186 matching studies

Study is registered in ResearchMatch
Sponsor Condition of Interest
A Study of Lasmiditan (LY573144) Treatment in Children Aged 6 to 17 With Migraine
Eli Lilly and Company Migraine
The reason for this study is to see if lasmiditan is safe and effective in children aged 6 to 17 with migraine. The study will last up to 20 weeks and may include up to 4 visits. expand

The reason for this study is to see if lasmiditan is safe and effective in children aged 6 to 17 with migraine. The study will last up to 20 weeks and may include up to 4 visits.

Type: Interventional

Start Date: Jun 2020

open study

Positive Psychology Intervention to Treat Diabetes Distress in Teens With Type 1 Diabetes
Vanderbilt University Medical Center Type 1 Diabetes Mellitus
The treatment regimen for type 1 diabetes is complex and demanding, and many adolescents experience diabetes distress related to the daily demands of diabetes care, which can cause problems with diabetes management and glycemic control. The proposed study will conduct a multisite,... expand

The treatment regimen for type 1 diabetes is complex and demanding, and many adolescents experience diabetes distress related to the daily demands of diabetes care, which can cause problems with diabetes management and glycemic control. The proposed study will conduct a multisite, randomized trial to test the effects of a positive psychology intervention aimed at treating diabetes distress and improving glycemic outcomes. The potential benefits include helping adolescents achieve better glycemic control, improved self-management, and psychosocial outcomes

Type: Interventional

Start Date: Dec 2019

open study

Efficacy and Safety of MK-1654 in Infants (MK-1654-004)
Merck Sharp & Dohme LLC Respiratory Syncytial Virus Infection
The primary objectives of this phase 2b/3 double-blind, randomized, placebo-controlled study are to evaluate the efficacy and safety of MK-1654 in healthy pre-term and full-term infants. It is hypothesized that MK-1654 will reduce the incidence of respiratory syncytial virus... expand

The primary objectives of this phase 2b/3 double-blind, randomized, placebo-controlled study are to evaluate the efficacy and safety of MK-1654 in healthy pre-term and full-term infants. It is hypothesized that MK-1654 will reduce the incidence of respiratory syncytial virus (RSV)-associated medically attended lower respiratory infection (MALRI) from Days 1 through 150 postdose compared to placebo.

Type: Interventional

Start Date: Apr 2021

open study

A Study of Axatilimab at 3 Different Doses in Participants With Chronic Graft Versus Host Disease (cGVHD)
Syndax Pharmaceuticals Chronic Graft-versus-host-disease
This is a Phase 2 study to evaluate the efficacy, safety, and tolerability of axatilimab at 3 different dose levels in participants with recurrent or refractory active chronic graft versus host disease (cGVHD) who have received at least 2 prior lines of systemic therapy. expand

This is a Phase 2 study to evaluate the efficacy, safety, and tolerability of axatilimab at 3 different dose levels in participants with recurrent or refractory active chronic graft versus host disease (cGVHD) who have received at least 2 prior lines of systemic therapy.

Type: Interventional

Start Date: Mar 2021

open study

Mesenchymal Stromal Cells for Infants With Congenital Heart Disease (MedCaP)
Catherine Bollard Congenital Heart Disease (CHD)
The proposed study will be a prospective, open-label, single-center, safety and feasibility phase 1 trial of allogeneic bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery though cardiopulmonary bypass (CPB) using a homogeneous population of infants with congenital... expand

The proposed study will be a prospective, open-label, single-center, safety and feasibility phase 1 trial of allogeneic bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery though cardiopulmonary bypass (CPB) using a homogeneous population of infants with congenital heart disease (CHD) who will be undergoing a two-ventricle repair within the first six months of life

Type: Interventional

Start Date: Jul 2020

open study

A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With...
Pediatric Brain Tumor Consortium Low Grade Glioma (LGG) of Brain With BRAF Aberration High Grade Glioma (HGG) of the Brain With BRAF Aberration Low Grade Glioma of Brain With Neurofibromatosis Type 1
This phase I/II trial is designed to study the side effects, best dose and efficacy of adding hydroxychloroquine to dabrafenib and/or trametinib in children with low grade or high grade brain tumors previously treated with similar drugs that did not respond completely (progressive)... expand

