NCT04933721

Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies

An Open-label Study to Provide Berotralstat Access to Subjects With Type 1 and 2 Hereditary Angioedema Who Were Previously Enrolled in Berotralstat Studies

• Status: Enrolling By Invitation
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 139 (estimated)
• Sponsor: BioCryst Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.

Detailed description

APeX-A, 7353-312, is a single-arm, open-label, multicenter study. The study will be conducted in countries where berotralstat is not available either commercially or via another mechanism and is planned in countries where pediatric participants have been enrolled in BCX7353-304 (Study 304). Adult and adolescent (≥12 years) participants will receive berotralstat 150 mg administered orally once daily (QD). Pediatric participants will receive a weight-appropriate dose. The study will assess the long term safety and tolerability of berotralstat. Eligible subjects will be enrolled directly into Study BCX7353-312 (Study 312) from Studies 302, 204, and 304. Subjects will receive berotralstat administered orally once daily (QD). Subjects will return to the clinic every 24 weeks for drug dispensation and safety monitoring. Participants form Studies 302 and 204 may remain on study for up to 480 weeks (approximately 10 years). Participants from Study 304 may remain in the study until 16 years of age and have access to berotralstat via another mechanism; or up to 5 years, whichever comes first. Safety and tolerability will be evaluated through assessment of serious adverse events (SAEs) and treatment-related, treatment-emergent adverse events (TEAEs). Up to 139 subjects are planned to enroll.

Conditions

• Hereditary Angioedema
• HAE

Interventions

Type	Name	Description
DRUG	berotralstat	BCX7353 capsules or granules administered orally once daily

Timeline

• Start date: 2021-07-23
• Primary completion: 2031-08-01
• Completion: 2031-08-01
• First posted: 2021-06-22
• Last updated: 2025-12-23

Locations

16 sites across 11 countries: Canada, Czechia, France, Germany, North Macedonia, Poland, Slovakia, South Africa, South Korea, Spain, United Kingdom

Regulatory

• FDA-regulated drug study