Search Clinical Trials
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A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Center for International Blood and Marrow Transplant Research
Hematologic Malignancies
Inherited Disorders of Metabolism
Inherited Abnormalities of Platelets
Histiocytic Disorders
Acute Myelogenous Leukemia (AML or ANLL)
This study is an access and distribution protocol for unlicensed cryopreserved cord blood
units (CBUs) in pediatric and adult patients with hematologic malignancies and other
indications. expand
This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications. Type: Observational Start Date: Oct 2011 |
ML-004 in Adolescents and Adults with Autism Spectrum Disorders (ASD)
MapLight Therapeutics
Autism Spectrum Disorder
ML-004-002 is a multi-center, randomized, double-blind, parallel-group,
placebo-controlled study that will enroll approximately 150 adolescent and adult subjects
with ASD. The primary objective is to evaluate the efficacy of ML-004 compared with
placebo in the improvement of social communication deficits... expand
ML-004-002 is a multi-center, randomized, double-blind, parallel-group, placebo-controlled study that will enroll approximately 150 adolescent and adult subjects with ASD. The primary objective is to evaluate the efficacy of ML-004 compared with placebo in the improvement of social communication deficits in subjects with ASD. Type: Interventional Start Date: Sep 2022 |
Flavored, Oral Irinotecan VAL-413 (Orotecan®) Given With Temozolomide for Treatment of Recurrent Pediatric...
Valent Technologies, LLC
Solid Tumors
Neuroblastoma
Rhabdomyosarcoma
Ewing Sarcoma
Hepatoblastoma
A pilot pharmacokinetic trial to determine the safety and efficacy of a flavored, orally
administered irinotecan VAL-413 (Orotecan®) given with temozolomide for treatment of
recurrent pediatric solid tumors including but not limited to neuroblastoma,
rhabdomyosarcoma, Ewing sarcoma, hepatoblastoma... expand
A pilot pharmacokinetic trial to determine the safety and efficacy of a flavored, orally administered irinotecan VAL-413 (Orotecan®) given with temozolomide for treatment of recurrent pediatric solid tumors including but not limited to neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, hepatoblastoma and medulloblastoma Type: Interventional Start Date: Oct 2021 |
Prospective Observational Cohort Study of Fetal Atrial Flutter & Supraventricular Tachycardia
Edgar Jaeggi
Atrial Flutter
Tachycardia, Supraventricular
Tachycardia, Atrial Ectopic
Tachycardia, Reciprocating
Tachycardia Atrial
The FAST Trial Registry is a prospective observational cohort study of fetuses with a new
diagnosis of atrial flutter (AF) or supraventricular tachycardia (SVT) that is severe
enough to consider prenatal treatment (see eligibility criteria below). Aims of the
Registry include to establish a large... expand
The FAST Trial Registry is a prospective observational cohort study of fetuses with a new diagnosis of atrial flutter (AF) or supraventricular tachycardia (SVT) that is severe enough to consider prenatal treatment (see eligibility criteria below). Aims of the Registry include to establish a large clinical database to determine and compare the efficacy and safety of different prenatal treatment strategies including observation without immediate treatment, transplacental antiarrhythmic fetal treatment and direct fetal treatment from the time of tachycardia diagnosis to death, neonatal hospital discharge or to a maximum of 30 days after birth. Type: Observational Start Date: Jun 2017 |
Liver Disease in Urea Cycle Disorders
Baylor College of Medicine
Urea Cycle Disorder
Ornithine Transcarbamylase Deficiency
Citrullinemia 1
ARGI Deficiency
ASL Deficiency
This is a multi-center, cross-sectional study to assess risk for liver fibrosis and
hepatic injury in individuals with urea cycle disorders (UCDs) using serum biomarkers,
Fibroscan, and MRE. This study will be conducted at 5 sites of the Urea Cycle Disorders
Consortium: Baylor College of Medicine... expand
This is a multi-center, cross-sectional study to assess risk for liver fibrosis and hepatic injury in individuals with urea cycle disorders (UCDs) using serum biomarkers, Fibroscan, and MRE. This study will be conducted at 5 sites of the Urea Cycle Disorders Consortium: Baylor College of Medicine in Houston, TX, Seattle Children's Hospital in Seattle, WA, Children's Hospital Colorado in Aurora, CO, Children's Hospital of Philadelphia in Philadelphia, PA, and Children's National Medical Center in Washington D.C. Type: Observational Start Date: Nov 2021 |
A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With...
