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

BCX7353-304

The purpose of this study is to evaluate the pharmacokinetics (PK) and safety of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to \< 12 years old for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).

2022-10-25

2025-07-01

2027-07-01

Active (not recruiting)

Early phase I

29

Inclusion Criteria: * Male and non-pregnant, non-lactating females 2 to \< 12 years of age * Body weight ≥ 12 kg * Clinical diagnosis of HAE * In the opinion of the investigator, the participant would benefit from long term oral HAE prophylaxis Exclusion Criteria: * Concurrent diagnosis of any other type of recurrent angioedema * Known family history of sudden cardiac death * Creatinine clearance using the modified Schwartz formula of ≤ 30 mL/min/1.73 m2 * Aspartate aminotransferase or alanine aminotransferase value ≥ 3 × the upper limit of the age-appropriate normal reference range value * Clinically significant abnormal ECG including but not limited to, a corrected QT interval calculated using Fridericia's correction \> 450 msec, or ventricular and/or atrial premature contractions that are more frequent than occasional, and/or as couplets or higher in grouping * Current participation in any other investigational drug study or received another investigational drug within 30 days of enrollment

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'This is a sequential, three-part, open-label study. Minimum participation in this study is expected to be 24 weeks in the SOC treatment period through Part 1 of the study and up to an additional 132 weeks in Parts 2 and 3.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE', 'maskingDescription': 'None (Open Label)'}}, 'enrollmentInfo': {'count': 29, 'type': 'ACTUAL'}}

2024-03-13