This phase I/II trial is designed to study the side effects, best dose and efficacy of adding hydroxychloroquine to dabrafenib and/or trametinib in children with low grade or high grade brain tumors previously treated with similar drugs that did not respond completely (progressive) or tumors that came back while receiving a similar agent (recurrent). Patients must also have specific genetic mutations including BRAF V600 mutations or BRAF fusion/duplication, with or without neurofibromatosis type 1. Neurofibromatosis type 1 is an inherited genetic condition that causes tumors to grow on nerve tissue. Hydroxychloroquine, works in different ways to stop the growth of tumor cells by killing the cells or stopping them from dividing. Trametinib and dabrafenib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving hydroxychloroquine with trametinib and/or dabrafenib may lower the chance of brain tumors growing or spreading compared to usual treatments.

Type: Interventional

Start Date: Nov 2019

open study

A Study of Selexipag as Add-On Treatment to Standard of Care in Children With Pulmonary Arterial Hypertension
Actelion Hypertension, Pulmonary
The purpose of this study is to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with Pulmonary Arterial Hypertension (PAH) in comparison to placebo. expand

The purpose of this study is to evaluate whether the addition of selexipag to standard of care treatment delays disease progression in children with Pulmonary Arterial Hypertension (PAH) in comparison to placebo.

Type: Interventional

Start Date: Jan 2020

open study

Dabrafenib Combined With Trametinib After Radiation Therapy in Treating Patients With Newly-Diagnosed...
National Cancer Institute (NCI) Anaplastic Astrocytoma Anaplastic Astrocytoma, Not Otherwise Specified Anaplastic Ganglioglioma Anaplastic Pleomorphic Xanthoastrocytoma Glioblastoma
This phase II trial studies how well the combination of dabrafenib and trametinib works after radiation therapy in children and young adults with high grade glioma who have a genetic change called BRAF V600 mutation. Radiation therapy uses high energy rays to kill tumor cells... expand

This phase II trial studies how well the combination of dabrafenib and trametinib works after radiation therapy in children and young adults with high grade glioma who have a genetic change called BRAF V600 mutation. Radiation therapy uses high energy rays to kill tumor cells and reduce the size of tumors. Dabrafenib and trametinib may stop the growth of tumor cells by blocking BRAF and MEK, respectively, which are enzymes that tumor cells need for their growth. Giving dabrafenib with trametinib after radiation therapy may work better than treatments used in the past in patients with newly-diagnosed BRAF V600-mutant high-grade glioma.

Type: Interventional

Start Date: Oct 2019

open study

Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid...
Pfizer Ewing Sarcoma Solid Tumors Rhabdoid Tumor Rhabdomyosarcoma Neuroblastoma
This study will evaluate palbociclib in combination with chemotherapy (temozolomide with irinotecan and/or topotecan with cyclophosphamide) in children, adolescents and young adults with recurrent or refractory solid tumors. The main purpose of phase 1 portion of this study... expand

This study will evaluate palbociclib in combination with chemotherapy (temozolomide with irinotecan and/or topotecan with cyclophosphamide) in children, adolescents and young adults with recurrent or refractory solid tumors. The main purpose of phase 1 portion of this study is to evaluate the safety of palbociclib in combination with chemotherapy in order to estimate the maximum tolerated dose. The main purpose of phase 2 portion is to compare the efficacy of palbociclib in combination with irinotecan and temozolomide vs irinotecan and temozolomide alone in the treatment of children, adolescents, and young adults with recurrent or refractory Ewing sarcoma (EWS). Pharmacokinetics and efficacy of palbociclib in combination with chemotherapy will be evaluated.