Pediatric Brain Tumor Consortium
Low Grade Glioma (LGG) of Brain With BRAF Aberration
High Grade Glioma (HGG) of the Brain With BRAF Aberration
Low Grade Glioma of Brain With Neurofibromatosis Type 1
This phase I/II trial is designed to study the side effects, best dose and efficacy of
adding hydroxychloroquine to dabrafenib and/or trametinib in children with low grade or
high grade brain tumors previously treated with similar drugs that did not respond
completely (progressive) or tumors that... expand
This phase I/II trial is designed to study the side effects, best dose and efficacy of adding hydroxychloroquine to dabrafenib and/or trametinib in children with low grade or high grade brain tumors previously treated with similar drugs that did not respond completely (progressive) or tumors that came back while receiving a similar agent (recurrent). Patients must also have specific genetic mutations including BRAF V600 mutations or BRAF fusion/duplication, with or without neurofibromatosis type 1. Neurofibromatosis type 1 is an inherited genetic condition that causes tumors to grow on nerve tissue. Hydroxychloroquine, works in different ways to stop the growth of tumor cells by killing the cells or stopping them from dividing. Trametinib and dabrafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving hydroxychloroquine with trametinib and/or dabrafenib may lower the chance of brain tumors growing or spreading compared to usual treatments. Type: Interventional Start Date: Jan 2020 |
Dabrafenib Combined With Trametinib After Radiation Therapy in Treating Patients With Newly-Diagnosed...
National Cancer Institute (NCI)
Anaplastic Astrocytoma
Anaplastic Astrocytoma, Not Otherwise Specified
Anaplastic Ganglioglioma
Anaplastic Pleomorphic Xanthoastrocytoma
Glioblastoma
This phase II trial studies how well the combination of dabrafenib and trametinib works
after radiation therapy in children and young adults with high grade glioma who have a
genetic change called BRAF V600 mutation. Radiation therapy uses high energy rays to kill
tumor cells and reduce the size of... expand
This phase II trial studies how well the combination of dabrafenib and trametinib works after radiation therapy in children and young adults with high grade glioma who have a genetic change called BRAF V600 mutation. Radiation therapy uses high energy rays to kill tumor cells and reduce the size of tumors. Dabrafenib and trametinib may stop the growth of tumor cells by blocking BRAF and MEK, respectively, which are enzymes that tumor cells need for their growth. Giving dabrafenib with trametinib after radiation therapy may work better than treatments used in the past in patients with newly-diagnosed BRAF V600-mutant high-grade glioma. Type: Interventional Start Date: Feb 2020 |
A Phase II Trial of Poly-ICLC for Low-Grade Gliomas
University of Alabama at Birmingham
NF1
Low-grade Glioma
This is a phase II, prospective, longitudinal, multi-center trial of poly-ICLC (Hiltonol
®) treatment for progressive low-grade gliomas in pediatric patients with NF1. The
primary objective is to evaluate the efficacy of poly-ICLC in pediatric NF1 patients with
progressive low-grade glioma (LGG) as... expand
This is a phase II, prospective, longitudinal, multi-center trial of poly-ICLC (Hiltonol ®) treatment for progressive low-grade gliomas in pediatric patients with NF1. The primary objective is to evaluate the efficacy of poly-ICLC in pediatric NF1 patients with progressive low-grade glioma (LGG) as measured by objective tumor response rate (CR+PR) within the first 48 weeks (12 cycles) of therapy. There will also be secondary and exploratory objectives listed in the detailed description below. Type: Interventional Start Date: Dec 2021 |
Safety Study of Unlicensed IND Cord Blood Units Manufactured by the National Cord Blood Program for Unrelated...
