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Berotralstat Treatment in Children With Hereditary Angioedema
Sponsor: BioCryst Pharmaceuticals
Summary
The purpose of this study is to evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to \< 12 years of age for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).
Official title: A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children With Hereditary Angioedema Who Are 2 to < 12 Years of Age
Key Details
Gender
All
Age Range
2 Years - 11 Years
Study Type
INTERVENTIONAL
Enrollment
29
Start Date
2022-10-25
Completion Date
2027-02
Last Updated
2026-02-25
Healthy Volunteers
No
Conditions
Interventions
Berotralstat
Administered orally once daily
Locations (15)
Investigative Site #1
Vienna, Ontario, Austria
Investigative Site #1
Ottawa, Ontario, Canada
Investigative Site #3
Grenoble, France
Investigative Site #2
Marseille, France
Investigative Site #1
Paris, France
Investigative Site #1
Berlin, Germany
Investigative Site #2
Frankfurt, Germany
Investigative Site #2
Haifa, Israel
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 #2
Málaga, Spain
Investigative Site #1
Bristol, United Kingdom