Berotralstat for Pediatric Hereditary Angioedema
(APeX-P Trial)

Recruiting in Palo Alto (17 mi)

+16 other locations

Overseen byMatthew Buckland, MBBS, PhD, FRCP

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: BioCryst Pharmaceuticals

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?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).

Eligibility Criteria

This trial is for children aged 2 to less than 12 with hereditary angioedema (HAE), weighing at least 12 kg, who could benefit from daily oral medication to prevent HAE attacks. It's not for kids with other types of angioedema, a family history of sudden heart death, certain liver issues, abnormal ECGs, or poor kidney function.

Inclusion Criteria

I am a boy or a girl not pregnant or breastfeeding, aged 2 to under 12.

My body weight is at least 12 kg.

In the opinion of the investigator, the participant would benefit from long term oral HAE prophylaxis

+1 more

Exclusion Criteria

I have been diagnosed with another type of recurring swelling attacks.

A family member has died suddenly from heart issues.

Current participation in any other investigational drug study or received another investigational drug within 30 days of enrollment

+3 more

Participant Groups

The study tests berotralstat in young patients to find the right dose based on their weight and assess its safety. The goal is to see if this drug can help prevent swelling attacks caused by HAE in these children.

1Treatment groups

Experimental Treatment

Group I: BerotralstatExperimental Treatment1 Intervention

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.