159 matching studies

Sponsor Condition of Interest
Observational Study of Pediatric Rheumatic Diseases: The CARRA Registry
Duke University Rheumatic Joint Disease
Continuation of the CARRA Registry as described in the protocol will support data collection on patients with pediatric-onset rheumatic diseases. The CARRA Registry will form the basis for future CARRA studies. In particular, this observational registry will be used to answer... expand

Continuation of the CARRA Registry as described in the protocol will support data collection on patients with pediatric-onset rheumatic diseases. The CARRA Registry will form the basis for future CARRA studies. In particular, this observational registry will be used to answer pressing questions about therapeutics used to treat pediatric rheumatic diseases, including safety questions.

Type: Observational [Patient Registry]

Start Date: Jul 2015

open study

Cytotoxic T Cells to Treat Relapsed EBV-positive Lymphoma
Catherine Bollard Hodgkin Disease Non Hodgkin Lymphoma Lymphoepithelioma Severe Chronic Active EBV Infection Syndrome (SCAEBV) Leiomyosarcoma
In this study, investigators are trying to see if LMP specific cytotoxic T lymphocytes (CTLs) will prevent or treat disease called Epstein Barr Virus (EBV) Disorder including either Hodgkin Lymphoma or non-Hodgkin Lymphoma or Lymphoepithelioma or severe chronic active EBV infection... expand

In this study, investigators are trying to see if LMP specific cytotoxic T lymphocytes (CTLs) will prevent or treat disease called Epstein Barr Virus (EBV) Disorder including either Hodgkin Lymphoma or non-Hodgkin Lymphoma or Lymphoepithelioma or severe chronic active EBV infection syndrome (SCAEBV) or Leiomyosarcoma which has come back or has not gone away after treatment, including the best treatment. Investigators are using special immune system cells called third party LMP specific cytotoxic T lymphocytes (CTLs), a new experimental therapy. Some patients with Lymphoma or SCAEBV or Leiomyosarcoma show evidence of infection with the virus that causes infectious mononucleosis Epstein Barr virus (EBV) before or at the time of their diagnosis. EBV is found in the cancer cells of up to half the patients with Hodgkin's and non-Hodgkin Lymphoma, suggesting that it may play a role in causing Lymphoma. The cancer cells (in lymphoma) and some B cells (in SCAEBV) infected by EBV are able to hide from the body's immune system and escape destruction. The investigators want to see if special white blood cells, called T cells, that have been trained to kill EBV infected cells can survive in patient's blood and affect the tumor or infection. Investigators used this sort of therapy to treat a different type of cancer that occurs after bone marrow or solid organ transplant called post transplant lymphoma. In this type of cancer the tumor cells have 9 proteins made by EBV on their surface. They grew T cells in the laboratory that recognized all 9 proteins and were able to successfully prevent and treat post transplant lymphoma. However in Hodgkin Lymphoma, the tumor cells and B cells only express 2 EBV proteins. In a previous study they made T cells that recognized all 9 proteins and gave them to patients with Hodgkin Lymphoma. Some patients had a partial response to this therapy but no patients had a complete response. They think one reason may be that many of the T cells reacted with proteins that were not on the tumor cells. In this present study the investigators are trying to find out if the investigators can improve this treatment by growing T cells that recognize proteins expressed on EBV infected Lymphoma cells and B cells called LMP-1 and LMP2. These special T cells are called third party LMP 1/2 -specific cytotoxic T-lymphocytes (CTLs). These LMP-specific cytotoxic T cells are an investigational product not approved by the Food and Drug Administration.

Type: Interventional

Start Date: Nov 2013

open study

Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors,...
Eisai Inc. Recurrent and Refractory Solid Tumors
Phase 1 of this study, utilizing a rolling 6 design, will be conducted to determine a maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D), and to describe the toxicities of lenvatinib administered in combination with everolimus once daily to pediatric participants... expand

Phase 1 of this study, utilizing a rolling 6 design, will be conducted to determine a maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D), and to describe the toxicities of lenvatinib administered in combination with everolimus once daily to pediatric participants with recurrent/refractory solid tumors. Phase 2, utilizing Simon's optimal 2-stage design, will be conducted to estimate the antitumor activity of lenvatinib in combination with everolimus in pediatric participants with selected recurrent/refractory solid tumors including Ewing sarcoma/peripheral primitive neuroectodermal tumor (pPNET), rhabdomyosarcoma, and high grade glioma (HGG) using objective response rate (ORR) at Week 16 as the outcome measure.

