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

NCT ID NCT05453968

Ongoing Disease control Sponsor: BioCryst Pharmaceuticals Source: ClinicalTrials.gov ↗

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

Summary

This study tests a daily medication called berotralstat in 29 children aged 2 to under 12 who have hereditary angioedema (HAE), a condition that causes sudden, painful swelling attacks. The goal is to find the right dose based on weight and to check if it is safe and effective at preventing these attacks. Participants take the pill daily and are monitored for side effects and attack frequency.

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Conditions

The condition(s) this trial relates to.

hereditary angioedema

As listed by the trial registrant

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

HEREDITARY ANGIOEDEMA PEDIATRIC

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

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