90 Participants Needed

Maralixibat for Itching in Liver Disease

Recruiting at 36 trial locations
MM
CT
Overseen ByClinical Trials Mirum
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: Mirum Pharmaceuticals, Inc.
Must be taking: Antipruritics, Ursodeoxycholic acid
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise
Approved in 3 JurisdictionsThis treatment is already approved in other countries

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?

Maralixibat has been studied in various clinical trials and is generally considered safe for humans, though some participants experienced side effects like gastrointestinal issues. Serious adverse events were similar between those taking maralixibat and those taking a placebo.12345

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

I can read and understand questionnaires.
I have been on a stable dose of medication for itching or ursodeoxycholic acid for the last 30 days.
Informed consent and assent (as applicable)
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Exclusion Criteria

Unstable and/or serious medical disease that is likely to impair the ability to participate in all aspects of the study, confound efficacy and/or safety assessments, or result in substantially shortened life expectancy (e.g., any active malignancy including hematological malignancy, end-stage heart failure, active infection, acute and chronic diarrhea). Exceptionally, previous history of malignancy, adequately treated/in remission, that in opinion of investigator and medical monitor does not impact participant safety and participation in the study, may be allowed
Known intolerance/hypersensitivity to maralixibat or its excipients
I have had skin itching or atopic dermatitis in the last year.
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive maralixibat or placebo for up to 20 weeks, followed by maralixibat for an additional 20 weeks

40 weeks
Weekly visits for the first 20 weeks, then bi-weekly visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Maralixibat
Trial Overview 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.
Participant Groups
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.

Maralixibat is already approved in United States, European Union, Canada for the following indications:

🇺🇸
Approved in United States as Livmarli for:
  • 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
🇪🇺
Approved in European Union as Livmarli for:
  • Treatment of cholestatic pruritus in patients with Alagille syndrome
🇨🇦
Approved in Canada as Livmarli for:
  • 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

Trials
32
Recruited
102,000+

Findings from Research

Maralixibat is the first FDA-approved treatment specifically for cholestatic pruritus in children with Alagille syndrome, showing significant efficacy in clinical trials with follow-up of up to 4 years.
A case report highlights a patient who experienced complete resolution of pruritus for over 7 years after starting maralixibat, allowing her to stop all other antipruritic medications, demonstrating the long-term effectiveness of this treatment.
Resolution of Pruritus in a Child With Alagille Syndrome Treated With Maralixibat for Seven Years: Durable Response and Discontinuation of Other Medications.Garcia, A., Hsu, E., Lin, HC.[2023]
In a multicenter trial involving 37 children with Alagille syndrome, maralixibat showed potential to reduce severe itching (pruritus) compared to placebo, particularly at doses of 70 and 140 µg/kg/day, with significant caregiver and clinician-reported improvements.
The treatment was found to be safe, with no significant changes in liver function or bile acid levels, and adverse events were similar between maralixibat and placebo groups.
Placebo-Controlled Randomized Trial of an Intestinal Bile Salt Transport Inhibitor for Pruritus in Alagille Syndrome.Shneider, BL., Spino, C., Kamath, BM., et al.[2023]
In a phase 2 study involving 66 adults with primary biliary cholangitis (PBC) and pruritus, maralixibat did not show a significant difference in reducing itching compared to placebo, indicating that its efficacy may be limited in this context.
Both treatment groups experienced high rates of adverse events, particularly gastrointestinal disorders, suggesting that while maralixibat is being explored as a treatment option, its safety profile needs careful consideration.
A Randomized, Controlled, Phase 2 Study of Maralixibat in the Treatment of Itching Associated With Primary Biliary Cholangitis.Mayo, MJ., Pockros, PJ., Jones, D., et al.[2021]

References

Resolution of Pruritus in a Child With Alagille Syndrome Treated With Maralixibat for Seven Years: Durable Response and Discontinuation of Other Medications. [2023]
Placebo-Controlled Randomized Trial of an Intestinal Bile Salt Transport Inhibitor for Pruritus in Alagille Syndrome. [2023]
A Randomized, Controlled, Phase 2 Study of Maralixibat in the Treatment of Itching Associated With Primary Biliary Cholangitis. [2021]
Maralixibat: First Approval. [2022]
Impact of long-term administration of maralixibat on children with cholestasis secondary to Alagille syndrome. [2023]
Use of levamisole in parasitic infections. [2018]
[Use of levamisole in W. bancrofti filariasis]. [2016]
Semiannual Treatment of Albendazole Alone is Efficacious for Treatment of Lymphatic Filariasis: A Randomized Open-label Trial in Cote d'Ivoire. [2022]
Vitamin A status of Indonesian children infected with Ascaris lumbricoides after dosing with vitamin A supplements and albendazole. [2018]
Levamisole and mebendazole in the treatment of bancroftian infection. [2013]
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