169 matching studies

Sponsor Condition of Interest
Study of Clofarabine in Patients With Recurrent or Refractory Langerhans Cell Histiocytosis and LCH-related...
Dana-Farber Cancer Institute Langerhans Cell Histiocytosis
This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders. expand

This research study is evaluating a drug called clofarabine as a possible treatment for Langerhans Cell Histiocytosis (LCH) and and other histiocytic disorders.

Type: Interventional

Start Date: May 2015

open study

Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1
Kristina Hardy Neurofibromatosis Type 1
The main objective of the study is to assess the efficacy of a home-based, computerized cognitive training (CT) program, called CogmedRM, targeted to improve working memory in children with NF1 and working memory difficulties.This is a Phase II randomized parallel group controlled... expand

The main objective of the study is to assess the efficacy of a home-based, computerized cognitive training (CT) program, called CogmedRM, targeted to improve working memory in children with NF1 and working memory difficulties.This is a Phase II randomized parallel group controlled clinical trial comparing two interventions on cognitive outcomes. Participants will be stratified by stimulant medication use and randomized equally between the two interventions within stratum. Participants will be in the study for to 11 weeks.

Type: Interventional

Start Date: May 2016

open study

National Collaborative to Improve Care of Children With Complex Congenital Heart Disease
Children's Hospital Medical Center, Cincinnati Hypoplastic Left Heart Syndrome (HLHS)
The purpose of this initiative is to improve care and outcomes for infants with HLHS by expanding the NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, by improving the use of... expand

The purpose of this initiative is to improve care and outcomes for infants with HLHS by expanding the NPC-QIC national registry to gather clinical care process, outcome, and developmental data on infants with HLHS between diagnosis and 12 months of age, by improving the use of standards into everyday practice across pediatric cardiology centers, and by engaging parents as partners in the process.

Type: Observational [Patient Registry]

Start Date: May 2016

open study

Minimizing Toxicity in HLA-identical Related Donor Transplantation for Children With Sickle Cell Disease
Allistair Abraham, MD 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

open study

Prospective Observational Cohort Study of Fetal Atrial Flutter & Supraventricular Tachycardia
The Hospital for Sick Children 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... 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

open study

Multicenter Prospective Cohort Study on Current Treatments of Legg-Calvé-Perthes Disease
Texas Scottish Rite Hospital for Children Legg Calve Perthes Disease
Legg-Calvé-Perthes disease is a childhood hip disorder which is common enough to be a significant public health problem (affects 1 in 740 boys between ages 0—14), but uncommon enough to have a sufficient number of patients from a single institution to perform a definitive prospective... expand

Legg-Calvé-Perthes disease is a childhood hip disorder which is common enough to be a significant public health problem (affects 1 in 740 boys between ages 0—14), but uncommon enough to have a sufficient number of patients from a single institution to perform a definitive prospective study comparing the results of current treatments. The present study will establish a database of prospectively identified patients with Legg-Calvé-Perthes (LCP) Disease and collect information regarding their presentation, treatment, and outcomes in the course of receiving currently available treatments. This study seeks to compare the outcomes of current treatments in the management of different age groups (ages 1-6, 6—8, 8—11, >11) of patients with Perthes disease at two- and five-year followup and at skeletal maturity. For each age group, two to three common treatment regimens currently used by practicing pediatric orthopaedic surgeons will be compared. The intervention a patient receives is determined through physician preference. Physicians pick an intervention for each age group and treat each patient with the same intervention.

Type: Observational

Start Date: Aug 2012

open study

Comparing the Effects of Lactated Ringers and Normal Saline in Acute Pancreatitis
Children's Hospital Medical Center, Cincinnati Pancreatitis
Acute pancreatitis is increasingly common diagnosis in children. Most of the guidelines related to the details of management of acute pancreatitis are extrapolated from the adult literature. There is only limited data regarding management of acute pancreatitis in children. The... expand

Acute pancreatitis is increasingly common diagnosis in children. Most of the guidelines related to the details of management of acute pancreatitis are extrapolated from the adult literature. There is only limited data regarding management of acute pancreatitis in children. The mainstay of management is bowel and pancreatic rest with significant fluid support to minimize the effect of the cytokines on the pancreas and other organs. The standard fluid choices are Lactated Ringer's solution (LR) and normal saline (NS). Currently, both LR and NS are used at the discretion of the treating physician as the standard of care for acute pancreatitis. The investigators hope to examine the question of ideal fluid choice for fluid resuscitation in children with acute pancreatitis by assessing recovery time in the context of measured inflammatory markers and SIRS status at 24 and 48 hours after admission. The ideal fluid choice in the initial resuscitation of acute pancreatitis has not been effectively evaluated before in the pediatric population. Even if there is no statistically significant difference between the two fluid options, this trial will still provide clinically significant information.

