166 matching studies

Sponsor Condition of Interest
BAX 111 rVWF in Pediatrics
Baxalta now part of Shire Von Willebrand Disease
The purpose of this study in pediatric participants (<18 years of age) with severe hereditary von Willebrand disease (VWD) is: 1. To assess the efficacy, safety, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and... expand

The purpose of this study in pediatric participants (<18 years of age) with severe hereditary von Willebrand disease (VWD) is: 1. To assess the efficacy, safety, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of nonsurgical bleeding events 2. To assess the efficacy and safety of rVWF with ADVATE during elective or emergency surgery 3. To determine the pharmacokinetic (PK) profile of rVWF

Type: Interventional

Start Date: Dec 2017

open study

Metabolic Optimization Through Diet/Lifestyle Improvements For Youth
Children's National Research Institute Dyslipidemias Obesity
The overarching goal of this study is to evaluate plasma ceramides (Cers) as early nutrition-sensitive biomarkers of metabolic health. The investigators will implement a diet and lifestyle intervention to improve cardiometabolic risk factors and test the corresponding change... expand

The overarching goal of this study is to evaluate plasma ceramides (Cers) as early nutrition-sensitive biomarkers of metabolic health. The investigators will implement a diet and lifestyle intervention to improve cardiometabolic risk factors and test the corresponding change in Cer levels. The intervention will incorporate: a) family-level engagement, enrolling both adolescents and one parent/adult caretaker (PAC); and b) a behavior change mobile health (m-health) app, which will offer real-time support, education and monitoring of diet and activity.

Type: Interventional

Start Date: Mar 2020

open study

Acetaminophen Dosing in Obese Adolescents
Children's National Research Institute Obesity, Childhood
Although there are numerous studies investigating the pharmacokinetic properties of intravenous acetaminophen in infants and children with normal weight, there are none in the obese pediatric patient . This study will investigate the pharmacokinetics and pharmacodynamics of acetaminophen... expand

Although there are numerous studies investigating the pharmacokinetic properties of intravenous acetaminophen in infants and children with normal weight, there are none in the obese pediatric patient . This study will investigate the pharmacokinetics and pharmacodynamics of acetaminophen (total Cl and Vd) in obese children and adolescents

Type: Interventional

Start Date: Aug 2016

open study

Safety Study of Unlicensed, Investigational Cord Blood Units Manufactured by the NCBP for Unrelated Transplantation
New York Blood Center Infusion Reactions
This study will evaluate the safety of infusion of the investigational cord blood units by carefully documenting all infusion-related problems. expand

This study will evaluate the safety of infusion of the investigational cord blood units by carefully documenting all infusion-related problems.

Type: Interventional

Start Date: Feb 2012

open study

A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension...
Actelion Pulmonary Arterial Hypertension
This is a prospective, multicenter, open-label, randomized, controlled, parallel group, group-sequential, event-driven Phase 3 study to evaluate efficacy, safety and pharmacokinetics (PK) of macitentan in children. expand

This is a prospective, multicenter, open-label, randomized, controlled, parallel group, group-sequential, event-driven Phase 3 study to evaluate efficacy, safety and pharmacokinetics (PK) of macitentan in children.

Type: Interventional

Start Date: Nov 2017

open study

A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function
David Nichols, MD Cystic Fibrosis
This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT) in people with one or more copies of the F508del mutation, study the effects of TCT across... expand

This is a prospective, multi-center observational study. The study is designed to measure the clinical effectiveness of elexacaftor, tezacaftor and ivacaftor triple combination therapy (TCT) in people with one or more copies of the F508del mutation, study the effects of TCT across a number of CF disease manifestations, and collect specimens for future research. Subjects in the study will have one "before TCT" visit within 30 days before initiation of the therapy and five "after TCT" visits over a 24-month follow-up period. Most participating sites will be divided into sub-study groups; each sub-study group will have specific non-optional procedures conducted in addition to the "Core" procedures. Finally there are four optional procedures (pH pill, transient elastography, and nasal cell procurement) that will be offered to subjects at certain sites. The duration of participation for each subject is 25 months. NOTE: FDA is currently reviewing the New Drug Application (NDA) for the TCT. Study will not begin unless and until FDA approval is granted.

