Your session is about to expire
← Back to Search
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 belowSelect...
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
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 • NCT0390533057%
Diarrhoea
36%
Pyrexia
21%
Abdominal pain
17%
Rhinorrhoea
15%
Cough
15%
Blood bilirubin increased
13%
Influenza
13%
Alanine aminotransferase increased
11%
Nasopharyngitis
11%
Pruritus
9%
Vitamin D deficiency
9%
Vitamin D decreased
9%
Constipation
9%
Vitamin E decreased
6%
Coronavirus infection
6%
Gastroenteritis
6%
Upper respiratory tract infection
6%
Vomiting
6%
Vitamin E deficiency
4%
Abdominal pain upper
4%
Urinary tract infection
2%
Idiopathic pneumonia syndrome
2%
Cholestasis
2%
International normalised ratio increased
100%
80%
60%
40%
20%
0%
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
Find a Location
Who is running the clinical trial?
Mirum Pharmaceuticals, Inc.Lead Sponsor
31 Previous Clinical Trials
101,647 Total Patients Enrolled
9 Trials studying Cholestasis
392 Patients Enrolled for Cholestasis