Type: Interventional

Start Date: Dec 2014

open study

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. expand

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

open study

Social Navigation for Adolescents in ED
Children's National Research Institute Adolescent Behavior Social Stress Emergencies
Social determinants of health affect patients throughout the life course. They may be particularly relevant for pediatric emergency department (ED) patients. Computerized screening for social and behavioral determinants of health has been deemed effective and acceptable. This... expand

Social determinants of health affect patients throughout the life course. They may be particularly relevant for pediatric emergency department (ED) patients. Computerized screening for social and behavioral determinants of health has been deemed effective and acceptable. This pilot study will characterize the cumulative burden of health related social problems experienced by patients and families in a pediatric ED. It will specifically examine those patients with a subset of 9 high-risk chief complaints, patients with obesity, patients with poor asthma control, and patients with a high number of non-urgent visits, who may be at particularly high risk for health related social problems. Our analysis will compare these subsets of patients with the general ED population, hypothesizing that these groups will have a higher number of health related social problems than the general ED population. Parent and adolescent participants will be approached during ED visits and administered a computerized screening tool. For patients aged 0-13, a survey administered to parents will test for thirteen distinct health related social problems. Two surveys will be administered to adolescent-parent dyads. The adolescent survey will test for thirteen health related social problems, seven of which overlap with those on the parent survey. The average total number of health related social problems in patient groups hypothesized to be at high risk will be compared to the average total number of HRSPs in the general ED population. For adolescent patients, an intervention group will receive social navigation consisting of rapid referrals to community resources based on survey responses by a community health liaison. Their ED recidivism, community resource use and number of unmet social needs at 12-month follow up will be compared with that of a control group that receives screening and written resources only.

Type: Interventional

Start Date: Aug 2017

open study

Treatment of Impulsive Aggression (IA) in Adolescent With ADHD in Conjunction With Standard ADHD Treatment
Supernus Pharmaceuticals, Inc. Attention Deficit Hyperactivity Disorder
The purpose of this study is to evaluate the effect of SPN-810 for the treatment of impulsive aggression (IA) in adolescents diagnosed with ADHD when taken in conjunction with standard ADHD treatment. expand

The purpose of this study is to evaluate the effect of SPN-810 for the treatment of impulsive aggression (IA) in adolescents diagnosed with ADHD when taken in conjunction with standard ADHD treatment.

Type: Interventional

Start Date: Jul 2018

open study

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 the... 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

open study

Patients Treated for SCID (1968-Present)
National Institute of Allergy and Infectious Diseases (NIAID) SCID ADA-SCID XSCID Leaky SCID Omenn Syndrome
People with Primary Immune Deficiency (PID) may develop severe, life-threatening infections as a result of inherited defects in the genes that normally instruct blood-forming cells to develop and to fight infections. PID diseases include Severe Combined Immune Deficiency (SCID),... expand

People with Primary Immune Deficiency (PID) may develop severe, life-threatening infections as a result of inherited defects in the genes that normally instruct blood-forming cells to develop and to fight infections. PID diseases include Severe Combined Immune Deficiency (SCID), leaky SCID, Omenn syndrome (OS), and Reticular Dysgenesis (RD). PIDs may be treated by transplantation of bone marrow stem cells from a healthy person or, in some cases, by enzyme replacement or by gene therapy. Patients with SCID were among the first to receive bone marrow stem cell (also called hematopoietic cells) transplantation (HCT) more than 40 years ago, and HCT is the standard treatment today for this group of diseases. Since PID diseases are rare, there are not enough patients at any single center to determine the full range of causes, natural history, or best methods of treatment. For this research study many PID centers across North America have organized into the Primary Immune Deficiency Treatment Consortium (PIDTC) to pool their experience and study PIDs together. Researchers will collect information on your general health, psychological and developmental health, and the current status of your immune system to help better define future approaches to PID treatments.