Type: Observational

Start Date: Oct 2019

open study

Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry...
Bayer Hemophilia A
This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A patients who have switched from an existing therapy to Kovaltry or... expand

This US study aims to assess hemophilia A patient characteristics and reasons for switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A patients who have switched from an existing therapy to Kovaltry or Jivi. In doing so, real world evidence will be obtained from both patient and physician perspectives offering key insights for effective therapeutic management of patients with hemophilia A and to more fully understand what drives patient switching from a patient perspective and a physician perspective.

Type: Observational

Start Date: Aug 2018

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

Adoptive Cellular Therapy in Pediatric Patients With High-grade Gliomas
University of Florida Malignant Glioma High Grade Glioma
It is believed that the body's immune system protects the body by attacking and killing tumor cells. T-lymphocytes (T-cells) are part of the immune system and can attack when they recognize special proteins on the surface of tumors. In most patients with advanced cancer, T-cells... expand

It is believed that the body's immune system protects the body by attacking and killing tumor cells. T-lymphocytes (T-cells) are part of the immune system and can attack when they recognize special proteins on the surface of tumors. In most patients with advanced cancer, T-cells are not stimulated enough to kill the tumor. In this research study, we will use a patient's tumor to make a vaccine which we hope will stimulate T-cells to kill tumor cells and leave normal cells alone. High grade gliomas (HGGs) are very aggressive and difficult for the body's immune system to attack. Before T-cells can become active against tumor cells, they require strong stimulation by special "stimulator" cells in the body called Dendritic Cells (DCs) which are also part of the immune system. DCs can recognize the cancer cells and then activate the T lymphocytes, and create this strong stimulation. The purpose of this research study is to learn whether anti-tumor T-cells and anti-tumor DC vaccines can be given safely. Most importantly, this study is also to determine whether the T-cells and DC vaccines can stimulate a person's immune system to fight off the tumor cells in the brain.

Type: Interventional

Start Date: May 2018

open study

Tacrolimus/Everolimus vs. Tacrolimus/MMF in Pediatric Heart Transplant Recipients Using the MATE Score
Boston Children’s Hospital Pediatric Heart Transplantation Immunosuppression Chronic Kidney Diseases Cardiac Allograft Vasculopathy Heart Transplant Failure and Rejection
The TEAMMATE Trial will enroll 210 pediatric heart transplant patients from 25 centers at 6 months post-transplant and follow each patient for 2.5 years. Half of the participants will receive everolimus and low-dose tacrolimus and the other half will receive tacrolimus and mycophenolate... expand

The TEAMMATE Trial will enroll 210 pediatric heart transplant patients from 25 centers at 6 months post-transplant and follow each patient for 2.5 years. Half of the participants will receive everolimus and low-dose tacrolimus and the other half will receive tacrolimus and mycophenolate mofetil. The trial will determine which treatment is better at reducing the cumulative risk of coronary artery vasculopathy, chronic kidney disease and biopsy proven-acute cellular rejection without an increase in graft loss due to all causes (e.g. infection, PTLD, antibody mediated rejection).

Type: Interventional

Start Date: Jan 2018

open study

A Trial of Temsirolimus With Etoposide and Cyclophosphamide in Children With Relapsed Acute Lymphoblastic...
Therapeutic Advances in Childhood Leukemia Consortium Lymphoblastic Leukemia, Acute, Childhood Lymphoblastic Lymphoma Peripheral T-cell Lymphoma
This is a phase I study of temsirolimus (Torisel) combined with dexamethasone, cyclophosphamide and etoposide in patients with relapsed acute lymphoblastic leukemia (ALL), lymphoblastic lymphoma (LL) or peripheral T-cell lymphoma (PTL). expand

This is a phase I study of temsirolimus (Torisel) combined with dexamethasone, cyclophosphamide and etoposide in patients with relapsed acute lymphoblastic leukemia (ALL), lymphoblastic lymphoma (LL) or peripheral T-cell lymphoma (PTL).

