Clinical Trials / Research Studies
Clinical Trials / Research Studies

Progressive Familial Intrahepatic Cholestasis (PFIC) Study for Children and Teens 1 to 17 Years Old

Why are we doing this research?

Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, to determine the safety and effectiveness of the study drug maralixibat as a treatment for children and teens with PFIC. In previous studies, maralixibat was shown to improve conditions in a small number of patients with PFIC. In this study, we are enrolling a larger number of children and teens.

At the end of this study, participants will have the option to enroll into the long-term extension study (MRX- 503). During the extension study, all participants will receive the study drug.

Who can participate?

Children and teens 1 to 17 years old who have been diagnosed with PFIC may be eligible to participate.


  • Gastroenterology, Hepatology and Nutrition

What will happen in the study?

This study includes 10 study visits to Cincinnati Children’s over a period of 8 months, as well as 9 scheduled phone calls. Here are some of the things that will happen in this study:

Participants will:

  • Complete tests and procedures, including physical exams, ultrasounds of the liver, and blood and urine tests.
  • Be randomized, similar to flipping a coin, into one of the dosing groups to either receive the study drug or placebo (a look-alike solution with no active drug).

Parents or guardians will:

  • Receive the supply of either the study drug or placebo, which will be given as an oral solution (liquid form) twice a day at home. Once their child’s maximum dose has been established, parents will give their child that dose for a minimum of 20 weeks and no more than 22 weeks.
  • Be asked to complete a study diary two times each day throughout the study.
  • Bring their child in for assessments and will be contacted via phone to review study-related information.

You will be given a consent form that thoroughly explains all of the details of the study. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered.

What are the good things that can happen from this research?

Your child may not have a direct medical benefit. However, at the end of this study, we hope to have more information about how maralixibat works and how children handle it. This may help other children with PFIC in the future.

What are the bad things that can happen from this research?

Possible risks and discomforts will be discussed with parents or guardians interested in learning more about the study.

Will you/your child be paid to be in this research study?

Families will receive:

  • Up to $1,035 for their time and effort
  • Reimbursement for travel and travel-related expenses


Contact Us.Sandi Bechtol, RN
Cincinnati Children’s Hospital Medical Center
3333 Burnet Ave.
Cincinnati, OH 45229-3039

Study Doctor

Contact Us.Alexander Miethke, MD
Gastroenterology, Hepatology & Nutrition
Cincinnati Children’s Hospital Medical Center