Type: Observational

Start Date: May 2011

open study

Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995
National Institute of Allergy and Infectious Diseases (NIAID) Granulomatous Disease, Chronic
Chronic granulomatous disease (CGD) is an inherited immune system abnormality in which bone marrow transplantation (BMT) has been shown to be curative. However the risks of transplantation are high and not all patients with CGD may need to undergo this high risk procedure. This... expand

Chronic granulomatous disease (CGD) is an inherited immune system abnormality in which bone marrow transplantation (BMT) has been shown to be curative. However the risks of transplantation are high and not all patients with CGD may need to undergo this high risk procedure. This study will determine the long term medical condition and daily functioning of participants with CGD after a transplant and if possible, compare these results to participants who do not undergo a transplant.

Type: Observational

Start Date: Jun 2014

open study

Study to Determine the Pharmacokinetics and Pharmacodynamic Effects of Phenylephrine on BP Via IV
West-Ward Pharmaceutical Hypotension
The primary objective of this study is to evaluate the dose effect of Phenylephrine Hydrochloride Injection on the treatment of clinically relevant decreased blood pressure in the pediatric population, ≥12 to 16 year old patients undergoing general and neuraxial anesthesia.... expand

The primary objective of this study is to evaluate the dose effect of Phenylephrine Hydrochloride Injection on the treatment of clinically relevant decreased blood pressure in the pediatric population, ≥12 to 16 year old patients undergoing general and neuraxial anesthesia. The secondary objectives are to describe changes in blood pressure and heart rate, time to onset and to maximal response, and the duration of response; to assess the safety of the product in this population; and to characterize the pharmacokinetics of phenylephrine hydrochloride.

Type: Interventional

Start Date: Feb 2015

open study

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
BioMarin Pharmaceutical Mucopolysaccharidosis IV Type A Morquio A Syndrome MPS IVA
The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of MPS IVA; to evaluate the long-term effectiveness and safety of Vimizim®, including,... expand

The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of MPS IVA; to evaluate the long-term effectiveness and safety of Vimizim®, including, but not limited to, the occurrence of serious hypersensitivity reactions, anaphylaxis, and changes in antibody status; to help the medical community with the development of recommendations for monitoring MPS IVA patients and reports on patient outcomes to optimize patient care; to collect data on other treatment paradigms, and evaluate the prevalences of their use and their effectiveness; to characterize the effects and safety of Vimizim treatment 5 years from enrollment in the Registry for patients under 5 years of age; to monitor pregnancy exposure, including maternal, neonatal, and infant outcomes; and to monitor patients who have completed the MOR-005 and MOR-007 clinical trials. These patients will be encouraged to enroll in the applicable Registry Substudy and will be monitored using the MOR-005 and MOR-007 assessment schedules, respectively.

Type: Observational [Patient Registry]

Start Date: Sep 2014

open study

Patient Centered Comprehensive Medication Adherence Management System in Patients With Sickle Cell Disease
Emory University Sickle Cell Disease
The purpose of this research study is to learn about ways to help children and adults with sickle cell disease who are taking the medication, hydroxyurea. expand

The purpose of this research study is to learn about ways to help children and adults with sickle cell disease who are taking the medication, hydroxyurea.

Type: Interventional

Start Date: Jun 2014

open study

Project: Every Child for Younger Patients With Cancer
Children's Oncology Group Central Nervous System Neoplasm Childhood Immature Teratoma Childhood Langerhans Cell Histiocytosis Childhood Malignant Neoplasm Childhood Mature Teratoma
This research trial studies the Project: Every Child for younger patients with cancer. Gathering health information over time from younger patients with cancer may help doctors find better methods of treatment and on-going care. expand

This research trial studies the Project: Every Child for younger patients with cancer. Gathering health information over time from younger patients with cancer may help doctors find better methods of treatment and on-going care.

Type: Observational

Start Date: Oct 2015

open study

Long-Term Follow-Up of Patients Who Have Participated in Children's Oncology Group Studies
Children's Oncology Group Acute Lymphoblastic Leukemia Brain Neoplasm Hematopoietic Cell Transplantation Recipient Hodgkin Lymphoma Osteosarcoma
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... 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: May 2008

open study

Biomarkers in Tumor Tissue Samples From Patients With Newly Diagnosed Neuroblastoma or Ganglioneuroblastoma
Children's Oncology Group Ganglioneuroblastoma Localized Resectable Neuroblastoma Localized Unresectable Neuroblastoma Regional Neuroblastoma Stage 4 Neuroblastoma
This research trial studies biomarkers in tumor tissue samples from patients with newly diagnosed neuroblastoma or ganglioneuroblastoma. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related... expand

This research trial studies biomarkers in tumor tissue samples from patients with newly diagnosed neuroblastoma or ganglioneuroblastoma. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to cancer.

Type: Observational

Start Date: Nov 2000

open study