169 matching studies

Sponsor Condition of Interest
Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome...
Children's Oncology Group Acute Myeloid Leukemia Down Syndrome Myelodysplastic Syndrome Myeloid Leukemia Associated With Down Syndrome Myeloproliferative Neoplasm
This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome. Drugs used in chemotherapy work in different ways to stop the growth of cancer cells, either by killing... expand

This phase III trial studies response-based chemotherapy in treating newly diagnosed acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome. Drugs used in chemotherapy 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. Response-based chemotherapy separates patients into different risk groups and treats them according to how they respond to the first course of treatment (Induction I). Response-based treatment may be effective in treating acute myeloid leukemia or myelodysplastic syndrome in younger patients with Down syndrome while reducing the side effects.

Type: Interventional

Start Date: Nov 2015

open study

Computer Training Program for Younger Patients With a Brain Tumor Who Underwent Radiation Therapy
Children's Oncology Group Brain Neoplasm Recurrent Brain Neoplasm
This randomized clinical trial studies how well an adaptive computerized cognitive training program works compared to a non-adaptive computerized cognitive training program in treating younger patients with brain tumor who underwent radiation therapy. Providing a computer training... expand

This randomized clinical trial studies how well an adaptive computerized cognitive training program works compared to a non-adaptive computerized cognitive training program in treating younger patients with brain tumor who underwent radiation therapy. Providing a computer training program may improve the well-being and quality of life of patients with cognitive (physical and mental) function difficulties caused by radiation therapy to the brain.

Type: Interventional

Start Date: Nov 2013

open study

Acupuncture for Pain in Sickle Cell Disease
Deepika Darbari Sickle Cell Disease
Sickle cell disease (SCD) is the most common genetic disorder in the United States affecting approximately 100,000 individuals primarily of African ancestry. Pain is the most common complication of SCD. Currently, the mainstay therapy for pain in SCD is opioids. The CDC recommends... expand

Sickle cell disease (SCD) is the most common genetic disorder in the United States affecting approximately 100,000 individuals primarily of African ancestry. Pain is the most common complication of SCD. Currently, the mainstay therapy for pain in SCD is opioids. The CDC recommends using non-opioid, non-pharmacologic therapies for pain. There is a growing body of literature to support the use of various integrative therapies for pain. Acupuncture therapy is a non-pharmacological Chinese medicine approach which has been used in many non-SCD conditions associated with pain. Proposed study will test acceptability and feasibility of use of acupuncture in SCD patients hospitalized for pain. It is hypothesized that the use of acupuncture as an adjuvant therapy will be acceptable to SCD patients admitted for pain control. Its impact on opioid use and circulating cytokines and neuropeptides will also be determined.

Type: Interventional

Start Date: Oct 2018

open study

Pediatric Red Reflex Study
Children's National Research Institute Abnormal Vitreous Humor Morphology Abnormal Choroid Morphology Optic Nerve Diseases Retinal Disease Retinal Hemorrhage
Our Pediatric Red Reflex Study is designed to investigate the sensitivity and specificity of the Red Reflex Test (RRT), with and without dilation, for early detection of ocular abnormalities in children and newborns. The RRT functions by shining a light from an ophthalmoscope... expand

Our Pediatric Red Reflex Study is designed to investigate the sensitivity and specificity of the Red Reflex Test (RRT), with and without dilation, for early detection of ocular abnormalities in children and newborns. The RRT functions by shining a light from an ophthalmoscope into a patient's eye and noting the presence or absence of a red glow. Despite its use in pediatric clinics for years, this test at times fails to detect significant ocular diseases, especially located in the back of the eye, threatening visual development in this population. Therefore, we aim to quantify the utility of this test as a tool for screening by comparing our findings on RRT with those of retinal photography. We hypothesize that the sensitivity and specificity of the RRT will be sufficient for detecting anterior segment pathology but will be insufficient for detecting posterior segment pathology with or without dilation.