Type: Interventional

Start Date: Mar 2015

open study

A Study of Dulaglutide (LY2189265) in Children and Adolescents With Type 2 Diabetes
Eli Lilly and Company Type 2 Diabetes
The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of the study drug dulaglutide compared to placebo in pediatric participants with type 2 diabetes. The study duration is approximately 60 weeks. expand

The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of the study drug dulaglutide compared to placebo in pediatric participants with type 2 diabetes. The study duration is approximately 60 weeks.

Type: Interventional

Start Date: Dec 2016

open study

Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease
Global Blood Therapeutics Sickle Cell Disease
This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease. Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent Sickle Cell Disease participants... expand

This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease. Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent Sickle Cell Disease participants who were 12-17 years of age. Part C is a multiple dose, safety, tolerability, and PK study, which includes the assessment of hematological effects and the effect on TCD flow velocity of voxelotor in pediatric participants with Sickle Cell Disease who are 4 to 17 years of age. Part D is a multiple dose, safety, tolerability, and PK study, which will examine the hematological effects of voxelotor in pediatric participants with Sickle Cell Disease who are between 9 months to < 4 years of age.

Type: Interventional

Start Date: May 2016

open study

Clinical Benefit of Using Molecular Profiling to Determine an Individualized Treatment Plan for Patients...
University of California, San Francisco Glioma Glioma of Brain Cancer Pediatric Cancer Pediatric Brain Tumor
This is a 2 strata pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). The study will use a new treatment approach based on each patient's tumor gene expression, whole-exome sequencing (WES), targeted panel profile (UCSF 500 gene panel), and RNA-Seq. The... expand

This is a 2 strata pilot trial within the Pacific Pediatric Neuro-Oncology Consortium (PNOC). The study will use a new treatment approach based on each patient's tumor gene expression, whole-exome sequencing (WES), targeted panel profile (UCSF 500 gene panel), and RNA-Seq. The current study will test the efficacy of such an approach in children with High-grade gliomas HGG.

Type: Interventional

Start Date: Nov 2018

open study

Efficacy, Safety, and Tolerability of Levomilnacipran ER in Pediatric (7-17 Years) With Major Depressive...
Allergan Major Depressive Disorder
The objective of this study is to evaluate the efficacy, safety, and tolerability of levomilnacipran compared with placebo in pediatric outpatients (7-17 years) with major depressive disorder (MDD) expand

The objective of this study is to evaluate the efficacy, safety, and tolerability of levomilnacipran compared with placebo in pediatric outpatients (7-17 years) with major depressive disorder (MDD)

Type: Interventional

Start Date: Jul 2018

open study

Efficacy and Safety of Pegzilarginase in Patients With Arginase 1 Deficiency
Aeglea Biotherapeutics Arginase I Deficiency Hyperargininemia
CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension;... expand

CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D. This study will consist of a screening period; a randomized, double-blind treatment period; a long-term extension; and a follow up visit for final safety assessments.

Type: Interventional

Start Date: Apr 2019

open study

A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma
St. Jude Children's Research Hospital Medulloblastoma
Historically, medulloblastoma treatment has been determined by the amount of leftover disease present after surgery, also known as clinical risk (standard vs. high risk). Recent studies have shown that medulloblastoma is made up of distinct molecular subgroups which respond differently... expand

