160 matching studies

Sponsor Condition of Interest
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 Elevated Alpha-Fetoprotein Hepatoblastoma SMARCB1 Gene Mutation
This partially randomized phase II/III trial studies how well cisplatin and combination chemotherapy works in treating children and young adults with hepatoblastoma or liver cancer after surgery. Drugs used in chemotherapy, such as cisplatin, doxorubicin, fluorouracil, vincristine... expand

This partially randomized phase II/III trial studies how well cisplatin and combination chemotherapy works in treating children and young adults with hepatoblastoma or liver cancer after surgery. 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 after surgery may kill more tumor cells.

Type: Interventional

Start Date: May 2018

open study

Preventing Epilepsy Using Vigabatrin In Infants With Tuberous Sclerosis Complex
Martina Bebin Tuberous Sclerosis Complex
Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to the onset of their first seizure expand

Study design is a Phase IIb prospective multi-center, randomized, placebo-controlled, double-blind clinical trial. The goal will be to enroll 80 infants with Tuberous Sclerosis Complex who are less than 6 months of age prior to the onset of their first seizure

Type: Interventional

Start Date: Dec 2016

open study

Adavosertib and Irinotecan Hydrochloride in Treating Younger Patients With Relapsed or Refractory Solid...
National Cancer Institute (NCI) Central Nervous System Embryonal Tumor With Rhabdoid Features Central Nervous System Embryonal Tumor, Not Otherwise Specified Central Nervous System Ganglioneuroblastoma Embryonal Tumor With Multilayered Rosettes, C19MC-Altered Pineoblastoma
This phase I/II trial studies the side effects and best dose of adavosertib and irinotecan hydrochloride in treating younger patients with solid tumors that have come back or that have not responded to standard therapy. Adavosertib and irinotecan hydrochloride may stop the growth... expand

This phase I/II trial studies the side effects and best dose of adavosertib and irinotecan hydrochloride in treating younger patients with solid tumors that have come back or that have not responded to standard therapy. Adavosertib and irinotecan hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Type: Interventional

Start Date: Mar 2014

open study

Combination Chemotherapy With or Without Ganitumab in Treating Patients With Newly Diagnosed Metastatic...
National Cancer Institute (NCI) Metastatic Ewing Sarcoma Metastatic Malignant Neoplasm in the Bone Metastatic Malignant Neoplasm in the Bone Marrow Metastatic Malignant Neoplasm in the Lung Metastatic Peripheral Primitive Neuroectodermal Tumor of Bone
This randomized phase III trial studies how well combination chemotherapy with or without ganitumab works in treating patients with newly diagnosed Ewing sarcoma that has spread to other parts of the body. Immunotherapy with monoclonal antibodies, such as ganitumab, may help... expand

This randomized phase III trial studies how well combination chemotherapy with or without ganitumab works in treating patients with newly diagnosed Ewing sarcoma that has spread to other parts of the body. Immunotherapy with monoclonal antibodies, such as ganitumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Drugs used in chemotherapy, such as vincristine sulfate, doxorubicin hydrochloride, cyclophosphamide, ifosfamide, and etoposide, 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. It is not yet known whether combination chemotherapy is more effective with or without ganitumab in treating patients with newly diagnosed Ewing sarcoma.

Type: Interventional

Start Date: Dec 2014

open study

A Study of Ramucirumab (LY3009806) in Children With Refractory Solid Tumors
Eli Lilly and Company Pediatric Solid Tumor Refractory Tumor Recurrent Tumor CNS Malignancies
The main purpose of this study is to evaluate the safety of the study drug known as ramucirumab in children with recurrent or refractory solid tumors including central nervous system (CNS) tumors. expand

The main purpose of this study is to evaluate the safety of the study drug known as ramucirumab in children with recurrent or refractory solid tumors including central nervous system (CNS) tumors.

Type: Interventional

Start Date: Dec 2015

open study

A Safety and Efficacy Study of Ibrutinib in Pediatric and Young Adult Participants With Relapsed or Refractory...
Janssen Research & Development, LLC Lymphoma, Non-Hodgkin
The purpose of this study is to confirm that the pharmacokinetics of ibrutinib in pediatric participants is consistent with that in adults (part 1) and to assess efficacy (event-free survival [EFS]) of ibrutinib in combination with rituximab, ifosfamide, carboplatin, and etoposide... expand

The purpose of this study is to confirm that the pharmacokinetics of ibrutinib in pediatric participants is consistent with that in adults (part 1) and to assess efficacy (event-free survival [EFS]) of ibrutinib in combination with rituximab, ifosfamide, carboplatin, and etoposide (RICE) or rituximab, vincristine, ifosfamide, carboplatin, and idarubicin (RVICI) background therapy compared to RICE or RVICI background therapy alone (part 2).