New York Blood Center
Infusion Reactions
This study will evaluate the safety of infusion of the investigational cord blood units
by carefully documenting all infusion-related problems. expand
This study will evaluate the safety of infusion of the investigational cord blood units by carefully documenting all infusion-related problems. Type: Interventional Start Date: Feb 2012 |
Therapeutic Endpoint in Pediatric IBD Conditions
Children's National Research Institute
Inflammatory Bowel Diseases
Colitis, Ulcerative
Crohn Disease
The purpose of this clinical study is the development of physiologic endpoint of
inflammation in pediatric patients diagnosed with inflammatory bowel disease (IBD),
specifically subtypes Crohn's disease (CD) and ulcerative colitis (UC). The novel medical
device evaluates the patient's sensory response... expand
The purpose of this clinical study is the development of physiologic endpoint of inflammation in pediatric patients diagnosed with inflammatory bowel disease (IBD), specifically subtypes Crohn's disease (CD) and ulcerative colitis (UC). The novel medical device evaluates the patient's sensory response to each of the three sensory nerve fiber types. Data from the device provides an assessment of disease activity and a more precise approach to treatment. Type: Observational Start Date: Nov 2023 |
Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas
Recursion Pharmaceuticals Inc.
Neurofibromatosis Type 2
This is a two-staged, Phase 2/3, randomized, multi-center study to investigate the
efficacy and safety of REC-2282 in patients with progressive NF2 mutated meningiomas. expand
This is a two-staged, Phase 2/3, randomized, multi-center study to investigate the efficacy and safety of REC-2282 in patients with progressive NF2 mutated meningiomas. Type: Interventional Start Date: Jun 2022 |
Development and Testing of a Pediatric Cervical Spine Injury Risk Assessment Tool
Julie Leonard
Cervical Spine Injury
Cervical spine injuries (CSI) are serious, but rare events in children. Spinal
precautions (rigid cervical collar and immobilization on a longboard) in the prehospital
setting may be beneficial for children with CSI, but are poorly studied. In contrast,
spinal precautions for pediatric trauma patients... expand
Cervical spine injuries (CSI) are serious, but rare events in children. Spinal precautions (rigid cervical collar and immobilization on a longboard) in the prehospital setting may be beneficial for children with CSI, but are poorly studied. In contrast, spinal precautions for pediatric trauma patients without CSI are common and may be associated with harm. Spinal precautions result in well-documented adverse physical and physiological sequelae. Of substantial concern is that the mere presence of prehospital spinal precautions may lead to a cascade of events that results in the increased use of inappropriate radiographic testing in the emergency department (ED) to evaluate children for CSI and thus an unnecessary, increased exposure to ionizing radiation and lifetime risk of cancer. Most children who receive spinal precautions and/or are imaged for potential CSI, and particularly those imaged with computed tomography (CT), are exposed to potential harm with no demonstrable benefit. Therefore, there is an urgent need to develop a Pediatric CSI Risk Assessment Tool that can be used in the prehospital and ED settings to reduce the number of children who receive prehospital spinal precautions inappropriately and are imaged unnecessarily while identifying all children who are truly at risk for CSI. Type: Observational [Patient Registry] Start Date: Dec 2018 |
Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised Hosts
Children's National Research Institute
Viral Infection
Hematopoietic Stem Cell Transplantation (HSCT)
Primary Immunodeficiency Disorders (PID)
This is a Phase I dose-escalation study to evaluate the safety of norovirus -specific
T-cell (NST) therapy for chronic norovirus infection in participants following
hematopoietic stem cell transplantation (HSCT) or with primary immunodeficiency disorders
(PID) who have not undergone HSCT. expand
This is a Phase I dose-escalation study to evaluate the safety of norovirus -specific T-cell (NST) therapy for chronic norovirus infection in participants following hematopoietic stem cell transplantation (HSCT) or with primary immunodeficiency disorders (PID) who have not undergone HSCT. Type: Interventional Start Date: Mar 2022 |
Safety and Efficacy of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML), a...
Daiichi Sankyo
Acute Myeloid Leukemia
Quizartinib is an experimental drug. It is not approved for regular use. It can only be
used in medical research.
