Maralixibat for Itching in Liver Disease
Trial Summary
Will I have to stop taking my current medications?
The trial does not require you to stop taking your current medications if you are on antipruritics or ursodeoxycholic acid, as long as you have been on a stable dose for at least 30 days before the screening and continue this dosing during the study.
What data supports the effectiveness of the drug Maralixibat for itching in liver disease?
Research shows that Maralixibat can significantly reduce itching in children with Alagille syndrome, a liver disease, with some patients experiencing complete relief and stopping other itch medications. In clinical trials, Maralixibat was found to be safe and led to a noticeable decrease in itching compared to a placebo.12345
Is Maralixibat safe for humans?
What makes the drug Maralixibat unique for treating itching in liver disease?
Maralixibat is unique because it targets the bile acid transport system, which is different from other treatments that may not address this specific mechanism. This approach can help reduce itching by decreasing the accumulation of bile acids in the body, which is a common issue in liver disease.678910
What is the purpose of this trial?
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.
Eligibility Criteria
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.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive maralixibat or placebo for up to 20 weeks, followed by maralixibat for an additional 20 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Maralixibat
Maralixibat is already approved in United States, European Union, Canada for the following indications:
- Treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older
- Treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 3 months of age and older
- Treatment of cholestatic pruritus in patients with Alagille syndrome
- Treatment of cholestatic pruritus in adults with Alagille syndrome
Find a Clinic Near You
Who Is Running the Clinical Trial?
Mirum Pharmaceuticals, Inc.
Lead Sponsor