Type: Observational

Start Date: Nov 2019

open study

The Genetic Basis of Congenital Heart Disease in Africa
National Human Genome Research Institute (NHGRI) Congenital Heart Disease Heart Disease
Recent advances in genomic techniques are making possible a new wave of genetic discovery in congenital heart disease (CHD). Existing data suggests that CHD occur in Sub-Saharan Africa at frequencies similar to the rest of the world. In this application, we propose to utilize... expand

Recent advances in genomic techniques are making possible a new wave of genetic discovery in congenital heart disease (CHD). Existing data suggests that CHD occur in Sub-Saharan Africa at frequencies similar to the rest of the world. In this application, we propose to utilize the unique advantages of Sub-Saharan Africa - a combination of the most genetically diverse populations in the world and of diminished environmental background effects (i.e. low prevalence of smoking, alcohol abuse, obesity in comparison to western countries) - to better understand the genetic basis for congenital heart disease. We will couple next generation genomic techniques with more traditional gene discovery methods to investigate CHD in two African countries: Uganda and Nigeria. The inclusion of syndromic and non-syndromic CHD observed in these populations as well as careful phenotyping (including echocardiography) will greatly enhance our potential to provide insight into the genetic architecture of CHD in African populations. To accomplish this, we plan to enroll families, in whom members have congenital heart malformations consistent with an error of early human development in our research protocol. Patients will be enrolled at the Uganda Heart Institute in Kampala, Uganda, and at the Department of Pediatrics, College of Medicine, University of Lagos, Nigeria, with the potential to include other African sites. High throughput genomic studies will be done at the NIH.

Type: Observational

Start Date: Sep 2013

open study

Cisplatin and Combination Chemotherapy in Treating Children and Young Adults With Hepatoblastoma or Liver...
Children's Oncology Group Childhood Hepatocellular Carcinoma Childhood Malignant Liver Neoplasm Fibrolamellar Carcinoma Hepatoblastoma Hepatocellular Malignant Neoplasm, Not Otherwise Specified
This partially randomized phase II/III trial studies how well, in combination with surgery, cisplatin and combination chemotherapy works in treating children and young adults with hepatoblastoma or hepatocellular carcinoma. Drugs used in chemotherapy, such as cisplatin, doxorubicin,... expand

This partially randomized phase II/III trial studies how well, in combination with surgery, cisplatin and combination chemotherapy works in treating children and young adults with hepatoblastoma or hepatocellular carcinoma. Drugs used in chemotherapy, such as cisplatin, doxorubicin, fluorouracil, vincristine sulfate, carboplatin, etoposide, irinotecan, sorafenib, gemcitabine and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving combination chemotherapy may kill more tumor cells than one type of chemotherapy alone.

Type: Interventional

Start Date: May 2018

open study

Feasibility Trial of Optune for Children With Recurrent or Progressive Supratentorial High-Grade Glioma...
Pediatric Brain Tumor Consortium Malignant Glioma Ependymoma
This feasibility trial studies how well the Optune device works in treating younger patients with recurrent/refractory/progressive supratentorial malignant glioma and ependymoma. The TTFields generated by Optune device may inhibit the growth of tumor. expand

This feasibility trial studies how well the Optune device works in treating younger patients with recurrent/refractory/progressive supratentorial malignant glioma and ependymoma. The TTFields generated by Optune device may inhibit the growth of tumor.

Type: Interventional

Start Date: Jan 2017

open study

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.... 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

open study

Tumor Associated Antigen Specific T Cells (TAA-T) With PD1 Inhibitor for Lymphoma
Catherine Bollard Hodgkin Lymphoma Diffuse Large B Cell Lymphoma
This is a Phase I, open-label multi-site trial designed to evaluate the safety and feasibility of administering rapidly-generated Tumor associated antigen specific T cells (TAA-T) with the Programmed Death1 (PD-1) inhibitor Nivolumab, in relapsed/refractory lymphoma (rel/ref)... expand

This is a Phase I, open-label multi-site trial designed to evaluate the safety and feasibility of administering rapidly-generated Tumor associated antigen specific T cells (TAA-T) with the Programmed Death1 (PD-1) inhibitor Nivolumab, in relapsed/refractory lymphoma (rel/ref) patients with measurable disease (group A) or as adjunctive therapy following autologous hematopoeitic stem cell transplant(HSCT) for patients at high risk of relapse (group B). The purpose of this study is to find out if the tumor specific T cells given with Nivolumab are safe and to learn what the side effects are and if the combination can help patients with relapsed lymphomas.

