Clinical Trial Finder

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

This is a single-arm, open-label study designed to evaluate the PK and safety of berotralstat weight-based treatment for the prevention of hereditary angioedema attacks in pediatric participants 2 to < 12 years of age. This study will consist of two treatment periods: a 12-week standard-of-care (SOC) treatment period followed by an open-label berotralstat treatment period lasting up to 144 weeks. Participants will be enrolled into 4 dose cohorts; participant weight will be 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 will then be used to confirm the weight bands for sequentially enrolling Cohorts 3 and 4.The effectiveness of berotralstat in this population will be summarized using descriptive statistical methods.

Arms

Experimental: Berotralstat

Berotralstat administered once daily in 4 dose cohorts determined by participant weight. Cohorts 1 and 2 will enroll in parallel. After 4 participants from Cohort 1 and 2, with ≥ 2 subjects from Cohort 2, have reached Week 2, Cohort 3 will open for enrollment. Cohort 4 will open for enrollment, after ≥ 4 subjects in Cohort 3 have reached Week 2. Prior to dosing Cohort 3 and 4, available PK and safety data will be reviewed to confirm it is safe to proceed and the appropriate weight bands for each. BioCryst will notify sites when Cohorts 3 and 4 are open for enrollment.

Interventions

Drug: - Berotralstat

Administered orally once daily at a weight-based dose in up to 4 cohorts