New safety study monitors liver risks in kids taking livmarli

NCT ID NCT07293897

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study is monitoring 50 children in Japan with Alagille syndrome (ALGS) or progressive familial intrahepatic cholestasis (PFIC) who are taking the drug Livmarli (maralixibat). Researchers are using a national registry to track how many children develop liver problems and how quickly they appear. The goal is to better understand the safety of Livmarli in these rare liver conditions.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

Maralixibat (Livmarli oral solution)

What this could lead to

If successful, this study will provide real-world safety data on liver risks for children with ALGS or PFIC taking Livmarli, helping doctors make informed treatment decisions.

What could go wrong

This is an observational registry study, not a controlled trial, so it cannot prove cause and effect. The small sample size (50 patients) may limit how widely the results apply.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

Alagille syndrome Cholestasis, progressive familial intrahepatic 1 progressive familial intrahepatic cholestasis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Takeda selected site

    RECRUITING

    Tokyo, Tokyo, Japan