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New hope for kids with rare swelling disorder: daily pill may prevent attacks

NCT ID NCT05453968

First seen Oct 31, 2025 · Last updated May 24, 2026 · Updated 25 times

Summary

This study tests a daily medication called berotralstat in children aged 2 to under 12 who have hereditary angioedema (HAE), a rare condition that causes sudden, painful swelling attacks. The goal is to find the right dose based on the child's weight and to check if the drug is safe and helps prevent attacks. About 29 children will take the pill for up to a year, and researchers will measure how the drug moves through their bodies and track any side effects.

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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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Contacts and locations

Locations

  • Investigative Site #1

    Vienna, Ontario, Austria

  • Investigative Site #1

    Ottawa, Ontario, Canada

  • Investigative Site #1

    Paris, France

  • Investigative Site #1

    Berlin, Germany

  • Investigative Site #1

    Tel Aviv, Israel

  • Investigative Site #1

    Padova, Italy

  • Investigative Site #1

    Krakow, Poland

  • Investigative Site #1

    Sângeorgiu de Mureş, Romania

  • Investigative Site #1

    Madrid, Spain

  • Investigative Site #1

    Bristol, United Kingdom

  • Investigative Site #2

    Marseille, France

  • Investigative Site #2

    Frankfurt, Germany

  • Investigative Site #2

    Haifa, Israel

  • Investigative Site #2

    Málaga, Spain

  • Investigative Site #3

    Grenoble, France

Conditions

Explore the condition pages connected to this study.