Berotralstat Treatment in Children With Hereditary Angioedema

• Status: Not yet recruiting
• Trial end date: 2027-07-01
• Target enrollment: 0
• Participant gender: All

Summary

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).

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: BioCryst Pharmaceuticals
• Treatments: Berotralstat

Criteria

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