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Berotralstat Treatment in Children With Hereditary Angioedema

NCT05453968 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 29

Last updated 2026-02-25

Study results available
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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).

Conditions

Interventions

DRUG

Berotralstat

Administered orally once daily

Sponsors & Collaborators

  • BioCryst Pharmaceuticals

    lead INDUSTRY

Principal Investigators

  • Jolanta Bernatoniene, MD · Bristol Royal Hospital for Children

Study Design

Allocation
NON_RANDOMIZED
Purpose
PREVENTION
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
11 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-10-25
Primary Completion
2024-09-11
Completion
2027-02-28
FDA Drug
Yes

Countries

  • Austria
  • Canada
  • France
  • Germany
  • Israel
  • Italy
  • Poland
  • Romania
  • Spain
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05453968 on ClinicalTrials.gov