Can a common cholesterol drug shrink skin lumps in kids with rare liver disease?

NCT ID NCT05488067

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

Summary

This study tested the safety and effectiveness of atorvastatin (Lipitor) in 15 children with Alagille syndrome who had skin cholesterol deposits called xanthomas. The children took the drug for 6 months, and doctors graded the size and number of the lumps. The goal was to see if the drug could reduce these deposits without causing serious side effects.

What this could mean

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

Active substance

atorvastatin (Lipitor)

What this could lead to

If it works, this could offer a way to shrink or prevent disfiguring skin cholesterol lumps in children with Alagille syndrome.

What could go wrong

This is a very small, single-center study with only 15 children. The results may not apply to all patients, and statins can cause muscle pain or liver issues.

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 xanthoma xanthomatosis

As listed by the trial registrant

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

Contacts and locations

Locations

  • Children's Hospital of Fudan University

    Shanghai, Shanghai Municipality, 201102, China