A Placebo-controlled study of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
To find out the effects of Maralixibat on the treatment of PFIC.
To find out the effects of Maralixibat on the treatment of PFIC.
To see if eye tracking (ET) or electroencephalogram (EEG) assessments have potential use in future clinical trials of Autism Spectrum Disorders (ASD).
We want to learn more about how cancer treatment, such as immunotherapy (i.e., CAR T cell treatment), affects the brain, learning, and behavior in patients with Acute Lymphoblastic Leukemia (ALL).
To evaluate long-term safety of Humira in pediatric patients (between the ages of 6 and 17 years inclusive at the time of enrollment) with moderately to severely active CD who are prescribed and treated in accordance with the approved local Humira product label under the conditions of routine clinical setting.
The objective of this study is to better understand how a single ventricular heart with Fontan physiology may affect brain health.
The first Phase IIb clinical trial in Down syndrome regression disorder (DSRD). An open-label, randomized study comparing the safety and efficacy of lorazepam, intravenous immunoglobulin (IVIg) and the Janus Kinase inhibitor (JAK inhibitor), tofacitinib.
Safety of Fecal Microbiota Transplantation (FMT) in children with Crohn's disease in remission.
To know if stool transplant from a healthy person into an ulcerative colitis patient can help treat the disease.
Develop better ways of screening and diagnosing certain types of brain injuries in infants and children who are born with and without congenital heart disease.
To better understand the genetic changes that make children susceptible to the development of autism spectrum disorder, and to study how these changes influence clinical outcomes.