Long-term safety and outcomes of Livmarli for Alagille syndrome in the EU

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multicenter, open-label, low-intervention study enrolling participants with clinically or genetically confirmed Alagille syndrome who have cholestatic pruritus and are prescribed Livmarli. Participants remain on standard-of-care treatment and are followed with scheduled safety and efficacy assessments, including liver function tests, fat-soluble vitamin labs, serum bile acids, and pruritus measures. The study includes a primary cohort of participants prescribed Livmarli at study entry and a supplemental cohort of those prescribed the drug prior to entry. Data collection focuses on long-term tolerability, safety outcomes, and sustained clinical effect on itching.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Understand and execute an Informed consent and assent (as applicable)
* For participants with ALGS ≥2 months of age at Day 1
* For participants with PFIC, ≥3 months of age at Day 1
* A clinically and/or genetically confirmed ALGS diagnosis with pruritus secondary to chronic cholestasis, or a clinically and/or genetically confirmed PFIC diagnosis
* For the ALGS primary cohort: Initiation of Livmarli at the time of study entry
* For the ALGS supplemental cohort, : Actively using Livmarli prior to study entry
* For participants with PFIC: Prescribed Livmarli at the time of study entry or prior to study entry

Exclusion Criteria:

* History of Liver Transplant
* Any Livmarli contraindications (as per SmPC)
* Any condition or abnormality that, in the opinion of the investigator, may interfere with the participation in or completion of the study
* Received an investigational drug within 30 days before the first dose of Livmarli (Participation in previous maralixibat studies or expanded-access programs is acceptable.)
* Received another IBAT inhibitor within 7 days before the first dose of Livmarli
* Baseline data before start of treatment of Livmarli are unavailable (\<2 values before treatment) for key safety (LFTs, FSV laboratory results) and key efficacy (sBA, pruritus) parameter

Where it's running

• Cliniques Universitaires Saint Luc (UCLouvain)
  Brussels, Belgium Recruiting
• University Hospital Gent (UZ Gent)
  Ghent, Belgium Recruiting
• Hospices Civils de Lyon - Hopital Femme Mère Enfant
  Bron, Auvergne-Rhône-Alpes, France Recruiting
• CHU de Toulouse - Hôpital des Enfants
  Toulouse, Occitanie, France Recruiting
• Bicetre University Hospital
  Le Kremlin-Bicêtre, Île-de-France Region, France Recruiting
• Charite Berlin
  Berlin, Germany Recruiting

Study contacts

• Study coordinator: Clinical Trials Mirum
• Email: clinicaltrials@mirumpharma.com
• Phone: +16506674085

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Evaluating the long-term safety and outcomes of Livmarli in patients with liver diseases

  This study is testing how safe and effective the medication Livmarli is for people with Alagille syndrome or Progressive familial intrahepatic cholestasis over a five-year period.

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• Odevixibat for PFIC in China: Real-world Safety and Effectiveness

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• See if odevixibat stays safe for kids and adults with Alagille syndrome over the long term

  This study tests whether continuing odevixibat is safe for people with Alagille syndrome who already take the drug in real life.

  Recruiting at 3 sites Ipsen · Bron and 13 other locations · NCT07585097

• Finding genetic causes of adult-onset cholestatic liver disease

  This study uses whole-genome sequencing to try to find rare genetic causes in adults with unexplained cholestatic liver disease so care can be better tailored.

  Recruiting Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico · Milan, Milano · NCT07317193

• Long-term outcomes of odevixibat (Bylvay) for people with PFIC in South Korea

  This registry will see how well odevixibat controls symptoms and how safe it is over time for people with Progressive Familial Intrahepatic Cholestasis (PFIC) in South Korea.

  Recruiting at 2 sites Ipsen · Gyeongsang and 4 other locations · NCT07185919

• Observing the long-term effects of odevixibat in patients with Alagille syndrome

  This study looks at how well the medication odevixibat works and its safety for people with Alagille syndrome over a long period of time by collecting their real-life experiences.

  Recruiting at 9 sites Ipsen · Los Angeles, California and 9 other locations · NCT06850038

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.