Type: Interventional

Start Date: Nov 2017

open study

Social Navigation for Adolescents in ED
Children's 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

Active Surveillance, Bleomycin, Carboplatin, Etoposide, or Cisplatin in Treating Pediatric and Adult...
Children's Oncology Group Adult Germ Cell Tumor Childhood Extracranial Germ Cell Tumor Childhood Germ Cell Tumor Extragonadal Embryonal Carcinoma Grade 2 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

Trial of Panobinostat in Children With Diffuse Intrinsic Pontine Glioma
Pediatric Brain Tumor Consortium Glioma
This phase I trial studies the side effects and best dose of panobinostat in treating younger patients with diffuse intrinsic pontine glioma (DIPG). Panobinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Stratum 1 treats patients... expand

This phase I trial studies the side effects and best dose of panobinostat in treating younger patients with diffuse intrinsic pontine glioma (DIPG). Panobinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Stratum 1 treats patients with DIPG that has returned or gotten worse (progressed). Stratum 2 treats patients with DIPG that has not yet gotten worse. Currently, only Stratum 2 is enrolling patients.

Type: Interventional

Start Date: Jun 2016

open study

Study to Assess Safety and Preliminary Activity of Eribulin Mesylate in Pediatric Participants With Relapsed/Refractory...
Eisai Inc. Relapsed/Refractory Rhabdomyosarcoma Non-rhabdomyosarcoma Soft Tissue Sarcoma Ewing Sarcoma
This study will be conducted as an assessment of the safety and preliminary activity of eribulin mesylate in pediatric participants with relapsed/refractory rhabdomyosarcoma (RMS), non-rhabdomyosarcoma soft tissue sarcoma (NRSTS), or Ewing sarcoma (EWS) to determine whether each... expand

This study will be conducted as an assessment of the safety and preliminary activity of eribulin mesylate in pediatric participants with relapsed/refractory rhabdomyosarcoma (RMS), non-rhabdomyosarcoma soft tissue sarcoma (NRSTS), or Ewing sarcoma (EWS) to determine whether each cohort warrants further investigation.

Type: Interventional

Start Date: Apr 2018

open study

Imatinib Mesylate and Combination Chemotherapy in Treating Patients With Newly Diagnosed Philadelphia...
Children's Oncology Group B Acute Lymphoblastic Leukemia BCR-ABL1 Fusion Protein Expression Minimal Residual Disease Philadelphia Chromosome Positive T Acute Lymphoblastic Leukemia
This randomized phase III trial studies how well imatinib mesylate and combination chemotherapy work in treating patients with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia. Imatinib mesylate may stop the growth of cancer cells by blocking some... expand

This randomized phase III trial studies how well imatinib mesylate and combination chemotherapy work in treating patients with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia. Imatinib mesylate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. 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. Giving imatinib mesylate and combination chemotherapy may work better in treating patients with Philadelphia chromosome positive acute lymphoblastic leukemia.

Type: Interventional

Start Date: Jul 2017

open study

Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell...
Monica Bhatia Sickle Cell Disease Graft Versus Host Disease
To assess the tolerability of the costimulation blocking agent abatacept (CTLA4-Ig) when added to the standard graft versus host disease (GVHD) prophylaxis regimen of a calcineurin inhibitor and methotrexate in patients receiving early alemtuzumab followed by fludarabine, thiotepa,... expand

To assess the tolerability of the costimulation blocking agent abatacept (CTLA4-Ig) when added to the standard graft versus host disease (GVHD) prophylaxis regimen of a calcineurin inhibitor and methotrexate in patients receiving early alemtuzumab followed by fludarabine, thiotepa, melphalan, and alemtuzumab for conditioning.

