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