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

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 5-year, prospective, observational cohort study aimed at assessing the long-term safety and clinical outcomes of patients diagnosed with Alagille syndrome (ALGS) or Progressive familial intrahepatic cholestasis (PFIC) who are treated with Livmarli. Livmarli is a novel medication that works by inhibiting the ileal bile acid transporter, thereby reducing bile acid levels in the body. The study will include participants who have a confirmed diagnosis of ALGS or PFIC and are prescribed Livmarli, allowing researchers to gather data on its effectiveness and safety over time.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* A clinically and/or genetically confirmed ALGS diagnosis or PFIC diagnosis
* Participant prescribed Livmarli

Exclusion Criteria:

* Refusal to provide informed consent/assent (if required by the local IRB)
* Previously or currently on Livmarli through participation in a clinical study or expanded access program
* Participants who have previously received an SBD or LT
* Any condition or abnormalities that, in the opinion of the investigator, may interfere with the participant participating in or completing the study
* Participants who have received an investigational drug within 30 days of the first dose of Livmarli

Where it's running

• Children's Hospital Los Angeles CHLA
  Los Angeles, California, United States Recruiting
• Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado
  Aurora, Colorado, United States Recruiting
• Children's Healthcare of Atlanta - Emory University School of Medicine
  Atlanta, Georgia, United States Recruiting
• Children's Mercy Kansas City, Department of Gastroenterology, Section of Hepatology
  Kansas City, Missouri, United States Recruiting
• Oregon Health and Science University, Division of Pediatric Gastroenterology, Department of Pediatrics
  Portland, Oregon, United States Recruiting
• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting

Study contacts

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

Similar studies

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

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

  This study will follow people with Alagille syndrome who take Livmarli to see if it is safe, well tolerated over the long term, and continues to reduce cholestatic itching.

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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.