NCT05453968

Berotralstat Treatment in Children With Hereditary Angioedema

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

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: BioCryst Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years – 11 Years
• Healthy volunteers: Not accepted

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).

Detailed description

This is a single-arm, 3-part, open-label study designed to evaluate the PK, safety, and effectiveness of berotralstat weight-based treatment for the prevention of HAE attacks in pediatric participants 2 to \< 12 years of age. Participation in this study is expected to be a minimum of 12 weeks in the standard of care (SOC) period, and in the berotralstat period, it is expected to be 12 weeks in Part 1, 36 weeks in Part 2, and 96 weeks in Part 3 of the study. Participants were enrolled into 4 cohorts; participant weight at baseline was used to determine assignment to each cohort with the higher weight cohorts (Cohorts 1 and 2) enrolling first and in parallel. Safety assessments and PK modelling from all available PK data were then used to confirm the dose and weight bands for sequentially enrolling Cohorts 3 and 4. The safety and effectiveness of berotralstat in this population was summarized using descriptive statistical methods.

Conditions

• Hereditary Angioedema
• Pediatric

Interventions

Type	Name	Description
DRUG	Berotralstat	Administered orally once daily

Timeline

• Start date: 2022-10-25
• Primary completion: 2024-09-11
• Completion: 2027-02-01
• First posted: 2022-07-12
• Last updated: 2026-02-25
• Results posted: 2026-02-25

Locations

15 sites across 10 countries: Austria, Canada, France, Germany, Israel, Italy, Poland, Romania, Spain, United Kingdom

Regulatory

• FDA-regulated drug study