Type: Interventional

Start Date: Jul 2016

open study

Allogeneic Multivirus - Directed Cytotoxic T Lymphocytes (CTL)
Catherine Bollard EBV CMV Adenovirus
In this study, investigators are trying to see if infusion of T cells (called CTLs) will prevent or treat cytomegalovirus (CMV), Epstein Barr Virus (EBV) and adenovirus (AdV) reactivation or infection. Patients with blood cell cancer, other blood disease or a genetic disease... expand

In this study, investigators are trying to see if infusion of T cells (called CTLs) will prevent or treat cytomegalovirus (CMV), Epstein Barr Virus (EBV) and adenovirus (AdV) reactivation or infection. Patients with blood cell cancer, other blood disease or a genetic disease may receive a stem cell transplant. After receiving transplant, they are at risk of infections until a new immune system to fight infections grows from the cord blood cells. In this study, investigators are trying to give special cells called T cells. These cells will try to fight viruses that can cause infection. Investigators will test to see if blood cells from donor that have been grown in a special way, can prevent patients from getting an infection. EBV, AdV and CMV are viruses that can cause serious life-threatening infections in patients who have weak immune systems after transplant. T lymphocytes can kill viral cells but normally there are not enough of them to kill all the virus infected cells after transplant. Some researcher have taken T cells from a person's blood, grown more of them in the laboratory and then given them back to the person during a viral infection after a bone marrow transplant. Some of these studies have shown a positive therapeutic effect in patients receiving the CTLs after a viral infection in the post-transplant period. Investigators will grow these cells from donor in the laboratory in a way that will train them to recognize and remove viruses when the T cells are given after a transplant. Since most donors have previously been infected with EBV, CMV, and adenovirus, investigators are able to use their T cells that remember these viruses to grow the CTLs. However, they now also have a new way of growing CTLs from donors who have not been infected with CMV.

Type: Interventional

Start Date: Feb 2014

open study

Evaluation of Fontan-Associated Liver Disease
Mezzion Pharma Co. Ltd Single Ventricle Heart Disease
A study to evaluate the efficacy of MZ101 therapy in reducing liver stiffness. expand

A study to evaluate the efficacy of MZ101 therapy in reducing liver stiffness.

Type: Observational

Start Date: Feb 2018

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

The Treatment of Progressive Early Onset Spinal Deformities: A Multi-Center Outcome Study
Growing Spine Study Group Early Onset Scoliosis Congenital Spinal Deformities Infantile Idiopathic Scoliosis Neuromuscular Spinal Deformity Syndromic Spinal Deformity
The purpose of this study is to examine the treatment, both surgical and non-surgical, of patients with any form of early onset scoliosis. Such treatment may include the use of growth friendly devices that are surgically or magnetically lengthened; or the use of serial body casting... expand

The purpose of this study is to examine the treatment, both surgical and non-surgical, of patients with any form of early onset scoliosis. Such treatment may include the use of growth friendly devices that are surgically or magnetically lengthened; or the use of serial body casting or bracing, or observation. Outcomes examined will be what can be seen physically on the patient and on x-ray, as well as parent perception of how the treatment effects their child with the use of a health-related quality of life (HRQOL) questionnaire.

Type: Observational [Patient Registry]

Start Date: Oct 2008

open study

Measuring Analgesic Interventions
Julia Finkel Pain
It is generally recognized that pain assessment and management especially in newborns, children and other nonverbal populations is an unmet need. According to the American Medical Association, "the pediatric population is at risk of inadequate pain management, with age-related... expand

It is generally recognized that pain assessment and management especially in newborns, children and other nonverbal populations is an unmet need. According to the American Medical Association, "the pediatric population is at risk of inadequate pain management, with age-related factors affecting pain management in children. Children are often given minimal or no analgesia for procedures that would routinely be treated aggressively in adults. Although much is now known about pain management in children, it has not been widely or effectively translated into routine clinical practice". These two factors combine to emphasize the necessity for an objective tool to quantify pain and monitor the effectiveness of analgesia, especially during treatments. Further, it is reported that many patients require a combination of treatments, and it is often necessary to test a variety of treatments before the personal match for treatment is found. The method in place to change the care on a subjective basis is difficult, time consuming, and not easily individualized. This pilot study is part of an ongoing effort to develop a method to objectively assess response to specific analgesic interventions. It specifically aims to discern the impact of analgesic interventions on sensory nerve fiber sensitivity in a diverse patient population.

