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Bile Acid Sequestrant

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
1. Informed consent and assent (as applicable)
2. Age ≥6 months at time of baseline visit
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline to average of week 13 to week 20
Awards & highlights

Summary

The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric and adult participants who have cholestatic liver disease with pruritus that has been refractory to other therapies, and who have no other treatment 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

Timeline

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
Secondary study objectives
Mean change in total sBA (serum bile acid) level

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%
Alanine aminotransferase increased
13%
Influenza
11%
Nasopharyngitis
11%
Pruritus
9%
Vitamin D decreased
9%
Vitamin E decreased
9%
Vitamin D deficiency
9%
Constipation
6%
Vomiting
6%
Vitamin E deficiency
6%
Gastroenteritis
6%
Upper respiratory tract infection
6%
Coronavirus infection
4%
Urinary tract infection
4%
Abdominal pain upper
2%
International normalised ratio increased
2%
Cholestasis
2%
Idiopathic pneumonia syndrome
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
Maralixibat

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

Find a Location

Who is running the clinical trial?

Mirum Pharmaceuticals, Inc.Lead Sponsor
30 Previous Clinical Trials
1,646 Total Patients Enrolled
9 Trials studying Cholestasis
392 Patients Enrolled for Cholestasis
~60 spots leftby Sep 2026
Unbiased ResultsWe believe in providing patients with all the options.
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