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Treating Neuroblastoma in Young People with Naxitamab
The purpose of this study is to test the safety and effectiveness of adding a new drug called naxitamab to the usual treatment for a type of cancer called neuroblastoma.
Study Topics
Cancer Cancer: Head, Neck Cancer: Thyroid, Other Glands Children, Infants, Teen Health
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
Studying a New Medicine for Juvenile Psoriatic Arthritis
Juvenile psoriatic arthritis is a disease in kids and teens that causes joint pain and swelling along with skin problems like red, scaly patches. The purpose of this study is to learn more about the medicine secukinumab for this disease. We will check if the medicine is safe and how the body handles it.
Study Topics
Arthritis, Lupus, and Autoimmune Disorders Children, Infants, Teen Health
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 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
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
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
Looking at Arm Size Differences in Kids with a Certain Type of Elbow Fracture
The purpose of this study is to gather information that might help us create a more accurate way to measure arm swelling when a child fractures their elbow. Using the same way to measure swelling might help doctors make decisions on treatment that can impact how kids heal from these types of injuries.
Study Topics
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
Helping Young Cancer Survivors Feel Better Using a Digital Tool
The purpose of this study is to learn more about a new digital tool to help adolescents and young adult cancer survivors manage their symptoms of depression. People in the study will use the tool, answer questions, and join a video call. The goal is to find out if the tool helps them manage their feelings better.
Study Topics
Mental Health Cancer Cancer: Children, Teens Children, Infants, Teen Health
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
Studying a New Vaccine for Infants and Toddlers
The purpose of this study is to learn about a new vaccine for healthy babies and toddlers. This vaccine is made to protect against a germ called pneumococcus, which can cause serious infections. The study will help researchers see how well the vaccine works and if it causes any side effects. We are looking for volunteers with a child who is 42 to 89 days old.
Study Topics
Healthy Volunteers Infectious Diseases Screening and Prevention Children, Infants, Teen Health
Tracking the Long-Term Safety of the Medicine AGAMREE
Duchenne muscular dystrophy (DMD) is a disease that makes muscles get weaker over time. The purpose of this study is to track the long-term safety of a medicine called AGAMREE for this disease. People in the study will have yearly checkups and answer questions about their health. We are looking for volunteers who have DMD and are currently taking AGAMREE.
Study Topics
Genetic Disorders Men's Health Rare Disease Bones, Joints, Muscles 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
Using Upadacitinib to Treat Arthritis in Children and Young People
The purpose of this study is to learn more about treating arthritis in children and young people with the study drug, upadacitinib. We want to see if this treatment works better than the usual treatment.
Study Topics
Arthritis, Lupus, and Autoimmune Disorders Children, Infants, Teen Health
Using Mavacamten to Treat Adolescents with Cardiomyopathy
Obstructive Hypertrophic Cardiomyopathy (HCM) is a heart condition that makes it hard for the heart to pump blood and may make a person feel weak and tired. The purpose of this study is to see if the study drug, mavacamten, helps young people with obstructive HCM.
Study Topics