Type: Observational

Start Date: Oct 2018

open study

Objective Assessment of Sensory Nerve Fiber Sensitivity
Julia Finkel Nerve Pain
This pilot study utilizes a unique technology to determine nerve fiber sensitivity. This will allow us to determine whether this method and device can discern changes in pain intensity and type, which is predicated on differing nerve fiber sensitivities. expand

This pilot study utilizes a unique technology to determine nerve fiber sensitivity. This will allow us to determine whether this method and device can discern changes in pain intensity and type, which is predicated on differing nerve fiber sensitivities.

Type: Observational

Start Date: Jun 2018

open study

Remodulin as Add-on Therapy for the Treatment of Persistent Pulmonary Hypertension of the Newborn
United Therapeutics Persistent Pulmonary Hypertension of the Newborn
This pilot study aims to assess the safety and treatment effect of acute dosing with IV Remodulin in neonates with persistent pulmonary hypertension of the newborn (PPHN). expand

This pilot study aims to assess the safety and treatment effect of acute dosing with IV Remodulin in neonates with persistent pulmonary hypertension of the newborn (PPHN).

Type: Interventional

Start Date: May 2015

open study

Orphan Europe Carbaglu® Surveillance Protocol
Nicholas Ah Mew N-acetylglutamate Synthase (NAGS) Deficiency
The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved by the United States Food and Drug Administration (FDA) for treatment of acute hyperammonemia due to N-acetylglutamate... expand

The purpose of this study is to conduct post-marketing surveillance of carglumic acid (Carbaglu) to obtain long-term clinical safety information. Carglumic acid was approved by the United States Food and Drug Administration (FDA) for treatment of acute hyperammonemia due to N-acetylglutamate synthase (NAGS) deficiency. Much of the FDA-required data is already collected through the Longitudinal Study of Urea Cycle Disorders (RDCRN Protocol #5101). This study will collect additional data on adverse events (interim events), adverse reactions, pregnancy, and fetal outcomes.

Type: Observational [Patient Registry]

Start Date: Apr 2012

open study

PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
BioMarin Pharmaceutical Phenylketonuria Hyperphenylalaninaemia
The objective of this study is to evaluate the safety of long-term treatment with Kuvan. expand

The objective of this study is to evaluate the safety of long-term treatment with Kuvan.

Type: Observational

Start Date: Sep 2008

open study

Palbociclib in Combination With Chemotherapy in Treating Children With Relapsed Acute Lymphoblastic Leukemia...
Children's Oncology Group Leukemia, Lymphocytic Lymphoblastic Lymphoma T-cell Lymphoma T-cell Leukemia Recurrent Disease
AINV18P1 is a Phase 1 study where palbociclib will be administrated in combination with a standard re-induction platform in pediatric relapsed Acute Lymphoblastic Leukemia (ALL) and lymphoblastic lymphoma (LL). LL patients are included because the patient population is rare and... expand

AINV18P1 is a Phase 1 study where palbociclib will be administrated in combination with a standard re-induction platform in pediatric relapsed Acute Lymphoblastic Leukemia (ALL) and lymphoblastic lymphoma (LL). LL patients are included because the patient population is rare and these patients are most commonly treated with ALL regimens. The proposed starting dose for this study will be 50 mg/m^2/day for 21 days.

Type: Interventional

Start Date: Apr 2019

open study

Carnitine, Aclycarnitine, Myocardial Function, and CRRT
Asha Moudgil Acute Renal Failure
Carnitine is essential for the transport of fatty acids into the mitochondria and energy production in different muscles, including the myocardium. It is also needed to protect myocyte cell membranes from oxidative damage by removing excess acyl carnitine groups. Patients receiving... expand

Carnitine is essential for the transport of fatty acids into the mitochondria and energy production in different muscles, including the myocardium. It is also needed to protect myocyte cell membranes from oxidative damage by removing excess acyl carnitine groups. Patients receiving chronic intermittent hemodialysis (HD) are known to be at increased risk for carnitine deficiency as a result of its removal during the dialysis procedure, lack of endogenous synthesis by the kidney, and inadequate dietary intake. The carnitine status of children undergoing continuous renal replacement therapy (CRRT) has not been studied. Children undergoing CRRT in the intensive care unit (ICU) setting may be at increased risk for carnitine deficiency due to its continuous removal, lack of carnitine production by the kidney, and absence of carnitine intake (as majority of these children can not eat and there is no carnitine added to total parenteral nutrition (TPN). Carnitine deficiency may increase the risk of cardiac dysfunction in critically ill children. This is the first study to examine carnitine status in children undergoing CRRT. Standard echo as well as more sensitive speckle tracking echo will be used to evaluate the effect of carnitine deficiency on myocardial function.