Type: Interventional

Start Date: Jun 2019

open study

Natural History Study of SCID Disorders
National Institute of Allergy and Infectious Diseases (NIAID) Severe Combined Immunodeficiency (SCID) Leaky SCID Omenn Syndrome Reticular Dysgenesis ADA SCID
This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine the patient, recipient and transplant-related variables that are most important... expand

This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions. In order to determine the patient, recipient and transplant-related variables that are most important in determining outcome, study investigators will uniformly collect pre-, post- and peri-transplant (or other treatment) information on all children enrolled into this study. Children will be divided into three strata: - Stratum A: Typical SCID with virtual absence of autologous T cells and poor T cell function - Stratum B: Atypical SCID (leaky SCID, Omenn syndrome and reticular dysgenesis with limited T cell diversity or number and reduced function), and - Stratum C: ADA deficient SCID and XSCID patients receiving alternative therapy including PEG-ADA ERT or gene therapy. Each Group/Cohort Stratum will be analyzed separately.

Type: Observational

Start Date: Sep 2010

open study

Dyslipidemia of Obesity Intervention in Teens
HealthCore-NERI Dyslipidemia Obesity
This trial of pitavastatin will determine efficacy and safety in this high risk population and provide evidence for clinicians to target this treatable risk factor to achieve an impact on early atherosclerosis, and potentially achieve primary prevention of adult cardiovascular... expand

This trial of pitavastatin will determine efficacy and safety in this high risk population and provide evidence for clinicians to target this treatable risk factor to achieve an impact on early atherosclerosis, and potentially achieve primary prevention of adult cardiovascular disease.

Type: Interventional

Start Date: May 2018

open study

Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
Association for Pediatric Pulmonary Hypertension Hypertension, Pulmonary
The TOPP-2 registry is an international, non-interventional, prospective registry including children and adolescents newly diagnosed with pulmonary hypertension (PH) to gain further insights in the disease course and long-term outcome of PH in childhood. Patients will undergo... expand

The TOPP-2 registry is an international, non-interventional, prospective registry including children and adolescents newly diagnosed with pulmonary hypertension (PH) to gain further insights in the disease course and long-term outcome of PH in childhood. Patients will undergo clinical assessments and receive standard medical care, as determined by treating physicians in their daily clinical practice. The TOPP-2 registry is specifically designed to capture the variables that have been proposed as treatment goals in PePH and the reasons for changes in treatment strategy. The TOPP-2 registry uses the new clinical classification of PH as outlined at the 5th World Symposium for Pulmonary Hypertension (WSPH) in Nice 2013 and includes new characterizations for children with PH. The registry is planned and implemented under the scientific leadership of the Association for Pediatric Pulmonary Hypertension (PePH), independently from the financial sponsors. At least 200 of the enrolled patients will have a follow-up period of 3 years.

Type: Observational [Patient Registry]

Start Date: Aug 2015

open study

Health Equity: Advance Care Planning for Spanish Speaking Teens With Cancer-1st Resubmission
Maureen Lyon Cancer
Pediatric ACP (Advance Care Planning) (pACP) (1) Supports communication with children/adolescents at any stage of a serious illness in understanding their illness, complications, fears, and hopes, as well as treatment preferences regarding future medical care with their family;... expand

Pediatric ACP (Advance Care Planning) (pACP) (1) Supports communication with children/adolescents at any stage of a serious illness in understanding their illness, complications, fears, and hopes, as well as treatment preferences regarding future medical care with their family; (2) Communicates these goals of care and treatment preferences with their physician; and (3) Documents these goals of care and end-of-life treatment preferences. Among adolescents, cancer is the leading cause of disease-related deaths. Survival has improved far less for 15 to 24 year-olds than it has for older patients. About one-fourth will die prematurely. No pediatric Advance Care Planning (pACP) model exists to serve Spanish speaking adolescents with cancer. Objective: To take first steps to implement pACP as a routine, structured intervention in pediatric hospitals with underserved Spanish speaking adolescents living with cancer through timely conversations with their families to relieve suffering (physical, psychological, spiritual) and maximize the quality of life

