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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
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 Upadacitinib for Children with Ulcerative Colitis
The purpose of this study is to learn more about the study drug upadacitinib for treating ulcerative colitis, a type of bowel disease. We want to find out if the study drug is safe and works well for children with ulcerative colitis who have not had a response to other types of treatment.
Study Topics
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
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
Studying a Treatment for Patients with Severe Sjögren'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
Studying a Treatment for Patients with Severe Sjögren'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
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
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
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
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
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