Active not recruitingPHASE3INTERVENTIONAL

Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)

This research trial, called EXPAND, is investigating a new treatment called maralixibat for children and adults who have a rare form of liver disease that leads to long-lasting and troublesome itching. This type of liver disease is called cholestatic liver disease, and it's not one of the more common types like ALGS, PFIC, PBC, or PSC. Participants are being sought who have tried other anti-itch treatments without success and who currently have no other treatment options available. The main goal is to find out if maralixibat is both safe to use and effective at reducing the severe itching associated with this condition. The study is in its final phase (Phase 3) and is taking place in many locations across North America, Europe, the Middle East, and South America.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Mirum Pharmaceuticals, Inc.

Enrolment target

Start

14 Oct 2024

Estimated completion

01 Feb 2027

Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)

What is this study about?

This study is called EXPAND, and it's looking into a new medicine called maralixibat. It's designed for people, both children (from 6 months old) and adults, who have a specific kind of liver problem called cholestatic liver disease. This condition can cause a really uncomfortable symptom: persistent and severe itching, which hasn't got better with other treatments. The specific types of liver disease being studied are rare and don't include more common ones like ALGS, PFIC, PBC, or PSC.

The main idea behind this study is to see if maralixibat can safely and effectively help reduce this difficult itching when other options haven't worked. It's an important step for those living with chronic liver disease and severe itching, as it could potentially offer a new way to manage their symptoms and improve their quality of life.

This is a 'Phase 3' study, which means it’s one of the final steps before a new medicine might become widely available. It's taking place in many different hospitals and clinics around the world, making sure the results are as robust and reliable as possible. The aim is to gather enough information to understand if maralixibat could be a helpful treatment option for people with this condition who currently have very limited choices.

Key takeaways

Exploring a new medication (maralixibat) for severe itching.
For specific, rare types of cholestatic liver disease (not common ones).
Aims to help those who haven't found relief with other treatments.
Compares maralixibat to a placebo to check safety and effectiveness.
Open to children (from 6 months) and adults with persistent itching.

Who may be eligible?

To join this study, people need to be at least 6 months old. They must have been diagnosed with a rare type of cholestatic liver disease (but not ALGS, PFIC, PBC, or PSC) that has caused severe itching for at least 90 days.

Your liver health will be checked through blood tests, and the itching will be carefully monitored using a special score. If you're currently taking other anti-itch medications or a medication called ursodeoxycholic acid, you must have been on a steady dose for a month before the study starts and be able to continue that dose during the study. Females who could become pregnant must not be pregnant or breastfeeding and must agree to use effective birth control.

Quick self-check

Are you at least 6 months old?
Do you have a rare cholestatic liver disease (not ALGS, PFIC, PBC, or PSC)?
Have you had severe itching for more than 3 months that other treatments haven't helped?
If taking anti-itch medicine or ursodeoxycholic acid, are you on a stable dose?
If female and able to get pregnant, are you not pregnant or breastfeeding and willing to use birth control during the study?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, you'll first go through a screening period to check if you meet all the study requirements, which involves closely recording your itching daily for two weeks. Once enrolled, you would either receive the study medication, maralixibat, or a placebo (an inactive substance that looks like the study drug). This is decided randomly, like flipping a coin, and neither you nor the study team will know which you are receiving, unless there's an emergency.

You'll have regular visits to the clinic for assessments, which will include blood tests, physical examinations, and continued monitoring of your itching and overall health. The study involves taking the medication as prescribed and reporting any changes in your health or symptoms. The total duration of participations and the number of visits would be explained in detail by the study team.

Potential risks and benefits

Participating in research always involves potential benefits and risks. The potential benefit of this study is that maralixibat could help improve your severe itching. However, as with any new medication, there might be side effects or it might not work for everyone. The study team will carefully monitor your health throughout. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (24)

Children's Hospital Los Angeles (CHLA)
Los Angeles, United States
Stanford Children's Health in Palo Alto
Palo Alto, United States
Lurie Children's Hospital
Chicago, United States
NYU Langone Health
New York, United States
Mount Sinai Hospital
New York, United States
Morgan Stanley Children's Hospital - NewYork Presbyterian
New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, United States
Baylor College of Medicine
Houston, United States
University of Utah
Salt Lake City, United States
Hospital de Criança de Brasília (HCB)
Brasília, Brazil
Hospital da Criança Santo Antonio
Porto Alegre, Brazil
Hospital Sírio-Libanês
São Paulo, Brazil

+12 more sites — see the official record for the full list.

Common questions

What is 'cholestatic liver disease'?

It's a type of liver disease where the flow of bile from the liver is reduced or blocked, leading to a build-up of substances in the liver. This can cause symptoms like itching.

What is 'pruritus'?

Pruritus is the medical term for severe and persistent itching.

What is a 'placebo'?

A placebo is a 'dummy' tablet or liquid that looks like the study drug but contains no active medicine. It helps researchers understand if the new drug is truly effective.

What does 'investigational treatment' mean?

It means the treatment is new and still being researched. It has not yet been approved for general use by healthcare authorities.

Will I know if I'm getting maralixibat or the placebo?

No, you won't know, and neither will your study doctor. This is called 'blinding' and it helps ensure the study results are fair and unbiased.