Type: Interventional

Start Date: Sep 2019

open study

Late Effects After Treatment in Patients With Previously Diagnosed High-Risk Neuroblastoma
Children's Oncology Group Recurrent Neuroblastoma Stage 2A Neuroblastoma Stage 2B Neuroblastoma Stage 3 Neuroblastoma Stage 4 Neuroblastoma
This research trial studies late effects after treatment in patients with previously diagnosed high-risk neuroblastoma. Studying late effects after treatment may help to decide which treatments for high-risk neuroblastoma are better tolerated with less side effects over time.... expand

This research trial studies late effects after treatment in patients with previously diagnosed high-risk neuroblastoma. Studying late effects after treatment may help to decide which treatments for high-risk neuroblastoma are better tolerated with less side effects over time.

Type: Observational

Start Date: Jun 2017

open study

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). expand

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

Type: Interventional

Start Date: Jul 2014

open study

Response and Biology-Based Risk Factor-Guided Therapy in Treating Younger Patients With Non-high Risk...
Children's Oncology Group Ganglioneuroblastoma Localized Resectable Neuroblastoma Localized Unresectable Neuroblastoma Neuroblastoma
This phase III trial studies how well response and biology-based risk factor-guided therapy works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may not need treatment until it progresses. In this case, observation may be sufficient. Measuring... expand

This phase III trial studies how well response and biology-based risk factor-guided therapy works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may not need treatment until it progresses. In this case, observation may be sufficient. Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and what the best treatment is. Response and biology-based risk factor-guided therapy may be effective in treating patients with non-high risk neuroblastoma and may help to avoid some of the risks and side effects related to standard treatment.

Type: Interventional

Start Date: Jul 2014

open study

Active Surveillance, Bleomycin, Carboplatin, Etoposide, or Cisplatin in Treating Pediatric and Adult...
Children's Oncology Group Childhood Extracranial Germ Cell Tumor Extragonadal Embryonal Carcinoma Germ Cell Tumor Malignant Germ Cell Tumor Malignant Ovarian Teratoma
This phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their... expand

This phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

Type: Interventional

Start Date: May 2017

open study

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,... 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 with known positivity for one or more Tumor Associated Antigens (TAA) (WT1, PRAME and/or survivin) will be eligible to receive Tumor Antigen Associated Cytotoxic T Lymphocytes (TAA-T). Patients will be enrolled in one of two groups: Group A includes patients with newly diagnosed diffuse intrinsic pontine gliomas (DIPGs) who will undergo irradiation as part of their upfront therapy and Group B includes patients with recurrent, progressive or refractory CNS tumors including medulloblastoma, non-brainstem high-grade glioma, and ependymoma, among others. TAA-T will be generated from patient's peripheral blood mononuclear cells (PBMCs) or by apheresis. This protocol is designed as a phase I dose-escalation study. Group A patients (DIPG patients): TAA-T will be infused any time > 2 week after completion of radiotherapy. Group B patients (other recurrent/progressive/refractory CNS tumors): TAA-T will be infused any time > 2 weeks 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

open study

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
Columbia University Mitochondrial Disorders Mitochondrial Genetic Disorders Mitochondrial Diseases Disorder of Mitochondrial Respiratory Chain Complexes Deletion and Duplication of Mitochondrial DNA
The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders. expand

The North American Mitochondrial Disease Consortium (NAMDC) maintains a patient contact registry and tissue biorepository for patients with mitochondrial disorders.

Type: Observational

Start Date: Dec 2010

open study

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform...
SpringWorks Therapeutics, Inc. Plexiform Neurofibroma Neurofibromatosis Type 1 (NF1)
This study evaluates PD-0325901 in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive PD-0325901. expand

This study evaluates PD-0325901 in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive PD-0325901.