Children or young adults with a certain kind of blood cancer (FLT3-ITD AML) might be able
to join this study if it has come back after remission or is not responding to treatment. expand
Quizartinib is an experimental drug. It is not approved for regular use. It can only be used in medical research. Children or young adults with a certain kind of blood cancer (FLT3-ITD AML) might be able to join this study if it has come back after remission or is not responding to treatment. Type: Interventional Start Date: Aug 2018 |
Outcomes and Health Care Resource Utilization in Pediatric Congenital Heart Disease Patients Undergoing...
Boston Children's Hospital
Congenital Heart Disease in Children
The incidence of moderate to severe congenital heart disease (CHD) in the United States
is estimated to be 6 per 1000 live-born full term infants. Recent advances in pediatric
cardiology, surgery and critical care have significantly improved the survival rates of
patients with CHD leading to an increase... expand
The incidence of moderate to severe congenital heart disease (CHD) in the United States is estimated to be 6 per 1000 live-born full term infants. Recent advances in pediatric cardiology, surgery and critical care have significantly improved the survival rates of patients with CHD leading to an increase in prevalence in both children and adults. Children with CHD significant enough to require cardiac surgery frequently also undergo non-cardiac surgical procedures. Analysis of the Pediatric Health Information System database between 2004 and 2012 demonstrated that 41% of children who had undergone surgery to correct CHD in the first year of life also underwent at least one non-cardiac surgery by age 5. With this increased demand for non-cardiac procedures, anesthesiologists, pediatricians and other healthcare providers will encounter patients with repaired or unrepaired CHD and other cardiac diseases in their practice. However, the information provided by national databases lack granularity and the information from single institutional data is limited. This project aims to address this knowledge gap in quantifying the risk for cardiac patients coming for noncardiac procedures and identify the health care resource utilization and system to best care for this patient population. To conduct this study, we will create a multi-institutional collaboration between large and small centers to create a unique dataset spanning all the different variables that need to be considered in risk prediction for these patients including patient variables, hospital setting, and providers. The aggregate multiinstitutional data set may be used for benchmarking for national quality improvement efforts. Type: Observational [Patient Registry] Start Date: Oct 2020 |
Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation
Emory University
Sickle Cell Disease
This study aims to enroll 58 pre-adolescent (<13 years) pediatric participants with
sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor. All
participants will go through a pre-transplant evaluation to find out if there are health
problems that will keep them from being able... expand
This study aims to enroll 58 pre-adolescent (<13 years) pediatric participants with sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor. All participants will go through a pre-transplant evaluation to find out if there are health problems that will keep them from being able to receive the transplant. It usually takes 2 to 3 months to complete the pre-transplant evaluation and make the arrangements for the transplant. Once they are found to be eligible for transplant, participants will be admitted to the hospital and will start transplant conditioning. Conditioning is the chemotherapy and other medicines given to prepare them to receive donor cells. It prevents the immune system from rejecting donor cells. Conditioning will start 21 days before transplant. Once they complete conditioning, participants will receive the bone marrow transplant. After the transplant, participants will stay in the hospital for 4-6 weeks. After they leave the hospital, participants will be followed closely in the clinic. Outpatient treatment and frequent clinic visits usually last 6 to 12 months. Routine medical care includes at least a yearly examination for many years after transplant by doctors and nurses familiar with sickle cell disease and transplant. The researchers will collect and study information about participants for 2 years after transplant. Type: Interventional Start Date: Mar 2019 |
BBD Longitudinal Study of Osteogenesis Imperfecta
Baylor College of Medicine
Osteogenesis Imperfecta
Osteogenesis Imperfecta (OI) is a rare disorder of increased bone fragility characterized
by fractures with minimal or absent trauma, dentinogenesis imperfecta (DI), and, in adult
years, hearing loss. It is seen in both genders and all races. The clinical features of
OI represent a continuum varying... expand
Osteogenesis Imperfecta (OI) is a rare disorder of increased bone fragility characterized by fractures with minimal or absent trauma, dentinogenesis imperfecta (DI), and, in adult years, hearing loss. It is seen in both genders and all races. The clinical features of OI represent a continuum varying from perinatal lethality to individuals with severe skeletal deformities, mobility impairments, and very short stature to nearly asymptomatic individuals with a mild predisposition to fractures, normal stature, and normal lifespan. Fractures can occur in any bone, but are most common in the extremities. These disorders can be devastating and progressive and result in deformity, chronic pain, impaired function and loss of quality of life. The overall goal of this study is to answer specific question about the natural history of brittle bone diseases as defined by molecular etiology and to develop the foundation for prospective clinical studies. Type: Observational Start Date: Jun 2015 |
A Study to See if Tolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than...