Historically, medulloblastoma treatment has been determined by the amount of leftover disease present after surgery, also known as clinical risk (standard vs. high risk). Recent studies have shown that medulloblastoma is made up of distinct molecular subgroups which respond differently to treatment. This suggests that clinical risk alone is not adequate to identify actual risk of recurrence. In order to address this, we will stratify medulloblastoma treatment in this phase II clinical trial based on both clinical risk (low, standard, intermediate, or high risk) and molecular subtype (WNT, SHH, or Non-WNT Non-SHH). This stratified clinical and molecular treatment approach will be used to evaluate the following: - To find out if participants with low-risk WNT tumors can be treated with a lower dose of radiation to the brain and spine, and a lower dose of the chemotherapy drug cyclophosphamide while still achieving the same survival rate as past St. Jude studies with fewer side effects. - To find out if adding targeted chemotherapy after standard chemotherapy will benefit participants with SHH positive tumors. - To find out if adding new chemotherapy agents to the standard chemotherapy will improve the outcome for intermediate and high risk Non-WNT Non-SHH tumors. - To define the cure rate for standard risk Non-WNT Non-SHH tumors treated with reduced dose cyclophosphamide and compare this to participants from the past St. Jude study. All participants on this study will have surgery to remove as much of the primary tumor as safely possible, radiation therapy, and chemotherapy. The amount of radiation therapy and type of chemotherapy received will be determined by the participant's treatment stratum. Treatment stratum assignment will be based on the tumor's molecular subgroup assignment and clinical risk. The participant will be assigned to one of three medulloblastoma subgroups determined by analysis of the tumor tissue for tumor biomarkers: - WNT (Strata W): positive for WNT biomarkers - SHH (Strata S): positive for SHH biomarkers - Non-WNT Non-SHH, Failed, or Indeterminate (Strata N): negative for WNT and SHH biomarkers or results are indeterminable Participants will then be assigned to a clinical risk group (low, standard, intermediate, or high) based on assessment of: - How much tumor is left after surgery - If the cancer has spread to other sites outside the brain [i.e., to the spinal cord or within the fluid surrounding the spinal cord, called cerebrospinal fluid (CSF)] - The appearance of the tumor cells under the microscope - Whether or not there are chromosomal abnormalities in the tumor, and if present, what type (also called cytogenetics analysis)

Type: Interventional

Start Date: Jun 2013

open study

Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency:
University of Florida Pyruvate Dehydrogenase Complex Deficiency
The objective of this research study is to conduct a pivotal phase 3 trial of treatment with the investigational drug dichloroacetate (DCA) in young children with deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency (PDCD) is the most common cause of congenital... expand

The objective of this research study is to conduct a pivotal phase 3 trial of treatment with the investigational drug dichloroacetate (DCA) in young children with deficiency of the pyruvate dehydrogenase complex (PDC). PDC deficiency (PDCD) is the most common cause of congenital lactic acidosis and is a frequently fatal metabolic disease of childhood for which no proven treatment exists. The investigators predict that DCA represents targeted potential therapy for PDCD because of its ability to increase both the catalytic activity and stability of the enzyme complex. The conclusions of numerous laboratory and clinical investigations are consistent with this postulate and have led to the designation of DCA as an Orphan Product for congenital lactic acidosis by the Food and Drug Administration. A novel Observer reported outcome (ObsRO) survey that is completed by study participant's parent/caregiver, is the efficacy outcome measure. Funding Source - FDA OOPD

Type: Interventional

Start Date: Nov 2019

open study

Safety and Effectiveness of Quizartinib in Children and Young Adults With Acute Myeloid Leukemia (AML),...
Daiichi Sankyo, Inc. Acute Myeloid Leukemia
Quizartinib is an experimental drug. It is not approved for regular use. It can only be used in medical research. Children or young adults with a certain kind of blood cancer (FLT3-ITD AML) might be able to join this study if it has come back after remission or is not responding... expand

Quizartinib is an experimental drug. It is not approved for regular use. It can only be used in medical research. Children or young adults with a certain kind of blood cancer (FLT3-ITD AML) might be able to join this study if it has come back after remission or is not responding to treatment.