Type: Interventional

Start Date: Sep 2019

open study

Adoptive Cord Blood Immunotherapy for EBV, CMV, BKV and Adenovirus Reactivation/Infection or Prophylaxis
Catherine Bollard Viral Infection
This Phase I-II dose-finding trial to determine the optimal dose of intravenous (IV) injection dose of donor-derived cytotoxic T lymphocytes (CTLs) specific for CMV, EBV, BKV and Adenovirus. A maximum of 36 patients will be treated in up to 18 cohorts each of size 2, with the... expand

This Phase I-II dose-finding trial to determine the optimal dose of intravenous (IV) injection dose of donor-derived cytotoxic T lymphocytes (CTLs) specific for CMV, EBV, BKV and Adenovirus. A maximum of 36 patients will be treated in up to 18 cohorts each of size 2, with the first cohort treated at the lowest dose level 1, all successive doses chosen by the EffTox method, and no untried dose level skipped when escalating. The scientific goal of the trial is to determine an optimal IV-CTL cell dose level among the three doses 1.0x107cells/m2, 2 x107cells/m2 and 5x107cells/m2., hereafter dose levels 1, 2, 3. Dose-finding will be done using the sequentially adaptive EffTox trade-off-based design of Thall et al.

Type: Interventional

Start Date: Jan 2018

open study

STRIDE Biorepository
Emory University Anemia, Sickle Cell
The STRIDE Biorepository is an optional substudy available to participants in "Bone Marrow Transplantation vs Standard of Care in Patients with Severe Sickle Cell Disease (BMT CTN 1503) (STRIDE)". expand

The STRIDE Biorepository is an optional substudy available to participants in "Bone Marrow Transplantation vs Standard of Care in Patients with Severe Sickle Cell Disease (BMT CTN 1503) (STRIDE)".

Type: Observational [Patient Registry]

Start Date: Mar 2017

open study

Near Infrared Spectroscopy in Sickle Cell Pediatric Patients
Children's National Research Institute Sickle Cell Disease
Endothelial dysfunction contributes to vaso-occlusion and acute pain in sickle cell disease. Near infrared spectroscopy (NIRS) technology can measure tissue oxygenation and endothelial function. The main objective of this study is to study the natural history of tissue muscle... expand

Endothelial dysfunction contributes to vaso-occlusion and acute pain in sickle cell disease. Near infrared spectroscopy (NIRS) technology can measure tissue oxygenation and endothelial function. The main objective of this study is to study the natural history of tissue muscle oxygenation using NIRS in pediatric sickle cell subjects experiencing acute pain and pediatric sickle cell patients in steady-state.

Type: Observational

Start Date: Jun 2019

open study

Nanoparticle Albumin-Bound Rapamycin, Temozolomide, and Irinotecan Hydrochloride in Treating Pediatric...
Children's Oncology Group Childhood Solid Neoplasm Recurrent Central Nervous System Neoplasm Recurrent Malignant Solid Neoplasm Refractory Central Nervous System Neoplasm Refractory Malignant Solid Neoplasm
This phase I trial studies the side effects and best dose of nanoparticle albumin-bound rapamycin when given together with temozolomide and irinotecan hydrochloride in treating pediatric patients with solid tumors that have come back after a period of time during which the tumor... expand

This phase I trial studies the side effects and best dose of nanoparticle albumin-bound rapamycin when given together with temozolomide and irinotecan hydrochloride in treating pediatric patients with solid tumors that have come back after a period of time during which the tumor could not be detected or has not responded to treatment. Nanoparticle albumin-bound rapamycin may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as temozolomide and irinotecan hydrochloride, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving nanoparticle albumin-bound rapamycin, temozolomide, and irinotecan hydrochloride may work better in treating pediatric patients with solid tumors.

Type: Interventional

Start Date: Jul 2017

open study

HIV-1 Specific T -Cells (HST-NEETs) for HIV-Infected Individuals
Catherine Bollard HIV Infections
This is a phase I, multi-site, study of the safety, immunologic and virologic responses of ex vivo expanded HIV-1 multi-antigen specific T-cell therapy (HST-NEET) as a therapeutic strategy in HIV-infected individuals suppressed on antiretroviral therapy (ART). expand

This is a phase I, multi-site, study of the safety, immunologic and virologic responses of ex vivo expanded HIV-1 multi-antigen specific T-cell therapy (HST-NEET) as a therapeutic strategy in HIV-infected individuals suppressed on antiretroviral therapy (ART).

Type: Interventional

Start Date: Mar 2019

open study