Active not recruitingPHASE2INTERVENTIONAL

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)

This study is a clinical trial looking at a new medicine called volixibat. It's for people who have a liver condition called Primary Sclerosing Cholangitis (PSC) and are troubled by itching. Researchers want to find out if volixibat is safe and if it can help reduce this itching. They also want to understand if the medicine might have an effect on the overall progression of PSC. Patients in the study will either receive volixibat or a dummy medicine (placebo). This research is currently in Phase 2, which means it’s still an early stage where doctors are mainly checking for safety and how well the medicine works.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Mirum Pharmaceuticals, Inc.

Enrolment target

182

Start

18 Dec 2020

Estimated completion

01 Apr 2027

Primary Sclerosing Cholangitis

What is this study about?

This clinical trial is all about a new investigational medicine called volixibat. Doctors are studying it because they want to find better ways to help people who have a liver condition called Primary Sclerosing Cholangitis, or PSC, especially if they experience persistent and bothersome itching. PSC is a chronic disease that affects the bile ducts in the liver. These ducts can become inflamed and scarred, which can lead to various symptoms, including severe itching. The research team wants to see if volixibat can ease this itching for people with PSC. They will also be looking to see if the medicine has any impact on how the disease develops over time, which is very important for people living with PSC. In this study, some participants will receive the investigational medicine, volixibat, while others will receive a placebo. A placebo is a dummy treatment that looks just like the real medicine but contains no active ingredients. This helps researchers to fairly compare the effects of volixibat. This is a Phase 2 study, which means it's an important step where the medicine's safety and effectiveness are being carefully evaluated.

Key takeaways

This study is testing a new medicine, volixibat, for itching related to PSC.
It aims to see if volixibat is safe and effective for this type of itching.
The study also looks at how the medicine might affect PSC progression.
Some participants will receive the active medicine; others will receive a placebo.
It's a Phase 2 study, focusing on early safety and effectiveness.
Your participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To join this study, you would need to be at least 12 years old if you live in certain areas, or 18 years old in others. You must have a confirmed diagnosis of PSC and be experiencing bothersome itching because of it. If you're already taking medicines for PSC or to help with itching, or if you also have Inflammatory Bowel Disease (IBD), you might still be able to join under certain conditions.

However, some things would prevent you from taking part. For example, if your itching is caused by something other than PSC, or if you have severe liver problems or a history of certain surgeries that affect your digestive system, you wouldn't be able to join. Also, if you've recently had certain procedures on your bile ducts or if some of your blood test results are outside a specific range, you might not be eligible.

It's important that you understand and agree to all the study requirements, including attending all visits and follow-up appointments.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

I have been diagnosed with Primary Sclerosing Cholangitis (PSC).
I experience bothersome itching because of my PSC.
I am at least 12 years old (or 18, depending on location).
I am generally in stable health and don't have very severe liver problems.
I am willing and able to attend all study appointments and follow instructions.
I haven't had recent surgery that affected my digestive system in a major way.

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will first have screening appointments to make sure you meet all the requirements. Throughout the study, you'll have regular visits to the clinic where doctors will monitor your health, check for any changes in your itching, and take blood samples. You will be given either the study medicine, volixibat, or a placebo, and you'll need to take it as instructed. There will also be follow-up appointments after you stop taking the study medicine to ensure your well-being. The total duration of your involvement in the study, including follow-up, will be explained in detail by the study team.

Potential risks and benefits

Participating in this study might offer potential benefits, such as gaining access to a new investigational medicine that could help reduce your itching from PSC. You'd also be contributing valuable information that could help others with PSC in the future. However, there are potential risks, as with any new medicine. Volixibat might cause side effects, and these will be carefully explained to you. You'll receive close medical monitoring throughout the study. Remember, joining is entirely voluntary, and you have the right to withdraw from the study at any time without affecting your medical care.

Locations (103)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Southern California Research Center
Verified postcode
Coronado, United States
Cedars Sinai Medical Center
Verified postcode
Los Angeles, United States
University of California, Davis
Verified postcode
Sacramento, United States
California Pacific Medical Center Research Institute
Verified postcode
San Francisco, United States
University of Colorado - Anschutz Medical Campus
Verified postcode
Aurora, United States
MedStar Georgetown University Hospital
Verified postcode
Washington D.C., United States
Florida Research Institute
Verified postcode
Lakewood Rch, United States
University of Miami - Schiff Center for Liver Diseases
Verified postcode
Miami, United States
Tampa General Hospital
Verified postcode
Tampa, United States
Beth Israel Deaconess Medical Center
Verified postcode
Boston, United States
Henry Ford Hospital
Verified postcode
Detroit, United States
Mayo Clinic
Verified postcode
Rochester, United States

Common questions

What is PSC?

PSC, or Primary Sclerosing Cholangitis, is a long-term liver disease where the bile ducts gradually become inflamed and scarred, which can lead to symptoms like itching.

What is volixibat?

Volixibat is an investigational medicine being tested in this study to see if it can help reduce itching in people with PSC and affect how the disease progresses.

What is a placebo?

A placebo is a 'dummy' medicine that looks like the real thing but doesn't contain any active drug. It's used in studies to compare the effects of the actual medicine fairly.

Will I know if I'm getting the real medicine or the placebo?

No, during the main part of the study, neither you nor your doctors will know whether you are receiving volixibat or the placebo. This is to ensure the study results are unbiased.

What 'phase' is this study?

This is a Phase 2 study. This means it's an early stage where doctors are mainly checking for the medicine's safety and how well it works.