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Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)

Study on Livmarli's long-term effects for Alagille Syndrome patients.

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Phase N/A

Study Overview: This study looks at the long-term safety and effects of a medicine called Livmarli for people with Alagille Syndrome (ALGS). ALGS is a rare and complicated genetic disorder that affects many parts of the body and causes a buildup of bile, which can make skin very itchy (pruritus). Livmarli helps reduce this itchiness by stopping the body from absorbing too much bile.

What is Livmarli? It's a special medicine approved by the FDA for treating itchy skin in kids who are at least 3 months old and have ALGS. Livmarli is not absorbed much into the body, which helps make it safer for long-term use.

Key Points:

  • Participants in this study must have a confirmed diagnosis of ALGS and be prescribed Livmarli.
  • Those previously involved in Livmarli trials or who took certain investigational drugs cannot join.
  • Participants must provide consent and must not have any conditions that interfere with the study.

This study is important for understanding how Livmarli works over time, ensuring it is safe and beneficial for patients with ALGS.

Study details
    Alagille Syndrome

NCT06193928

Mirum Pharmaceuticals, Inc.

5 January 2025

Step 1 Get in touch with the nearest study center
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