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Studying Digoxin to Help Treat Brain Cancer
The purpose of this study is to learn more about a medicine called digoxin. We want to see if this medicine can help stop a type of brain cancer called medulloblastoma from getting worse. The goal is to learn if this treatment can help and to understand any side effects it may cause. We are looking for volunteers who have been diagnosed with medulloblastoma.
Study Topics
Cancer Cancer: Brain, Nervous System, Spinal Cord Cancer: Rare Cancers Children, Infants, Teen Health
Studying a Medicine for Babies with Alagille Syndrome
The purpose of this study is to learn more about a medicine called Odevixibat for babies with Alagille Syndrome. We want to see if this medicine can safely help reduce itching in babies with this health condition.
Study Topics
Genetic Disorders Rare Disease Children, Infants, Teen Health
Studying an Exercise Program for Teens and Young Adults After Cancer Treatment
The purpose of this study is to find the best ways to help Hispanic or Latino/a teens and young adults who had cancer stay physically active at home. We will use a program that uses a Fitbit and social media to encourage exercise. The goal is to make sure the program fits the language and culture of the participants.
Study Topics
Cancer Cancer: Children, Teens Children, Infants, Teen Health
Studying the Medicine Apitegromab for Babies with Muscle Weakness
Spinal muscular atrophy is a disease that makes muscles weak and hard to move. The purpose of this study is to test a new medicine called apitegromab. We want to see if it is safe and helpful for babies under 2 years old who still have trouble moving even though they already got another treatment called survival motor neuron therapy.
Study Topics
Genetic Disorders Movement Disorder Neurology Disorders Rare Disease Bones, Joints, Muscles Children, Infants, Teen Health
Testing Seizure Medicine for Kids
The purpose of this study is to test a liquid medicine called zonisamide for kids with seizures. We want to see how the medicine moves in the body and if it is safe. The study is for kids aged 1 month to 17 years who have partial-onset seizures.
Study Topics
Testing Genes for Cystic Fibrosis Diabetes
The purpose of this study is to understand why people with cystic fibrosis get diabetes. Both diseases are caused by changes in DNA and other molecules in the blood. These changes can cause thick mucus in the pancreas, which damages cells that make insulin, leading to diabetes.
Study Topics
Genetic Disorders Lung Health Children, Infants, Teen Health Diabetes
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
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
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
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
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