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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
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
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
Study of Bempedoic Acid for Treating High Cholesterol in Children
Heterozygous familial hypercholesterolemia (HeFH) is an inherited genetic disorder. It causes dangerously high cholesterol levels. This study will see if bempedoic acid works at lowering cholesterol in kids with HeFH. Bempedoic has been tested in adults, but it has not been tested in children or young adults yet.
Study Topics
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
Learning More About Asthma and Coach McLungs, an Asthma Intervention
The purpose of this study is to learn more about asthma and to explore a new way to treat it with Coach McLungs. Coach McLungs is an asthma health communication model that allows patients and healthcare providers to work together to make health care related decisions. Your participation in this research will involve one interview.
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
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
Treating Childhood Seizures with Perampanel
The purpose of this study is to find out how well Perampanel works for treating seizures in children.
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
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
Concussion Symptoms in Young People Ages 11-17
The purpose of this study is to learn more about concussion symptoms in young people ages 11 to 17. This may help us develop future treatments.
Study Topics