What side effects are associated with this type of intervention?

Common treatments for Primary Biliary Cholangitis (PBC) include ursodeoxycholic acid (UDCA), obeticholic acid, and fibrates. UDCA is generally well-tolerated but may cause weight gain and, in rare cases, hair thinning. Obeticholic acid can lead to pruritus (itching), fatigue, and abdominal pain. Fibrates, such as fenofibrate, are associated with gastrointestinal disturbances and potential liver enzyme elevations. The investigational drug volixibat, an IBAT inhibitor, is being studied for its efficacy in treating pruritus associated with PBC, but specific side effects are not detailed in the provided context.

What prior FDA approvals exist?

The FDA has approved several treatments for Primary Biliary Cirrhosis (PBC). Ursodeoxycholic acid (UDCA) is the first-line treatment, improving liver function and delaying disease progression. Obeticholic acid, a farnesoid X receptor agonist, is another approved drug for patients with an inadequate response to UDCA. While Volixibat, an IBAT inhibitor, is still under investigation, it aims to alleviate pruritus associated with PBC, a symptom not directly targeted by UDCA or obeticholic acid.

What other types of research exist?

Promising, novel alternatives to Volixibat for Primary Biliary Cirrhosis (PBC) patients in 2024 may include other investigational drugs targeting pruritus or disease progression in PBC. While specific alternatives are not mentioned in the provided context, patients should discuss with their healthcare providers about ongoing clinical trials and emerging therapies in the field of liver diseases, particularly those focusing on symptom management and disease modification.

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Bile Acid Sequestrant

Volixibat for Primary Biliary Cholangitis (VANTAGE Trial)

Phase 2

Recruiting

Research Sponsored by Mirum Pharmaceuticals, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Qualified pruritus associated with PBC as assessed by Adult ItchRO

Confirmed diagnosis of PBC in line with the AASLD guidelines

Must not have

Pruritus associated with an etiology other than PBC

History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to week 28

Awards & highlights

Summary

This trial is testing a medicine called volixibat that may help reduce itching in people with a liver disease that often causes severe itching. Volixibat works by blocking certain chemicals in the body that cause itching. The study also aims to see if the medicine can slow down the progression of the liver disease.

Who is the study for?

This trial is for adults over 18 with itching due to Primary Biliary Cholangitis (PBC), as per AASLD guidelines. Participants must sign a consent form and follow study procedures. Those on UDCA or anti-itch meds can join if they meet extra rules. People with certain bowel surgeries, other liver diseases, non-PBC related itching, or severe cirrhosis cannot participate.

What is being tested?

The study tests Volixibat's effectiveness in treating PBC-related itching and its impact on the disease's progression. Participants will either receive Volixibat or a placebo (a substance with no active drug) to compare outcomes between the two groups.

What are the potential side effects?

While specific side effects of Volixibat are not listed here, common side effects may include digestive issues since it targets bile acid processes linked to PBC and pruritus.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have itching due to primary biliary cholangitis.

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I have been diagnosed with primary biliary cholangitis according to AASLD guidelines.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have itching not caused by primary biliary cholangitis.

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I've had surgery on my small intestine that affects bile acid circulation.

Select...

I am currently experiencing symptoms of gallstones or gallbladder inflammation.

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I have or might have severe liver damage with symptoms.

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I have or might have liver disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to week 28

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to week 28

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 28 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Pruritus

Secondary study objectives

Fatigue

Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire

Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40)

Side effects data

From 2015 Phase 1 trial • 84 Patients • NCT02287779

100%

Diarrhoea

22%

Pyrexia

11%

Proctalgia

11%

Abdominal discomfort

11%

Anorectal discomfort

11%

Tachycardia

11%

Vomiting

11%

Pain

11%

Headache

11%

Dry throat

11%

Nausea

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Volixibat 5 mg BID

Volixibat 10 mg QD

Volixibat 20 mg QD

Volixibat 2-5-10-20 mg QD

Volixibat 30 mg QD

Volixibat 40 mg QD

Volixibat 80-40-20 mg QD

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Volixibat 20mgExperimental Treatment1 Intervention

Participants randomized to this arm will receive volixibat 20mg twice daily.

Group II: PlaceboPlacebo Group1 Intervention

Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Volixibat

2015

Completed Phase 1

~90

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Primary Biliary Cirrhosis (PBC) include Ursodeoxycholic Acid (UDCA) and Obeticholic Acid. UDCA works by reducing the concentration of toxic bile acids, thereby protecting liver cells and improving bile flow. Obeticholic Acid, a potent farnesoid X receptor agonist, enhances bile acid homeostasis and reduces liver inflammation. Investigational therapies like Volixibat, an ileal bile acid transporter (IBAT) inhibitor, aim to reduce bile acid reabsorption in the intestines, thereby lowering bile acid levels in the liver and alleviating pruritus. These treatments are crucial for PBC patients as they help manage symptoms, slow disease progression, and improve quality of life.