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Bile Acid Sequestrant

Maralixibat for Itching in Liver Disease

Phase 3

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

Diagnosis of cholestatic liver disease with cholestatic pruritus based on the presence of chronic liver biochemical abnormalities (>90 days) and/or pathological evidence of progressive liver disease

Must not have

Diagnosis of ALGS, ICP, PBC, PFIC, or PSC with native liver

Suspected or proven cholangiocarcinoma or hepatocellular carcinoma

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to average of week 13 to week 20

Awards & highlights

Pivotal Trial

Summary

This trial aims to find out if a new treatment called maralixibat is safe and works well for people with liver disease and itching that hasn't improved with other treatments and have no other options

Who is the study for?

This trial is for children and adults with cholestatic liver disease who suffer from severe itching that hasn't improved with other treatments. Participants must have no remaining treatment options available to them.

What is being tested?

The study is testing the safety and effectiveness of a drug called Maralixibat compared to a placebo in reducing itchiness associated with cholestatic liver disease.

What are the potential side effects?

Possible side effects of Maralixibat may include gastrointestinal symptoms, changes in liver enzyme levels, fatigue, headache, and potential nutrient deficiencies.

Eligibility Criteria

Inclusion Criteria

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

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I have been diagnosed with a liver condition that causes itching, confirmed by tests or liver disease signs for over 3 months.

Exclusion Criteria

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

Select...

I have a liver disease like ALGS, ICP, PBC, PFIC, or PSC and still have my original liver.

Select...

I have or might have bile duct or liver cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to average of week 13 to week 20

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to average of week 13 to week 20

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to average of week 13 to week 20 for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Mean change in the ItchRO(Obs) severity score

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

Pivotal Trial

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

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: MaralixibatExperimental Treatment1 Intervention

Participants will receive maralixibat oral solution 300 μg/kg orally once daily for 1 week and then twice daily for 39 weeks.

Group II: PlaceboPlacebo Group1 Intervention

Participants will receive placebo matched to maralixibat oral solution orally once daily for 1 week and then twice daily for 19 weeks. After 20 weeks, participants will receive maralixibat oral solution 300 μg/kg orally once daily for 1 week and then twice daily for 19 weeks.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Maralixibat

2015

Completed Phase 3

~260

0 / 3Eligibility criteria met