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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
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
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
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
Studying a New Pneumococcal Disease Vaccine Among Healthy Toddlers
Your child is being asked to join this study because they are healthy, between 12 to 15 months old, and have had 3 doses of Prevnar 20®. The study will test a new vaccine to see if it can protect against a germ that causes infections in children. The goal is to check if the vaccine is safe and helps the body make antibodies to fight the germ.
Study Topics
Healthy Volunteers Infectious Diseases Screening and Prevention Children, Infants, Teen Health
Finding out if Antiviral Medicine Worked for Babies or Toddlers with Cytomegalovirus Infection
The purpose of this study is to learn more about the treatment with antiviral medicine for babies born with cytomegalovirus. We want to see if antiviral medicine has any impact on hearing, cancer risk, overall growth, or sexual development.
Study Topics
Infectious Diseases Children, Infants, Teen Health Ears, Nose, Throat, Mouth
A Study of the Drug Valacyclovir for Babies at Risk of Herpes
The purpose of this study is to learn more about the study drug Valacyclovir. We want to figure out the best amount of the study drug that is needed for babies who may have been exposed to herpes when they were born.
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
Using CTX001 to Treat Severe Sickle Cell Disease in Children
The purpose of this study is to learn more about using the study drug, CTX001, to treat sickle cell disease in children. We want to see how effective it is and if there are any side effects.
Study Topics
Collecting Data on Patients with Cystic Fibrosis
The purpose of this study is to gather information on people with cystic fibrosis through a patient registry. The patient registry helps track important changes in people with cystic fibrosis. It also helps doctors understand the disease better, improve treatments, and help patients live longer.
Study Topics
Genetic Disorders Lung Health 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
Comparing Treatments in Children Newly Diagnosed with Crohn's Disease
The purpose of this study is to learn more about why some children with Crohn's disease heal their bowel inflammation after one type of treatment and why others do not. Volunteers must have suspected Crohn's disease.
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
Treating Hyperactivity in Children with Down Syndrome using Guanfacine
The purpose of this study is to learn more about how well the study drug, guanfacine, can treat hyperactivity in children with Down syndrome.
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
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