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Collecting Data from Hemophilia Patients
The purpose of this study is to collect data on a blood disorder called hemophilia. The data will be used to improve the care of patients.
Study Topics
Heart Health Cancer Cancer: Children, Teens Children, Infants, Teen Health
Study to See How Well the Study Drug EPX-100 Works for Treating Dravet Syndrome
The purpose of this study is to see how well the study drug, EPX-100, works for treating Dravet syndrome. We also want to learn more about possible side effects.
Study Topics
Learning More About Treating Low-Grade Glioma with Vinblastine and Selumetinib
The purpose of this study is to learn more about treating low-grade gliomas. We want to find the highest dose of Vinblastine that can be given safely along with Selumetinib. We also want to find out if Selumetinib and Vinblastine combined work better than Selumetinib alone. Studies like this can help improve treatment and quality of life.
Study Topics
Cancer Cancer: Brain, Nervous System, Spinal Cord Children, Infants, Teen Health
Learning More About Treating Migraine in Children and Young Adults with Rimegepant
The purpose of this study is to learn how well the study drug, Rimegepant, treats migraines in children and young adults. We also want to learn more about the possible side effects of the study drug.
Study Topics
Study of Selinexor for Treating High-Grade Glioma in Pediatric Patients
A glioma is a type of cancer that occurs in the brain or spine. Diffuse intrinsic pontine glioma is a type of high-grade glioma. The purpose of this study is to look at how well selinexor works when given to children and young adults with high-grade glioma or diffuse intrinsic pontine glioma. We also want to find the highest dose of selinexor that we can give safely along with radiation therapy.
Study Topics
Cancer Cancer: Brain, Nervous System, Spinal Cord Children, Infants, Teen Health
Finding Out How Well Rimegepant Works for Treating Migraines in People Ages 12 to 17
The purpose of this study is to find out how well the study drug, Rimegepant, works for treating migraines in people age 12 to 17. We also want to find out how safe it is in children and young people.
Study Topics
Neurology Disorders Pain Brain and Memory Children, Infants, Teen Health
Learning More About Treating Childhood Absence Epilepsy and Juvenile Absence Epilepsy with Brivaracetam
The purpose of this study is to see how well the study drug, brivaracetam, treats childhood absence epilepsy or juvenile absence epilepsy. We also want to learn more about any possible side effects it may cause.
Study Topics
High Blood Pressure in Children and Teenagers
The purpose of this study is to understand what causes high blood pressure in young people. We also want to learn more about how high blood pressure can cause damage to the heart and kidneys.
Study Topics
Healthy Volunteers Heart Health Children, Infants, Teen Health
Combination Chemotherapy for Newly Diagnosed and Relapsed Wilms Tumor
The purpose of this study is to find out if we can improve treatment for those with Wilms tumors. We will be testing three different combinations of radiation and chemotherapy. The combination you receive will depend on your type of Wilms tumor.
Study Topics
Cancer Cancer: Kidney, Liver, Pancreas Children, Infants, Teen Health
Head Trauma Among Student Athletes
We want to learn about how head movement during a contact sport-related hit to the head affects the brain. Contact sports include those which may involve contact to the head during play such as football and soccer.
Study Topics
Healthy Volunteers Brain and Memory Children, Infants, Teen Health
Concussion Symptoms in Young People Ages 11-17
The purpose of this study is to learn more about concussion symptoms in young people ages 11 to 17. This may help us develop future treatments.
Study Topics
Using Special Eyeglasses to Detect Absence Seizures in Children
The purpose of this study is to learn more about a new type of eyeglasses. We want to find out if eyeglasses can tell if a child is having absence seizures or having attentional difficulties.
Study Topics
Study of Bempedoic Acid for Treating High Cholesterol in Children
Heterozygous familial hypercholesterolemia (HeFH) is an inherited genetic disorder. It causes dangerously high cholesterol levels. This study will see if bempedoic acid works at lowering cholesterol in kids with HeFH. Bempedoic has been tested in adults, but it has not been tested in children or young adults yet.
Study Topics
Can You Help the Tooth Fairy?
The purpose of this study is to learn more about how early life exposures can affect a child's or young adult's health. These exposures can include different things such as medications and foods. This study will be done completely online. The required baby tooth and saliva samples will be sent in using prepaid envelopes.
Study Topics
Healthy Volunteers Allergies and Immunity Stomach and Intestines Children, Infants, Teen Health
How Salt Affects Blood Pressure in Young Adults Who Were Born Prematurely
The purpose of this study is to learn about how salt in the diet affects blood pressure in young adults who were born prematurely. We also want to find out if the medication, allopurinol, can change how salt in the diet affects blood pressure.
Study Topics
Healthy Volunteers Heart Health Kidneys, Liver, Pancreas Children, Infants, Teen Health
Learning More About Intestinal Health in Infants and Children with a New Imaging Device
The purpose of this study is to learn more about a new type of imaging device called the iThera MSOT Acuity. We want to find out how well the device works for measuring intestinal health.
Study Topics
Healthy Volunteers Stomach and Intestines Children, Infants, Teen Health
Treating Dravet Syndrome with Fenfluramine Hydrochloride
The purpose of this study is to find out how well the study drug, fenfluramine hydrochloride, works for treating Dravet syndrome. We also want to learn more about side effects that may be caused by the study drug.
Study Topics
A Study of Telehealth to Treat Children with Medical Complexity
The purpose of this study is to learn more about using telehealth visits for children with medical complexity. We want to find out if telehealth visits can improve the health of children with chronic health problems.
Study Topics
New Migraine Medicine for Teens
The purpose of this study is to learn more about a new medicine called Rimegepant to help teenagers with chronic migraine. We want to see if this medicine is safe and useful to prevent chronic migraine.
Study Topics
Studying Risk Factors for Type 2 Diabetes in Youth
Type 2 diabetes is a health problem where the body has too much sugar in the blood. This study wants to find out what makes young people more likely to get this condition early. The information we learn will help us create future studies on how to stop and treat type 2 diabetes in young people.
Study Topics