Type: Interventional

Start Date: Jul 2016

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

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

open study

Phase II Pediatric Study With Dabrafenib in Combination With Trametinib in Patients With HGG and LGG
Novartis Pharmaceuticals Diffuse Astrocytoma Anaplastic Astrocytoma Astrocytoma Oligodendroglioma, Childhood Anaplastic Oligodendroglioma
The purpose of this study is to investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive low grade glioma or relapsed or refractory high grade glioma. expand

The purpose of this study is to investigate the activity of dabrafenib in combination with trametinib in children and adolescent patients with BRAF V600 mutation positive low grade glioma or relapsed or refractory high grade glioma.

Type: Interventional

Start Date: Dec 2017

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 an antibody therapy called Nivolumab, in relapsed/refractory lymphoma (rel/ref) patients with... 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 an antibody therapy called 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: Apr 2019

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

Sofosbuvir/Velpatasvir in Adolescents and Children With Chronic HCV Infection
Gilead Sciences Hepatitis C Virus Infection
This study will have 2 parts: Pharmacokinetics (PK) Lead-in Phase and the Treatment Phase. The primary objective of the PK Lead-in Phase is to evaluate the steady state PK and confirm the dose of sofosbuvir/velpatasvir (SOF/VEL) fixed-dose combination (FDC) in pediatric participants... expand

This study will have 2 parts: Pharmacokinetics (PK) Lead-in Phase and the Treatment Phase. The primary objective of the PK Lead-in Phase is to evaluate the steady state PK and confirm the dose of sofosbuvir/velpatasvir (SOF/VEL) fixed-dose combination (FDC) in pediatric participants with chronic hepatitis C virus (HCV) infection. The primary objective of the Treatment Phase is to evaluate the safety and tolerability of SOF/VEL for 12 weeks in pediatric participants with chronic HCV.

Type: Interventional

Start Date: Jan 2017

open study

HIV-1 Specific T -Cells (HST-NEETs) for HIV-Infected Individuals
Michael Keller 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: Apr 2019

open study

Multi-institutional Prospective Research of Expanded Multi-antigen Specifically Oriented Lymphocytes...
Catherine Bollard Relapsed/Refractory Hematopoietic Malignancies, Acute Myeloid Leukemia and MDS
This Phase I dose-escalation trial is designed to evaluate the safety of administering rapidly-generated multi-antigen-specific T lymphocytes, to HSCT recipients (Arm A) or future HSCT recipients (Arm B) for the treatment or relapsed or refractory hematopoietic malignancies,... expand

This Phase I dose-escalation trial is designed to evaluate the safety of administering rapidly-generated multi-antigen-specific T lymphocytes, to HSCT recipients (Arm A) or future HSCT recipients (Arm B) for the treatment or relapsed or refractory hematopoietic malignancies, to determine if event-free survival (EFS) at six months after HSCT is improved with TAA-L administration at six months after HSCT for acute myeloid leukemia and MDS (Arm C).

Type: Interventional

Start Date: Jan 2015

open study

Adavosertib and Local Radiation Therapy in Treating Children With Newly Diagnosed Diffuse Intrinsic Pontine...
National Cancer Institute (NCI) Anaplastic Astrocytoma Anaplastic Oligoastrocytoma Diffuse Intrinsic Pontine Glioma Diffuse Midline Glioma, H3 K27M-Mutant Glioblastoma
This phase I trial studies the side effects and the best dose of adavosertib when given together with local radiation therapy in treating children with newly diagnosed diffuse intrinsic pontine gliomas. Adavosertib may stop the growth of tumor cells by blocking some of the enzymes... expand

This phase I trial studies the side effects and the best dose of adavosertib when given together with local radiation therapy in treating children with newly diagnosed diffuse intrinsic pontine gliomas. Adavosertib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Radiation therapy uses high energy x-rays, gamma rays, neutrons, protons, or other sources to kill tumor cells and shrink tumors. Giving adavosertib with local radiation therapy may work better than local radiation therapy alone in treating diffuse intrinsic pontine gliomas.

Type: Interventional

Start Date: Sep 2013

open study

Entinostat in Treating Pediatric Patients With Recurrent or Refractory Solid Tumors
National Cancer Institute (NCI) Brain Stem Neoplasm Pineal Region Neoplasm Recurrent Central Nervous System Neoplasm Recurrent Lymphoma Recurrent Malignant Solid Neoplasm
This phase I trial studies the side effects and best dose of entinostat in treating pediatric patients with solid tumors that have come back or have not responded to treatment. Entinostat may block some of the enzymes needed for cell division and it may help to kill tumor cells.... expand

This phase I trial studies the side effects and best dose of entinostat in treating pediatric patients with solid tumors that have come back or have not responded to treatment. Entinostat may block some of the enzymes needed for cell division and it may help to kill tumor cells.

