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.
Hydroxychloroquine is given as an investigational drug to relatives of someone with type 1 diabetes who have positive diabetes antibodies in an attempt to prevent or delay the onset of type 1 diabetes. See website for more details https://www.trialnet.org/our-research/prevention-studies/hydroxychloroquine-hcq
This research is being done to test the safety and effectiveness of intranasal carbetocin to treat Prader Willi Syndrome.
To find out which of two commonly used IV fluids given in the Emergency Department for sepsis is most effective.
Two main purposes of the study: To see if serious bacterial infections occur less than once a year in each study subject. This will determine if study drug is effective. Also to find out if study drug is safe and tolerable for study subjects.
To demonstrate that an ultra-fast-acting formulation of insulin lispro is non-inferior to Humalog on measures of glycemic control when administered 0 to 2 minutes before eating in combination with basal insulin.
To find out the effects of tenofovir alafenamide (TAF) on children and adolescents with chronic hepatitis B.
To evaluate the efficacy of tofacitinib based on remission in pediatric participants with moderately to severely active Ulcerative Colitis (UC).