Does Your Child Have Progressive Familial Intrahepatic Cholestasis (PFIC)?

Why are we doing this research?

Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, to learn more about an investigational drug that may help treat the liver and control the itching in people who have Progressive Familial Intrahepatic Cholestasis (PFIC).

Investigational means that the drug is being tested and is not approved by the U.S. Food and Drug Administration (FDA).

Who can participate?

Infants, children and teens, 1 to 18 years old, who have PFIC may be eligible for participation.

However, those who have had any of the following CANNOT be in the study:


  • Surgery to divert bile flow
  • Liver transplant
  • A history or presence of liver disease other than PFIC, inflammatory bowel disease or any other intestinal disease



  • Gastroenterology - Digestive Tract - Stomach - Liver

What will happen in the study?


If you (if you are 18) or your child qualifies for this study, and you agree to participate, you will be in this study for about 1 year.

You or your child will first have a screening visit during which the study will be explained to you and some questions/tests will be performed. This visit will take a few hours to complete.

Next, if the tests from the screening are okay, you or your child will have a baseline visit. The baseline visit happens within 6 weeks of the screening and may take an hour-and-a-half to complete.

After the baseline, you or your child will enter the treatment phase of the study.

During the treatment phase, there are a total of 8 clinic visits with study staff at Cincinnati Children’s. Most clinic visits will take about an hour, but a few may take more or less time. Parents and/or guardians must accompany children under the age of 18 to all study visits.

You and/or your child will also complete 10 scheduled telephone calls, lasting about 15 to 30 minutes each.

You or your child will receive the study drug at each clinic visit and will also be given written instructions on when and how to take it. This is not a placebo-controlled study, so all eligible participants will receive the investigational study drug . All participants will take the study drug for 48 weeks.

  A detailed list of tests and procedures will be provided to anyone interested in learning more about this study. Some of the tests and procedures include:  

  • Demographic data: We will ask questions to collect information about you or your child, including birth date, gender, race and ethnicity (screening only). 
  • Medical and medication history: We want to know more about you or your child’s current and past medical history as well as know of any prior and current medications (screening only).
  • Ultrasound of liver: This is a machine that uses high frequency sound waves to create an image of the liver (screening only).
  • Genetic testing: Blood will be tested for any presence of a change in genes found in people with PFIC. This will only be done if there is no record of it in you or your child’s chart or if the record of it cannot be obtained from another hospital (screening only).
  • Physical exam: We will collect information including weight, height and vital signs (blood pressure, heart rate, breathing rate and temperature).
  • Blood and urine samples: The samples will be collected for routine testing. For visits when blood and urine are collected, you or your child will need to be fasting (no food or drink except for water) for 4 hours before the visit.
  • Electronic diary (eDiary): This looks like a cell phone and you or your child will use it to report how the itching is in the morning and before bed. You or your child will need to bring this to every study visit.
  •  Paper diary: You or your child will be asked to record all dates and times you took the study drug, how much, and what time you ate breakfast. You will also be asked to record any missed doses. You or your child will need to bring this to every study visit.
  • Questionnaires: You or your child will answer questions about how you have been feeling; if there are any changes to medications; quality of life; how itch affects you; and how often you itch, scratch or rub.
It’s important to remember that if you or your child chooses to take part in the study, you will need to come to all of the study visits and follow all of the instructions provided by study staff. 



Participants or parents interested in having their child participate 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?

Being in this study may not help you or your child right now. We hope the information learned from this study may help other people with PFIC later on.  

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

A detailed list of possible side effects will be provided to those participants, parents or guardians interested in knowing more about this study.

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

Participants may receive up to $750 ($75 for each of the 10 study visits) for their time and travel.  


Susan McMahan
Cincinnati Children’s Hospital Medical Center
Gastroenterology, Hepatology, & Nutrition
3333 Burnet Avenue
Cincinnati, OH 45229-3039

Study Doctor

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