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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
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
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
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
Studying Risk Factors for Type 2 Diabetes in Youth
Type 2 diabetes is a health problem where the body has too much sugar in the blood. This study wants to find out what makes young people more likely to get this condition early. The information we learn will help us create future studies on how to stop and treat type 2 diabetes in young people.
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
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
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
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