocytosis cells. Four different groups of patients will be enrolled. Type: Interventional Start Date: Apr 2021 |
LCH-IV, International Collaborative Treatment Protocol for Children and Adolescents With Langerhans Cell...
North American Consortium for Histiocytosis
Langerhans Cell Histiocytosis
The LCH-IV is an international, multicenter, prospective clinical study for pediatric
Langerhans Cell Histiocytosis LCH (age < 18 years).
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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 |
Comparison of Methods of Pulmonary Blood Flow Augmentation in Neonates: Shunt Versus Stent (The COMPASS...
Carelon Research
Congenital Heart Disease in Children
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... expand
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 |
GM-CSF for Reversal of Immunoparalysis in Pediatric Sepsis-induced MODS
Nationwide Children's Hospital
Pediatric Sepsis-induced Multiple Organ Dysfunction Syndrome
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.
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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 |
Technology-Enhanced Executive Functioning Intervention for ADHD
Children's National Research Institute
ADHD
Attention Deficit Hyperactivity Disorder
Attention Deficit Disorder
ADD
ADHD Predominantly Inattentive Type
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... expand
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 |
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... 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 |
Molecular Analysis of Samples From Patients With Diffuse Intrinsic Pontine Glioma and Brainstem Glioma
Children's National Research Institute
Diffuse Intrinsic Pontine Glioma
Brainstem Glioma
The purpose of this study is to prospectively collect specimens from pediatric patients with
diffuse intrinsic pontine glioma or brainstem glioma, either during therapy or at autopsy, in
order to characterize the molecular abnormalities of this tumor.
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The purpose of this study is to prospectively collect specimens from pediatric patients with diffuse intrinsic pontine glioma or brainstem glioma, either during therapy or at autopsy, in order to characterize the molecular abnormalities of this tumor. Type: Observational Start Date: Apr 2010 |
Development of a Therapeutic Endpoint in Pediatric Rheumatologic Conditions
Children's National Research Institute
Juvenile Idiopathic Arthritis
Systemic Lupus Erythematosus
Fibromyalgia
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... expand
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. Type: Observational Start Date: Jul 2021 |
Digital Dysmorphology Project
Kevin Cleary
Down Syndrome
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... expand
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 |
EXCOR Active Driving System for the EXCOR Pediatric VAD IDE Study
Berlin Heart, Inc
Heart Failure
Transplant; Failure, Heart
Congenital Heart Disease
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... expand
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 |
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