Search Studies
Studying the Medicine Migalastat for Children with Fabry Disease
Fabry disease is a rare genetic condition where the body cannot break down certain fats, causing them to build up and damage organs like the heart, kidneys, and nerves. The purpose of this study is to learn more about the medicine migalastat for this condition. Our researchers want to see if it is safe and helpful for children.
Study Topics
Genetic Disorders Rare Disease 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
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. All study visits and consultations will be done virtually.
Study Topics
Mental Health Cancer Cancer: Children, Teens Children, Infants, Teen Health
Studying Letermovir in Children and Teens After Kidney Transplant
The purpose of this study is to learn more about a medicine called letermovir among children and teens who have had a kidney transplant. Researchers want to see how the medicine works in the body and if it is safe. This will help doctors find the right dose for young patients.
Study Topics
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
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
Learning More About Chronic Kidney Disease
The purpose of this study is to find out what helps kidneys stay healthy and what makes them stop working well. Our researchers will look at how kidney problems can affect the brain, behavior, heart, growth, and overall health. We hope to learn more about how to make medical care better for kids, teenagers and young adults.
Study Topics
Studying New Medicines for Inflammatory Bowel Disease
Inflammatory Bowel Disease is a condition that causes swelling and pain in the digestive tract, making it hard for your body to digest food normally. The purpose of this study is to compare new medicines for this condition. Our researchers want to see if the medicines are safe and helpful for people who did not get better with other treatments.
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
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
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