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 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.
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.
Determine how potentially stressful situations, including prematurity, hospitalizations, maternal mental health and rapid repeat births can impact a baby’s development in the first 12 months of their life.
The purpose of this study is to understand the impact of the COVID-19 pandemic on vulnerable families in Los Angeles, including those caring for a child with special health care needs. Information from this research will be used to understand what the key resource and service needs of families are at this time as well as identify interventions that can be developed or adapted to address these needs.