Type: Observational

Start Date: Dec 2014

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

Phase 2 Study of Alisertib Therapy for Rhabdoid Tumors
St. Jude Children's Research Hospital Malignant Rhabdoid Tumor Atypical Teratoid Rhabdoid Tumor
This study incorporates alisertib, the small-molecule inhibitor of Aurora A activity, in the treatment of patients younger than 22 years of age. Patients with recurrent or refractory AT/RT or MRT will receive alisertib as a single agent. Patients with newly diagnosed AT/RT will... expand

This study incorporates alisertib, the small-molecule inhibitor of Aurora A activity, in the treatment of patients younger than 22 years of age. Patients with recurrent or refractory AT/RT or MRT will receive alisertib as a single agent. Patients with newly diagnosed AT/RT will receive alisertib as part of age- and risk-adapted chemotherapy. Radiation therapy will be given to children ≥12 months of age. Patients with AT/RT and concurrent extra-CNS MRT are eligible. Alisertib will be administered as a single agent on days 1-7 of each 21-day cycle in all recurrent patients enrolled on Stratum A. For the patients on the newly diagnosed strata (B, C or D), alisertib will be administered in sequence with chemotherapy and radiotherapy. This study has 3 primary strata: (A) children with recurrent/progressive AT/RT or extra-CNS MRT, (B) children < 36 months-old with newly diagnosed AT/RT, (C) children > 36 months old with newly diagnosed AT/RT. Children with concurrent MRT will be treated according to age and risk stratification schemes outlined for strata B and C and will have additional treatment for local control. Children with synchronous AT/RT will be treated with age and CNS risk-appropriate therapy, and also receive surgery and/or radiation therapy for local control of the non-CNS tumor. PRIMARY OBJECTIVES - To estimate the sustained objective response rate and disease stabilization in pediatric patients with recurrent or progressive AT/RT (atypical teratoid rhabdoid tumor in the CNS) (Stratum A1) treated with alisertib and to determine if the response is sufficient to merit continued investigation of alisertib in this population. - To estimate the sustained objective response rate and disease stabilization in pediatric patients with recurrent or progressive extra-CNS MRT (malignant rhabdoid tumor outside the CNS) (Stratum A2) treated with alisertib and to determine if the response is sufficient to merit continued investigation of alisertib in this population. - To estimate the 3-year PFS rate of patients with newly diagnosed AT/RT who are younger than 36 months of age at diagnosis with no metastatic disease (Stratum B1) treated with alisertib in sequence with induction and consolidation chemotherapy and radiation therapy (depending on age) and to determine if the rates are sufficient to merit continued investigation of alisertib in this population. - To estimate the 1-year PFS rate of patients with newly diagnosed AT/RT who are younger than 36 months of age at diagnosis, with metastatic disease (Stratum B2) treated with alisertib in sequence with induction and consolidation chemotherapy and to determine if the rates are sufficient to merit continued investigation of alisertib in this population. - To estimate the 3-year PFS rate of patients with newly diagnosed AT/RT who are 3 years of age or greater at diagnosis with no metastatic disease and gross total resection or near total resection (Stratum C1) treated with alisertib in sequence with radiation therapy and consolidation chemotherapy and to determine if the rates are sufficient to merit continued investigation of alisertib in this population. - To estimate the 1-year PFS rate of patients with newly diagnosed AT/RT who are 3 years of age or greater at diagnosis with metastatic or residual disease (Stratum C2) treated with alisertib in sequence with radiation therapy and consolidation chemotherapy and to determine if the rates are sufficient to merit continued investigation of alisertib in this population. - To characterize the pharmacokinetics and pharmacodynamics of alisertib in pediatric patients and to relate drug disposition to toxicity. SECONDARY OBJECTIVES - To estimate the duration of objective response and PFS in patients with recurrent/progressive AT/RT and MRT (Strata A1 and A2). - To estimate PFS and OS distributions in patients with newly diagnosed AT/RT (Strata B1, B2, B3, C1 and C2). - To describe toxicities experienced by patients treated on this trial, specifically any toxicities of alisertib when administered as a single agent or in combination with other therapy over multiple courses and toxicities related to proton or photon radiation therapy. - To describe the patterns of local and distant failure in newly diagnosed patients (Strata B1, B2, B3, C1 and C2). Local control relative to primary-site radiation therapy, with criteria for infield, marginal, or distant failure will also be reported descriptively.

