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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
New Treatment for ANCA Vasculitis in Kids and Teens
Active ANCA-associated Vasculitis is a disease where the body's immune system accidentally attacks its own small to medium-sized blood vessels. This makes the blood vessels swollen and damaged. The purpose of this study is to learn more about a treatment to help kids and teens with this disease. We want to see if the treatment called avacopan is safe and helpful.
Study Topics
Rare Disease Arthritis, Lupus, and Autoimmune Disorders Children, Infants, Teen Health
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
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
Helping Families Make Kidney Treatment Choices
Nephrotic syndrome is a kidney problem that makes your body lose protein in your pee, causing swelling and other health issues. This study aims to help families choose the best treatments for this condition. We are looking for volunteers with children who have nephrotic syndrome. By sharing your experiences, you will help us find better ways to support families in making treatment decisions.
Study Topics
Using Special Eyeglasses to Detect Absence Seizures in Children
The purpose of this study is to learn more about a new lupus nephritis treatment called Anifrolumab. We want to find out how well it works and what side effects it may have.
Study Topics
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
Comparing Treatments for Severe Aplastic Anemia in Young People
The purpose of this study is to find out whether immune suppressive therapy or bone marrow transplant is better for treating a disease called severe aplastic anemia in young patients who don't have a matched sibling donor.
Study Topics
Rare Disease Cancer Cancer: Blood Cancer: Children, Teens 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
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
Using Tagraxofusp to Treat Cancer in Young People
The purpose of this study is to see if adding the study drug, Tagraxofusp, to the usual treatment helps people with refractory (difficult to treat) and/or relapsed (cancer that has returned after previous treatment) hematologic malignancy (cancer). We also want to learn more about any side effects of the study drug.
Study Topics
Cancer Cancer: Blood Cancer: Children, Teens Children, Infants, Teen Health
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
Learning More About Duchenne Muscular Dystrophy Patients and Their Treatment
The purpose of this study is to get a better understanding of patients with Duchenne muscular dystrophy who were treated with Eteplirsen, Golodirsen, or Casimersen. We want to learn more about how well those medications worked for treating Duchenne muscular dystrophy.
Study Topics
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
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