Otsuka Pharmaceutical Development & Commercialization, Inc.
Autosomal Recessive Polycystic Kidney (ARPKD)
The primary objective of this study is to evaluate the safety of tolvaptan in pediatric
subjects with autosomal recessive polycystic kidney disease (ARPKD) expand
The primary objective of this study is to evaluate the safety of tolvaptan in pediatric subjects with autosomal recessive polycystic kidney disease (ARPKD) Type: Interventional Start Date: Jul 2022 |
Nerivio Device for Treatment of New Daily Headache Persistence (NDHP)
Children's National Research Institute
New Daily Persistent Headache (NDPH)
The goal of this study is to examine the effects of the Remote Electrical Neuromodulation
(REN) device on adolescents ages 12-17 who have been diagnosed with New Daily Persistent
Headache (NDPH). Pediatric patients with a diagnosis of new daily persistent headache are
typically resistant to standard... expand
The goal of this study is to examine the effects of the Remote Electrical Neuromodulation (REN) device on adolescents ages 12-17 who have been diagnosed with New Daily Persistent Headache (NDPH). Pediatric patients with a diagnosis of new daily persistent headache are typically resistant to standard pharmacologic treatments and often experience systemic side effects related to medications; thus, REN offers the potential for an exciting new treatment option for patients with refractory headache disorders. The device delivers transcutaneous electrical stimulation to the upper arm to induce conditioned pain modulation (CPM) that activates a descending endogenous analgesic mechanism. Ultimately, the investigators hope to gain insights into the safety and efficacy of Nerivio™ for the acute treatment of NDPH in adolescents. The goal of this study is to demonstrate headache relief without unexpected device-related adverse effects Type: Interventional Start Date: Feb 2022 |
Intravenous L-Citrulline for Vaso-occlusive Pain Episode in Sickle Cell Disease
Suvankar Majumdar
Sickle Cell Disease
Vaso-occlusive Pain Episode
The goal of this clinical trial is to learn if intravenous citrulline works to treat
acute pain in hospitalized patients with sickle cell disease. It will also learn about
the safety of intravenous citrulline. The main questions it aims to answer are:
- Does intravenous citrulline decrease the... expand
The goal of this clinical trial is to learn if intravenous citrulline works to treat acute pain in hospitalized patients with sickle cell disease. It will also learn about the safety of intravenous citrulline. The main questions it aims to answer are: - Does intravenous citrulline decrease the duration of sickle cell pain during hospitalization - What medical problems do participants have when taking intravenous citrulline? Researchers will compare intravenous citrulline to a placebo (a look-alike substance that contains no drug) to see if intravenous citrulline works to treat acute pain. Participants will: - Receive baseline tests and intravenous citrulline for 16 hours during the hospital stay - After hospital discharge, visit the clinic in about 30 days for checkup and tests Type: Interventional Start Date: Nov 2024 |
Volitinib in Treating Patients With Recurrent or Refractory Primary CNS Tumors
National Cancer Institute (NCI)
Recurrent Diffuse Intrinsic Pontine Glioma
Recurrent Malignant Glioma
Recurrent Medulloblastoma
Recurrent Primary Central Nervous System Neoplasm
Refractory Diffuse Intrinsic Pontine Glioma
This phase I trial studies the side effects and best dose of volitinib in treating
patients with primary central nervous system (CNS) tumors that have come back (recurrent)
or does not respond to treatment (refractory). Volitinib may stop the growth of tumor
cells by blocking some of the enzymes needed... expand
This phase I trial studies the side effects and best dose of volitinib in treating patients with primary central nervous system (CNS) tumors that have come back (recurrent) or does not respond to treatment (refractory). Volitinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Type: Interventional Start Date: Nov 2018 |
Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases...