Type: Interventional

Start Date: May 2014

open study

A Extension Study of Udenafil in Adolescents
Mezzion Pharma Co. Ltd Functional Single Ventricle Heart Disease
This study is a 12-month (52 week) safety extension study to supplement the FUEL Phase III clinical trial to provide safety information regarding the long-term use of udenafil in adolescents with single ventricle congenital heart disease. expand

This study is a 12-month (52 week) safety extension study to supplement the FUEL Phase III clinical trial to provide safety information regarding the long-term use of udenafil in adolescents with single ventricle congenital heart disease.

Type: Interventional

Start Date: Jan 2017

open study

Stem Cells in NF1 Patients With Tumors of the Central Nervous System
Children's Research Institute Neurofibromatosis Type 1 Tumors of the Central Nervous System
Objectives 1. Establish an induced pluripotent stem cell (iPSC) bank for phenotypically well-characterized patients with NF1. 2. Develop isogenic NF1 wild-type (NF1+/+), NF1 heterozygous (NF1+/-) and NF1 homozygous (NF1-/-) iPSC lines from individual patients using CRISPR/CAS9... expand

Objectives 1. Establish an induced pluripotent stem cell (iPSC) bank for phenotypically well-characterized patients with NF1. 2. Develop isogenic NF1 wild-type (NF1+/+), NF1 heterozygous (NF1+/-) and NF1 homozygous (NF1-/-) iPSC lines from individual patients using CRISPR/CAS9 technology. 3. Differentiate and characterize disease-relevant brain cells such as excitatory and inhibitory neurons, astrocytes and oligodendrocytes from patient-specific iPSC lines. 4. Screen and identify the drug(s) that can reverse or alleviate the disease phenotypes.

Type: Observational

Start Date: Nov 2015

open study

A Study of Ribociclib and Everolimus Following Radiation Therapy in Children With Newly Diagnosed Non-biopsied...
Children's Hospital Medical Center, Cincinnati Diffuse Intrinsic Pontine Glioma Malignant Glioma of Brain High Grade Glioma Bithalamic High Grade Glioma Brainstem Glioma
In this research study, we want to learn about the safety of the study drugs, ribociclib and everolimus, when given together at different doses after radiation therapy. We also want to learn about the effects, if any, these drugs have on children and young adults with brain tumors.... expand

In this research study, we want to learn about the safety of the study drugs, ribociclib and everolimus, when given together at different doses after radiation therapy. We also want to learn about the effects, if any, these drugs have on children and young adults with brain tumors. We are asking people to be in this research study who have been diagnosed with a high grade glioma, their tumor has been screened for the Rb1 protein, and they have recently finished radiation therapy. If a patient has DIPG or a Bi-thalamic high grade glioma, they do not need to have the tumor tissue screened for the Rb1 protein, but do need to have finished radiation therapy. Tumor cells grow and divide quickly. In normal cells, there are proteins that control how fast cells grow but in cancer cells these proteins no longer work correctly making tumor cells grow quickly. Both study drugs work in different ways to slow down the growth of tumor cells. The researchers think that if the study drugs are given together soon after radiation therapy, it may help improve the effect of the radiation in stopping or slowing down tumor growth. The study drugs, ribociclib and everolimus, have been approved by the United States Food and Drug Administration (FDA). Ribociclib is approved to treat adults with breast cancer and everolimus is approved for use in adults and children who have other types of cancers. The combination of ribociclib and everolimus has not been tested in children or in people with brain tumors and is considered investigational. The goals of this study are: - Find the safest dose of ribociclib and everolimus that can be given together after radiation. - Learn the side effects (both good and bad) the study drugs have on the body and tumor. - Measure the levels of study drug in the blood over time. - Study the changes in the endocrine system that may be caused by the tumor, surgery or radiation.

Type: Interventional

Start Date: Nov 2017

open study