What side effects are associated with this type of intervention?

Common treatments for cholestatic liver disease include ursodeoxycholic acid (UDCA), bezafibrate, and obeticholic acid. UDCA is generally well-tolerated but can cause diarrhea and weight gain. Bezafibrate, used in combination with UDCA, is associated with gastrointestinal disturbances and muscle pain. Obeticholic acid can lead to pruritus, fatigue, and abdominal pain. Similar to Maralixibat, an IBAT inhibitor, these treatments aim to improve liver function and reduce symptoms, but they come with their own set of potential side effects that should be monitored by healthcare providers.

What prior FDA approvals exist?

FDA-approved treatments for cholestatic liver disease, particularly primary biliary cholangitis (PBC), include ursodeoxycholic acid (UDCA) and obeticholic acid. UDCA is the first-line treatment and has been shown to improve liver function and delay disease progression. Obeticholic acid is used for patients with an inadequate response to UDCA. These treatments aim to improve bile flow and reduce liver damage. Maralixibat, an IBAT inhibitor, is being studied for its potential benefits in treating cholestatic liver diseases by reducing bile acid reabsorption in the intestines.

What other types of research exist?

Promising novel alternatives to Maralixibat for cholestatic liver disease patients include obeticholic acid, which has shown efficacy in primary biliary cholangitis, and bezafibrate, which has demonstrated positive outcomes in patients with suboptimal response to ursodeoxycholic acid. Additionally, emerging therapies such as gene therapy and other bile acid modulators are being investigated and may offer new treatment options in the near future.

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IBAT Inhibitor

Long-Term Safety of Maralixibat for Cholestatic Liver Disease (MERGE Trial)

Phase 2

Waitlist Available

Research Sponsored by Mirum Pharmaceuticals, Inc.

Eligibility Criteria Checklist

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

Must have

Must not have

Experienced an AE or SAE related to maralixibat during the lead-in protocol that led to permanent discontinuation of the subject from maralixibat.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through study completion, up to approximately 3 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing the long-term safety of maralixibat, a medication for people with certain liver diseases that cause bile flow problems. The study includes patients with conditions like Alagille Syndrome, PFIC, and Biliary Atresia who have already tried maralixibat before. Maralixibat works by reducing bile acids in the body to help improve liver function and symptoms. It was developed by Mirum Pharmaceuticals for treating rare liver diseases.

Who is the study for?

This trial is for individuals who have previously participated in a maralixibat study for cholestatic liver disease, including conditions like Alagille Syndrome and Biliary Atresia. Participants must be at least one year old, able to give consent, and agree to use contraception if of childbearing potential. Those with adverse reactions leading to previous discontinuation or conditions that may affect safety or compliance are excluded.

What is being tested?

The study tests the long-term safety of maralixibat (MRX-800) in treating various forms of cholestatic liver disease. It's designed for those who've completed earlier phases of maralixibat trials and will involve regular check-ins via email or phone.

What are the potential side effects?

While specific side effects aren't listed here, participants from prior studies would only be excluded if they had serious adverse events related to maralixibat that led them to stop taking it permanently.

Eligibility Criteria

Exclusion Criteria

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

Select...

I stopped taking maralixibat permanently due to a severe side effect.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through study completion, up to approximately 3 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through study completion, up to approximately 3 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion, up to approximately 3 years for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Frequency of reported adverse events AEs

Secondary study objectives

Evaluate the long-term effects of maralixibat on growth

Side effects data

From 2022 Phase 3 trial • 93 Patients • NCT03905330

57%

Diarrhoea

36%

Pyrexia

21%

Abdominal pain

17%

Rhinorrhoea

15%

Cough

15%

Blood bilirubin increased

13%

Influenza

13%

Alanine aminotransferase increased

11%

Nasopharyngitis

11%

Pruritus

Vitamin D deficiency

Vitamin D decreased

Constipation

Vitamin E decreased

Gastroenteritis

Coronavirus infection

Upper respiratory tract infection

Vomiting

Vitamin E deficiency

Abdominal pain upper

Urinary tract infection

Idiopathic pneumonia syndrome

Cholestasis

International normalised ratio increased

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Maralixibat

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: MaralixibatExperimental Treatment1 Intervention

Participants will all receive Maralixibat oral solution

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Maralixibat

2015

Completed Phase 3

~260

0 / 1Eligibility 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

Cholestatic liver diseases are often treated with agents that modify bile acid metabolism to reduce liver damage and alleviate symptoms. Ursodeoxycholic Acid (UDCA) is a common treatment that works by making the bile acid pool more hydrophilic, which helps reduce bile acid toxicity and improve bile flow. Maralixibat, an ileal bile acid transporter (IBAT) inhibitor, reduces the reabsorption of bile acids in the intestine, thereby decreasing the bile acid load in the liver. This mechanism is particularly important for patients with cholestatic liver disease as it helps to lower the levels of toxic bile acids, reduce liver inflammation, and improve overall liver function. These treatments are crucial as they address the underlying issues of bile acid accumulation and liver damage, providing symptomatic relief and potentially slowing disease progression.

Ursodeoxycholic acid for the treatment of primary biliary cirrhosis. Interim analysis of a double-blind multicentre randomized trial. The UDCA-PBC Study Group.