National Human Genome Research Institute (NHGRI)
Genetic Disease
Without an explanation for severe and sometimes life-threatening symptoms, patients and
their families are left in a state of unknown. Many individuals find themselves being
passed from physician to physician, undergoing countless and often repetitive tests in
the hopes of finding answers and insight... expand
Without an explanation for severe and sometimes life-threatening symptoms, patients and their families are left in a state of unknown. Many individuals find themselves being passed from physician to physician, undergoing countless and often repetitive tests in the hopes of finding answers and insight about what the future may hold. This long and arduous journey to find a diagnosis does not end for many patients- the Office of Rare Diseases Research (ORDR) notes that 6% of individuals seeking their assistance have an undiagnosed disorder. In 2008, the National Institutes of Health (NIH) Undiagnosed Diseases Program (UDP) was established with the goal of providing care and answers for these individuals with mysterious conditions who have long eluded diagnosis. The NIH UDP is a joint venture of the NIH ORDR, the National Human Genome Research Institute Intramural Research Program (NHGRI-IRP), and the NIH Clinical Research Center (CRC) (1-3). The goals of the NIH UDP are to: (1) provide answers for patients with undiagnosed diseases; (2) generate new knowledge about disease mechanisms; (3) assess the application of new approaches to phenotyping and the use of genomic technologies; and (4) identify potential therapeutic targets, if possible. To date, the UDP has evaluated 3300 medical records and admitted 750 individuals with rare and undiagnosed conditions to the NIH Clinical Center. The NIH UDP has identified more than 70 rare disease diagnoses and several new conditions. The success of the NIH UDP prompted the NIH Common Fund to support the establishment of a network of medical research centers, the Undiagnosed Diseases Network (UDN), for fiscal years 2013-2020. The clinical sites will perform extensive phenotyping, genetic analyses, and functional studies of potential disease-causing variants. The testing performed on patients involves medically indicated studies intended to help reach a diagnosis, as well as research investigations that include a skin biopsy, blood draws, and DNA analysis. In addition, the UDN will further the goals of the UDP by permitting the sharing of personally identifiable phenotypic and genotypic information within the network. By sharing participant information and encouraging collaboration, the UDN hopes to improve the understanding of rare conditions and advance the diagnostic process and care for individuals with undiagnosed diseases.... Type: Observational Start Date: Sep 2015 |
Clinical and Laboratory Study of Methylmalonic Acidemia
National Human Genome Research Institute (NHGRI)
Organic Acidemia
Methylmalonic Acidemia
Inborn Errors of Metabolism
Methylmalonic acidemia (MMA), one of the most common inborn errors of organic acid
metabolism, is heterogeneous in etiology and clinical manifestations. Affected patients
with cblA, cblB and mut classes of MMA are medically fragile and can suffer from
complications such as metabolic stroke or infarction... expand
Methylmalonic acidemia (MMA), one of the most common inborn errors of organic acid metabolism, is heterogeneous in etiology and clinical manifestations. Affected patients with cblA, cblB and mut classes of MMA are medically fragile and can suffer from complications such as metabolic stroke or infarction of the basal ganglia, pancreatitis, end stage renal failure, growth impairment, osteoporosis, and developmental delay. The frequency of these complications and their precipitants remain undefined. Furthermore, current treatment protocol outcomes have continued to demonstrate substantial morbidity and mortality in the patient population. Increasingly, solid organ transplantation (liver, and/or kidney) has been used to treat patients. Disordered transport and intracellular metabolism of vitamin B12 produces a distinct group of disorders that feature methylmalonic acidemia as well as (hyper)homocysteinemia. These conditions are named after the corresponding cellular complementation class (cblC, cblD, cblF, cblJ and cblX) and are also heterogenous, clinically and biochemically. The genetic disorders underlying cblE and cblG feature an isolated impairment of the activity of methionine synthase, a critical enzyme involved in the conversion of homocysteine to methionine and these disorders feature (hyper)homocysteinemia. Lastly, a group of