This is a randomized Double-blind Placebo-controlled Phase 3 Study to determine whether a study drug called maralixibat is safe and effective in treating itchy skin (pruritus) in children with Progressive Familial Intrahepatic Cholestasis (PFIC). There is currently no treatment approved for PFIC and available medical approaches have limited success.
Randomized means by chance, like the flip of a coin. Subjects will be randomly assigned to one of the two arms of the study. Subjects will have a 50:50 chance of receiving maralixibat as compared to placebo.
Double blinded means a study in which neither the investigators nor the subjects know which intervention the subject is receiving.
A Phase 3 study is large scale trial to confirm and expand information on safety and usefulness of a new drug.
Subjects who qualify to participate in this study will be randomly assigned to receive the study drug or a placebo. The dose of the study drug will be gradually increased during the study upto a tolerable dose . Subjects will continue to take the medication for about 5 months. Subjects will have approximately 9 study visits over 8 months in this study. Subjects will also be asked questions about health and complete questionnaires at the study visits and as well as at home. A Physical exam, Electrocardiogram, Liver ultrasound will be performed at some study visits. A blood and urine sample will also be collected at some of the study visits.
It is possible that the maralixibat may or may not improve symptoms from PFIC. Even if there is no benefit, other children may benefit from what is learned in this study.