Type: Interventional

Start Date: Aug 2018

open study

Clinical and Laboratory Study of Methylmalonic Acidemia
National Human Genome Research Institute (NHGRI) Organic Acidemia Methylmalonic Acidemia Inborn Errors of Metabolism
This study will evaluate patients with methylmalonic acidemia (MMA) to learn more about the genetic causes of the various types of this inherited metabolic disorder and the medical complications associated with it. People with MMA may have problems with learning and development... expand

This study will evaluate patients with methylmalonic acidemia (MMA) to learn more about the genetic causes of the various types of this inherited metabolic disorder and the medical complications associated with it. People with MMA may have problems with learning and development and kidney malfunctioning. They can become seriously ill, sometimes with little warning. There is no cure for any MMA, but special diets and vitamin therapies are used for treatment. Patients between 2 and 70 years of age with MMA may be eligible for this study. Participants are admitted to the NIH Clinical Center for 4-5 days each year for 5-10 years for the following tests and procedures: - Medical history, physical examination, eye examination - Consultations from dentists and specialists in the nervous system, digestive tract, endocrine, and kidney, as needed. - 24-hour urine collection to examine for methylmalonic acid, other acids, sugar, and proteins for measuring kidney function. - Blood test to assess liver and thyroid function, blood counts and blood chemistries, methylmalonic acid levels, and for genetic tests and basic research studies. - Blood test to measure growth hormone production. For this test, a very small amount of blood is collected overnight (every 20-30 minutes from 8:00 PM to 8:00 AM) through an intravenous catheter (plastic tube placed in a vein). The total amount of blood drawn is approximately 1 tablespoon. Patients who have stopped growing or whose weight does not permit collection of 1 tablespoon of blood do not undergo this procedure. - Frequent blood pressure measurements, including overnight monitoring - Skin biopsy for cell culture (cells to grow in the laboratory for future testing). For this procedure, an area of skin is numbed with an anesthetic such as lidocaine. A 4-mm diameter circular area is then removed using a sharp punch and scissors. The wound is dressed and usually heals within a week. - Photographs of the face and body (wearing underwear) to help track growth and appearance. - Ultrasound of the kidneys - Hand x-ray to determine bone age - Dual energy x-ray absorptionometry (DEXA) scan to assess bone density. For the DEXA scan, the patient lies still on a table while the spine and hip are scanned using a small amount of radiation. Any patient who becomes seriously ill during the evaluation may be cared for at the NIH or transferred to another hospital if it is deemed advisable.

Type: Observational

Start Date: Jun 2004

open study

Pediatric cGVHD Symptom Scale
National Cancer Institute (NCI) Graft vs Host Disease
Background: Some children/adolescents who have had a stem cell transplant live with chronic graft-versus-host-disease (cGVHD). cGVHD is a side effect of the transplant that can cause multiple bothersome symptoms and negatively affect a child/adolescent squality of daily life.... expand

Background: Some children/adolescents who have had a stem cell transplant live with chronic graft-versus-host-disease (cGVHD). cGVHD is a side effect of the transplant that can cause multiple bothersome symptoms and negatively affect a child/adolescent squality of daily life. The questionnaires that measure thesymptoms caused by cGVHD are designed for adults. Children/adolescents may not describe their symptoms in the same way. The goal of this research is to improve the way we measure how bothersome these symptoms are for children/adolescents living with cGVHD. Objective: To develop a questionnaire (The Pediatric cGHVD Symptom Scale) for children/adolescents living with cGVHD to identify the symptoms they are experiencing and describe how bothersome those symptoms are to them. An additional goal is to design a parent/guardian companion questionnaire that can be used to capture the symptom experiences of very young children who may not be able to complete a questionnaire. Eligibility: Children/adolescents ages 5-17 who are receiving treatment for cGVHD after a stem cell transplant, and their parent/guardian.. Design: This study consists of 2 projects. Children/adolescents with cGVHD and their parent/guardianparticipants will be grouped by the child/adolescent s age: 5-7, 8-12, and 13-17. In project 1, participants will complete an age-appropriate questionnaire about cGVHD symptoms. The questionnaire will ask about the child/adolescent s physical functioning and emotional well-being. The parent/guardian will out fill out a companion questionnaire online. The child/adolescent will then review their completed questionnaire during an interview with a researcher and will be asked whether the questions about their symptoms were difficult to understand. The parent/guardian and child/adolescent will then be interviewed together to further explore their responses to the questionnaires. Interviews will be done in person, by phone, and online. . Based on what is learned through these interviews, the wording of the questionnaire will be improved for better comprehension and ease of response. In project 2, participants will complete this revised questionnairefor their age group along with some other questionnaires that ask about quality of life. Both the child/adolescent and parent/guardian will fill out the questionnaires online at three separate time points. In both projects, children/adolescents with cGVHD and their parent/guardian participants will be grouped by the child/adolescent s age: 5-7, 8-12, and 13-17. ...

