What is the mechanism of action of this treatment?

The most common treatments for Progressive Familial Intrahepatic Cholestasis (PFIC) include bile acid sequestrants, ursodeoxycholic acid (UDCA), and ASBT inhibitors like Maralixibat. ASBT inhibitors work by blocking the ileal bile acid transporter, which reduces the reabsorption of bile acids in the intestines. This leads to lower bile acid levels in the liver, thereby alleviating cholestasis and its associated symptoms such as pruritus and liver damage. This mechanism is particularly important for PFIC patients as it helps manage the disease's progression and improves their quality of life.

What side effects are associated with this type of intervention?

Maralixibat, an ASBT inhibitor, is primarily being evaluated for its long-term safety and tolerability in treating PFIC. Common side effects associated with ASBT inhibitors like maralixibat may include gastrointestinal issues such as diarrhea, abdominal pain, and nausea. Other potential side effects could involve liver enzyme abnormalities and pruritus (itching). It is important for patients to discuss these potential side effects with their healthcare provider to manage and monitor any adverse reactions effectively.

What prior FDA approvals exist?

As of now, there are limited FDA-approved treatments specifically for Progressive Familial Intrahepatic Cholestasis (PFIC). Maralixibat, an apical sodium-dependent bile acid transporter (ASBT) inhibitor, is currently being studied for its long-term safety and tolerability in PFIC patients. Other treatments for cholestatic liver diseases, such as ursodeoxycholic acid (UDCA), have been used to manage symptoms and improve liver function, but they are not specifically approved for PFIC. The development of targeted therapies like Maralixibat represents a significant advancement in addressing the underlying mechanisms of PFIC.

What other types of research exist?

For PFIC patients considering treatment in 2024, elafibranor, a dual PPARα/δ agonist, is a promising alternative. It has shown efficacy and safety in patients with primary biliary cholangitis who had an incomplete response to ursodeoxycholic acid. Additionally, bezafibrate, used as a second-line agent for primary biliary cholangitis, may also be considered due to its positive impact on long-term outcomes.

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IBAT Inhibitor

Maralixibat for Cholestasis

Phase 3

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

Be younger than 65 years old

Must not have

Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial aims to see if maralixibat, a medication, is safe and can be tolerated by patients over time. It involves patients who have already been using the medication. The study is being conducted at various locations around the world.

Who is the study for?

This trial is for patients with Progressive Familial Intrahepatic Cholestasis who have completed the MRX-502 study. They must be able to give informed consent and not have any health issues or lab results that could make the trial unsafe for them. Pregnant or breastfeeding women, those planning pregnancy, or anyone with a history of non-compliance in the previous study cannot participate.

What is being tested?

The trial is testing Maralixibat's long-term safety and effectiveness in treating cholestasis conditions like PFIC. It's an open label extension, meaning everyone knows they're getting Maralixibat and there’s no placebo group.

What are the potential side effects?

While specific side effects are not listed here, participants will be monitored for any adverse reactions since this study aims to assess the long-term safety and tolerability of Maralixibat.

Eligibility Criteria

Exclusion Criteria

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

Select...

I stopped taking maralixibat due to a serious side effect in the MRX-502 study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

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.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: MaralixibatExperimental Treatment1 Intervention

All subjects will 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

The most common treatments for Progressive Familial Intrahepatic Cholestasis (PFIC) include bile acid sequestrants, ursodeoxycholic acid (UDCA), and ASBT inhibitors like Maralixibat. ASBT inhibitors work by blocking the ileal bile acid transporter, which reduces the reabsorption of bile acids in the intestines. This leads to lower bile acid levels in the liver, thereby alleviating cholestasis and its associated symptoms such as pruritus and liver damage. This mechanism is particularly important for PFIC patients as it helps manage the disease's progression and improves their quality of life.