Type: Interventional

Start Date: Dec 2016

open study

Long-Term Safety Study Of Tofacinib In Patients With Juvenile Idiopathic Arthritis
Pfizer Juvenile Idiopathic Arthritis
Evaluate long-term safety and tolerability of tofacitinib in patients with JIA, who have previously participated in tofacitinib JIA studies. expand

Evaluate long-term safety and tolerability of tofacitinib in patients with JIA, who have previously participated in tofacitinib JIA studies.

Type: Interventional

Start Date: Mar 2013

open study

Response-Based Chemotherapy in Treating Newly Diagnosed Acute Myeloid Leukemia or Myelodysplastic Syndrome...
Children's Oncology Group Acute Myeloid Leukemia Blasts 5 Percent or More of Bone Marrow Nucleated Cells Cytopenia Down Syndrome Myelodysplastic Syndrome
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

Pharmacokinetics, Safety, and Antiviral Activity of the Elvitegravir/Cobicistat/Emtricitabine/Tenofovir...
Gilead Sciences Acquired Immune Deficiency Syndrome (AIDS) HIV Infections
There will be 3 cohorts in this study. The primary objectives of Cohort 1 are to evaluate the steady state pharmacokinetics (PK) for elvitegravir (EVG) and tenofovir alafenamide (TAF) and confirm the dose of the elvitegravir/cobicistat/emtricitabine/tenofovir alafenamide (E/C/F/TAF)... expand

There will be 3 cohorts in this study. The primary objectives of Cohort 1 are to evaluate the steady state pharmacokinetics (PK) for elvitegravir (EVG) and tenofovir alafenamide (TAF) and confirm the dose of the elvitegravir/cobicistat/emtricitabine/tenofovir alafenamide (E/C/F/TAF) single tablet regimen (STR) (Part A) and to evaluate the safety and tolerability of E/C/F/TAF STR through Week 24 (Part B) in HIV-1 infected, antiretroviral (ARV) treatment-naive adolescents. The primary objectives of Cohort 2 are to evaluate the PK of EVG and TAF in virologically suppressed HIV-1 infected children 6 to < 12 years of age weighing ≥ 25 kg administered E/C/F/TAF STR (Part A) and to evaluate the safety and tolerability of E/C/F/TAF STR through Week 24 in virologically suppressed HIV-1 infected children 6 to < 12 years of age weighing ≥ 25 kg (Part B). The primary objectives of cohort 3 are to evaluate the PK of EVG and TAF and confirm the dose of the STR, and to evaluate the safety and tolerability of E/C/F/TAF low dose (LD) STR in virologically suppressed HIV-1 infected children ≥ 2 years of age and weighing ≥ 14 to < 25 kg.

Type: Interventional

Start Date: May 2013

open study

ATHN 8: PUPs Matter Study
American Thrombosis and Hemostasis Network Hemophilia Hemophilia A Hemophilia B Hemophilia A With Inhibitor Hemophilia B With Inhibitor
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs). Participants will be followed as they receive their first 50 exposure... expand

This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs). Participants will be followed as they receive their first 50 exposure days (ED) to clotting factor replacement product, both prospectively and retrospectively. The data collected on evolving treatment practices will define the incidence and risk factors for inhibitor development during the high risk period of first 50 ED and improve the outcomes of this vulnerable population.

Type: Observational

Start Date: Oct 2018

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

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

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

Farber Disease Natural History Study
Enzyvant Farber GmbH Farber Disease Farber's Disease Farber Lipogranulomatosis Acid Ceramidase Deficiency Ceramidase Deficiency
The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber disease. All patients diagnosed with Farber disease... expand

The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber disease. All patients diagnosed with Farber disease are eligible, including both those who have and have not undergone hematopoietic stem cell transplantation (HSCT). Additionally, data and records from deceased patients will provide valuable retrospective data for this study. The secondary objective of the study is to establish a set of clinical data, laboratory data (biomarkers), and functional data potentially useful for: - Assessing the efficacy of HSCT and the efficacy of potential future therapies (for example with RVT-801, recombinant human acid ceramidase) in Farber disease - Characterizing changes in symptoms of patients over time - Characterizing distinct groups (phenotypes) within the patient population - Documenting the disease histories of individual patients to serve as intra-subject control data for those who may enroll in any future clinical studies with therapies for Farber disease The exploratory objectives of the study are: - To explore the relationship between patient disease activity or phenotype and specific ceramide levels or specific immunologic markers (cytokines/chemokines) in blood - To evaluate a standardized tool, the Farber Disease Natural History Instrument (FDNI), to be used for the collection of patient history information, data from clinical, laboratory, genetic, and functional studies, and data from review of medical records

Type: Observational

Start Date: Nov 2017

open study

Prexasertib in Treating Pediatric Patients With Recurrent or Refractory Solid Tumors
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 prexasertib 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 does not respond to treatment. Checkpoint kinase 1 inhibitor LY2606368... expand

This phase I trial studies the side effects and best dose of prexasertib 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 does not respond to treatment. Checkpoint kinase 1 inhibitor LY2606368 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Type: Interventional

Start Date: Feb 2017

open study