Type: Observational

Start Date: Oct 2019

open study

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis...
National Cancer Institute (NCI) Neurofibromatosis 1 Plexiform Neurofibromas
Background: People with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (PNs) can have pain that affects their daily lives. This study aims to improve questionnaires that measure their pain, daily living, and physical functioning. Objectives: To examine... expand

Background: People with neurofibromatosis 1 (NF1) who have plexiform neurofibromas (PNs) can have pain that affects their daily lives. This study aims to improve questionnaires that measure their pain, daily living, and physical functioning. Objectives: To examine and improve questionnaires about daily living for people with NF1 and PNs. Eligibility: People ages 5 and older with NF1 and a PN Design: Participants will be screened with medical history. This study will have 2 phases. Phase 1 participants will talk about existing pain assessment questionnaires and how PNs affect their life. They will have group discussions of up to 8 people of a similar age with NF1 and PNs, or the parents of children with it. These will last about 90 minutes. Children ages 5 to 7 and their parents will have one-on-one meetings instead. These will last about 45 minutes. Discussions will be audiotaped. After the questionnaires have been changed, individual interviews will discuss the new wording, instructions, questions, and electronic format of the new forms. Phase 1 participants may be invited to Phase 2. Phase 2 participants will complete the new questionnaires. These may be pen-and-paper or electronic. The questionnaires will take about 30 minutes for adults and teens. Children will work one-on-one with a staff member and may need up to 45 minutes. A small group of participants will be complete the forms twice in clinic and 1 month later at home. Also, a small group who start a new pain treatment or have a dose increase in their treatment will complete the forms twice before the treatment change and 1 month later.

Type: Observational

Start Date: Nov 2015

open study

Treatment of Children With Recurrent Refractory Brain/Solid Tumors and Recurrent Ependymoma
The Hospital for Sick Children Recurrent Childhood CNS Tumor Ependymoma, Recurrent Childhood Childhood Solid Tumor
Many pediatric brain and solid tumors have altered epigenetic landscapes, and altered DNA methylation. As such this study is a Phase I/Ib study of combined 5'Azacitidine with an escalating dose of carboplatin for all recurrent/refractory pediatric brain and solid tumors. The... expand

Many pediatric brain and solid tumors have altered epigenetic landscapes, and altered DNA methylation. As such this study is a Phase I/Ib study of combined 5'Azacitidine with an escalating dose of carboplatin for all recurrent/refractory pediatric brain and solid tumors. The phase I component will establish with maximum tolerated dose of carboplatin with azacytidine. An expansion cohort will be recruited of up to 30 patients will follow consisting of 20 recurrent posterior fossa ependymoma and 10 recurrent supratentorial ependymoma.

Type: Interventional

Start Date: Aug 2017

open study

Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients
Bayer Mixed Tumor, Malignant
This study is designed to investigate whether the use of copanlisib is safe, feasible and beneficial to pediatric patients with solid solid tumors or lymphoma that are recurrent or refractory to standard therapy. expand

This study is designed to investigate whether the use of copanlisib is safe, feasible and beneficial to pediatric patients with solid solid tumors or lymphoma that are recurrent or refractory to standard therapy.

Type: Interventional

Start Date: Apr 2018

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