Type: Interventional

Start Date: May 2019

open study

Efficacy and Safety of KD025 in Subjects With cGVHD After At Least 2 Prior Lines of Systemic Therapy
Kadmon Corporation, LLC Chronic Graft-versus-host-disease
This is a Phase 2, randomized, multicenter study to evaluate the efficacy and safety of KD025 in subjects with Chronic Graft Versus Host Disease (cGVHD) after at least 2 prior lines of systemic therapy expand

This is a Phase 2, randomized, multicenter study to evaluate the efficacy and safety of KD025 in subjects with Chronic Graft Versus Host Disease (cGVHD) after at least 2 prior lines of systemic therapy

Type: Interventional

Start Date: Oct 2018

open study

Study of Dose Confirmation and Safety of Crizanlizumab in Pediatric Sickle Cell Disease Patients
Novartis Pharmaceuticals Sickle Cell Disease (SCD)
The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 6 months to <18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years. The efficacy and... expand

The purpose of the Phase 2 CSEG101B2201 study is to confirm and to establish appropriate dosing and to evaluate the safety in pediatric participants ages 6 months to <18 years with a history of VOC with or without HU/HC, receiving crizanlizumab for 2 years. The efficacy and safety of crizanlizumab was already demonstrated in adults with sickle cell disease. The approach is to extrapolate from the PK/pharmacodynamics (PD) already established in the adult population. The study is designed as a Phase II, multicenter, open-label study.

Type: Interventional

Start Date: Oct 2018

open study

Nerivio Device for Treatment of New Daily Headache Persistence (NDHP)
Children's National Research Institute New Daily Persistent Headache (NDPH)
The goal of this study is to examine the effects of the Remote Electrical Neuromodulation (REN) device on adolescents ages 12-17 who have been diagnosed with New Daily Persistent Headache (NDPH). Pediatric patients with a diagnosis of new daily persistent headache are typically... expand

The goal of this study is to examine the effects of the Remote Electrical Neuromodulation (REN) device on adolescents ages 12-17 who have been diagnosed with New Daily Persistent Headache (NDPH). Pediatric patients with a diagnosis of new daily persistent headache are typically resistant to standard pharmacologic treatments and often experience systemic side effects related to medications; thus, REN offers the potential for an exciting new treatment option for patients with refractory headache disorders. The device delivers transcutaneous electrical stimulation to the upper arm to induce conditioned pain modulation (CPM) that activates a descending endogenous analgesic mechanism. Ultimately, the investigators hope to gain insights into the safety and efficacy of Nerivio™ for the acute treatment of NDPH in adolescents. The goal of this study is to demonstrate headache relief without unexpected device-related adverse effects

Type: Interventional

Start Date: Feb 2022

open study

A Phase 1/2/3 Study to Evaluate the Safety, Tolerability, and Immunogenicity of an RNA Vaccine Candidate...
BioNTech SE SARS-CoV-2 Infection, COVID-19
This is a Phase 1/2/3 study in healthy children and young adults. Dependent upon safety and/or immunogenicity data generated during the course of this study, and the resulting assessment of benefit-risk, the safety, tolerability, and immunogenicity of BNT162b2 in participants... expand

This is a Phase 1/2/3 study in healthy children and young adults. Dependent upon safety and/or immunogenicity data generated during the course of this study, and the resulting assessment of benefit-risk, the safety, tolerability, and immunogenicity of BNT162b2 in participants <6 months of age may subsequently be evaluated.

Type: Interventional

Start Date: Mar 2021

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

A Feasibility and Safety Study of Dual Specificity CD19 and CD22 CAR-T Cell Immunotherapy for CD19+CD22+...
Seattle Children's Hospital Leukemia Lymphoma
Patients with relapsed or refractory leukemia often develop resistance to chemotherapy and some patients who relapse following CD19 directed therapy relapse with CD19 negative leukemia. For this reason, the investigators are attempting to use T-cells obtained directly from... expand

Patients with relapsed or refractory leukemia often develop resistance to chemotherapy and some patients who relapse following CD19 directed therapy relapse with CD19 negative leukemia. For this reason, the investigators are attempting to use T-cells obtained directly from the patient, which can be genetically modified to express two chimeric antigen receptors (CARs). One is to recognize CD19 and the other is to recognize CD22, both of which are proteins expressed on the surface of the leukemic cell in patients with CD19+CD22+ leukemia. The CAR enables the T-cell to recognize and kill the leukemic cell through recognition of CD19 and CD22. This is a phase 1 study designed to determine the safety of the CAR+ T-cells and the feasibility of making enough to treat patients with CD19+CD22+ leukemia.

