MRX-502 MRX-502: Randomized Double-blind placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) - MARCH-PFIC

NCT03905330

MRX-502: Randomized Double-blind placebo-controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC) - MARCH-PFIC

Associated Conditions

dermatology

Principal Investigator

Sponsor

Mirium Pharmaceuticals

The goal of this study is to determine whether maralixibat is safe and effective in the treatment of Progressive Familial Intrapeatic Cholestasis (PFIC). Currently, there is no approved treatment for PFIC and available medical approaches have limited efficacy. The purpose of this study is to help answer the following questions: (1) Can maralixibat help patients with PFIC?; (2) How safe is maralixibat and what side effects might be related to it? ; and (3) How does maralixibat compare to placebo (a look-alike solution that has no active drug).