patients can have increased methylmalonic acid and/or homocysteine in the blood or urine caused by variant(s)in recently identified (ACSF3) and unknown genes. In this protocol, we will clinically evaluate patients with methylmalonic acidemia and cobalamin metabolic defects. Routine inpatient admissions will last up to 4-5 days and involve urine collection, blood drawing, ophthalmological examination, radiological procedures, MRI/MRS, skin biopsies in some, and developmental testing. In a subset of patients who have or will receive renal, hepato- or hepato-renal transplants or have an unusual variant or clinical course and have MMA, a lumbar puncture to examine CSF metabolites will be performed. In this small group of patients, CSF metabolite monitoring may be used to adjust therapy. The study objectives will be to further delineate the spectrum of phenotypes and characterize the natural history of these enzymopathies, query for genotype/enzymatic/phenotype correlations, search for new genetic causes of methylmalonic acidemia and/or homocysteinemia, identify new disease biomarkers and define clinical outcome parameters for future clinical trials. The population will consist of participants previously evaluated at NIH, physician referrals, and families directed to the study from clinicaltrials.gov as well as the Organic Acidemia Association, Homocystinuria Network America and other national and international support groups. Most participants will be evaluated only at the NIH Clinical Center. However, if the NIH team decides that a patient under the age of 2 years is a candidate subject for this research protocol, that patient may enroll at the Children s National Medical Center (CNMC) site, pending approval by Dr Chapman, the Principal Investigator of the CNMC location Individuals may also enroll in the tissue collection only part of the study at the UPMC Children s Hospital of Pittsburgh or share medical history and clinical data via telemedicine visits remotely. Outcome measures will largely be descriptive and encompass correlations between clinical, biochemical and molecular parameters. Type: Observational Start Date: Jun 2004 |
Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT)
Children's National Research Institute
Medulloblastoma, Childhood
Atypical Teratoid/Rhabdoid Tumor of CNS
Embryonal Tumor With Multilayered Rosettes
Pineoblastoma
Embryonal Tumor of CNS
This is an open-label phase 1 safety and feasibility study that will employ multi-tumor
antigen specific cytotoxic T lymphocytes (TSA-T) directed against proteogenomically
determined personalized tumor-specific antigens (TSA) derived from a patient's primary
brain tumor tissues. Young patients with... expand
This is an open-label phase 1 safety and feasibility study that will employ multi-tumor antigen specific cytotoxic T lymphocytes (TSA-T) directed against proteogenomically determined personalized tumor-specific antigens (TSA) derived from a patient's primary brain tumor tissues. Young patients with embryonal central nervous system (CNS) malignancies typically are unable to receive irradiation due to significant adverse effects and are treated with intensive chemotherapy followed by autologous stem cell rescue; however, despite intensive therapy, many of these patients relapse. In this study, individualized TSA-T cells will be generated against proteogenomically determined tumor-specific antigens after standard of care treatment in children less than 5 years of age with embryonal brain tumors. Correlative biological studies will measure clinical anti-tumor, immunological and biomarker effects. Type: Interventional Start Date: Sep 2024 |
A Master Protocol (LY900023) That Includes Several Clinical Trials of Drugs for Children and Young Adults...
Eli Lilly and Company
Neoplasms
Child
Adolescent
The main purpose of the master is to help the research sites and sponsor carry out
several clinical trials more efficiently by providing a common research protocol.
Individual clinical trials under this master protocol define drug/disease-specific
research goals and activities to test them. New studies... expand
The main purpose of the master is to help the research sites and sponsor carry out several clinical trials more efficiently by providing a common research protocol. Individual clinical trials under this master protocol define drug/disease-specific research goals and activities to test them. New studies will be added as new drugs emerge against different cancers. Participation in the trial will depend on how long the benefit lasts. Type: Interventional Start Date: Jan 2020 |
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