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Using Technology to Improve Care for Teens with Inflammatory Bowel Disease
Inflammatory bowel disease (IBD) affects the digestive system. Types of IBD include Ulcerative Colitis and Crohn's disease. The purpose of this study is to find out if text messaging can be used to help care for people with IBD. Volunteers must have a smartphone with text messaging.
Study Topics
Comparing Ways to Improve Blood Flow in Newborns' Lungs
The purpose of this study is to looks at two different methods to help increase blood flow in the lungs of newborn babies. One method uses a shunt, which is a small tube, and the other uses a stent, which is a tiny mesh-like device. We want to learn if one of the two options is better than the other.
Study Topics
Improving the Care and Health of Children, Adolescents, and Young Adults with Inflammatory Bowel Disease
The purpose of this study is to improve the care and health for children, teens, and young adults with IBD (Inflammatory Bowel Disease). We want to gather information from your regular care visits to help improve the care for patients with inflammatory bowel disease. For example, we could learn which type of medication works best for treating this disease.
Study Topics
Finding Signs of Hearing Problems in Babies with Cytomegalovirus Infection Present at Birth
The purpose of this study is to learn more about tests that can be used to show how a baby's hearing will be affected if they have cytomegalovirus (CMV) infection present at birth.
Study Topics
Infectious Diseases Screening and Prevention Children, Infants, Teen Health Ears, Nose, Throat, Mouth
Studying a New Medicine for Kids and Teens with Generalized Myasthenia Gravis
Generalized myasthenia gravis is a disease that makes muscles weak because the body's defense system attacks the spot where nerves and muscles connect. The purpose of this study is to learn more about a new medicine for kids and teens with this disease. Our researchers want to find out if the medicine is safe, how it works inside the body, and if it helps kids feel better and do everyday things more easily.
Study Topics
Rare Disease Brain and Memory Children, Infants, Teen Health
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
Preventing Chickenpox Among Children
The purpose of this study is to learn more about a new chickenpox vaccine for young children. Chickenpox, also called varicella, is common in children and can cause fever, itchy rashes, and serious infections. Doctors have safely used chickenpox vaccines in children for many years, but there is currently only one option available. This study will help researchers see how well a new vaccine works compared to the one already in use.
Study Topics
Healthy Volunteers Screening and Prevention Children, Infants, Teen Health
Studying Axatilimab for Children and Teens with Chronic Graft‑Versus‑Host Disease
Chronic graft‑versus‑host disease (GVHD) is a condition where donated stem cells attack the patient's body after a transplant, causing ongoing health problems. The purpose of this study is to learn more about the medicine axatilimab for children and teens with this condition. The goal is to see if this treatment works better and is safe compared to other medicines already used.
Study Topics
Cancer Cancer: Blood Cancer: Children, Teens Children, Infants, Teen Health
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 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
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