Type: Interventional

Start Date: Nov 2017

open study

A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von...
Baxalta now part of Shire Von Willebrand Disease
The main aim of the study is to check effectiveness, side effects, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric participants (less than (<)18 years of age) with... expand

The main aim of the study is to check effectiveness, side effects, and tolerability of recombinant von Willebrand Factor (rVWF), with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric participants (less than (<)18 years of age) with severe hereditary von Willebrand disease (VWD). The participants will be treated with rVWF for 12-18 months. Their von Willebrand Disease will be treated by their doctor according to their doctor's usual clinical practice. During the study, participants will be followed up at clinics or over telephone calls.

Type: Interventional

Start Date: Dec 2017

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 Phase 3 study with an open-label single-arm extension period to evaluate pharmacokinetics (PK), safety and efficacy of macitentan in children with pulmonary arterial hypertension (PAH). expand

This is a prospective, multicenter, open-label, randomized, controlled, parallel Phase 3 study with an open-label single-arm extension period to evaluate pharmacokinetics (PK), safety and efficacy of macitentan in children with pulmonary arterial hypertension (PAH).

Type: Interventional

Start Date: Oct 2017

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

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 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

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

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

Type: Observational

Start Date: Mar 2020

open study

Fimepinostat in Treating Brain Tumors in Children and Young Adults
Sabine Mueller, MD, PhD Diffuse Intrinsic Pontine Glioma Recurrent Anaplastic Astrocytoma Recurrent Glioblastoma Recurrent Malignant Glioma Recurrent Medulloblastoma
This trial studies how well fimepinostat works in treating patients with newly diagnosed diffuse intrinsic pontine glioma, or medulloblastoma, or high-grade glioma that have come back. Fimepinostat may stop the growth of tumor cells by blocking some of the enzymes needed for... expand

This trial studies how well fimepinostat works in treating patients with newly diagnosed diffuse intrinsic pontine glioma, or medulloblastoma, or high-grade glioma that have come back. Fimepinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Type: Interventional

Start Date: Aug 2019

open study

A Study to Evaluate Isavuconazonium Sulfate for the Treatment of Invasive Aspergillosis (IA) or Invasive...
Astellas Pharma Global Development, Inc. Invasive Mucormycosis Invasive Aspergillosis
The purpose of this study is to evaluate the safety, tolerability, and efficacy of isavuconazonium sulfate in pediatric participants. expand

The purpose of this study is to evaluate the safety, tolerability, and efficacy of isavuconazonium sulfate in pediatric participants.

Type: Interventional

Start Date: Aug 2019

open study

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

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

Type: Observational

Start Date: Oct 2015

open study

Liver Disease in Urea Cycle Disorders
Baylor College of Medicine Urea Cycle Disorder Ornithine Transcarbamylase Deficiency Citrullinemia 1 ARGI Deficiency ASL Deficiency
This is a multi-center, cross-sectional study to assess risk for liver fibrosis and hepatic injury in individuals with urea cycle disorders (UCDs) using serum biomarkers, Fibroscan, and MRE. This study will be conducted at 5 sites of the Urea Cycle Disorders Consortium: Baylor... expand

This is a multi-center, cross-sectional study to assess risk for liver fibrosis and hepatic injury in individuals with urea cycle disorders (UCDs) using serum biomarkers, Fibroscan, and MRE. This study will be conducted at 5 sites of the Urea Cycle Disorders Consortium: Baylor College of Medicine in Houston, TX, Seattle Children's Hospital in Seattle, WA, Children's Hospital Colorado in Aurora, CO, Children's Hospital of Philadelphia in Philadelphia, PA, and Children's National Medical Center in Washington D.C.

Type: Observational

Start Date: Nov 2021

open study