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.
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.
1) To determine if 5 doses of Epo (Erythropoietin) 1000 U/kg (birth weight) intravenous (IV) reduces the rate of death or neurodevelopmental impairment (mild, moderate, or severe) at 24 months of age.
2) To assess safety of Epo.
3) To determine whether Epo decreases the severity of HIE-induced brain injury as evidenced by early MRI and plasma biomarkers of brain injury.
The collection of the research data we hope will help better screening, diagnosing procedures and treatment of brain injury in newborns and identify a connection between MR imaging and neurodevelopmental outcomes.
To evaluate the efficacy of tofacitinib based on remission in pediatric participants with moderately to severely active Ulcerative Colitis (UC).