Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies (APeX-A)

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

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

Study Overview

• Status: Enrolling by invitation
• Conditions: Hereditary Angioedema; HAE
• Intervention / Treatment: Drug: berotralstat

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.

• Study Type: Interventional
• Enrollment (Estimated): 139
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • Ottawa, Canada
    • Study Center
• Czechia
  • Brno, Czechia
    • Study Center
  • Pilsen, Czechia
    • Study Center
• France
  • Grenoble, France
    • Study Center
  • Marseille, France
    • Study Center
  • Paris, France
    • Study Center
• Germany
  • Frankfurt, Germany
    • Study Center
• North Macedonia
  • Skopje, North Macedonia
    • Study Center
• Poland
  • Krakow, Poland
    • Study Center
• Slovakia
  • Martin, Slovakia
    • Study Center
• South Africa
  • Cape Town, South Africa
    • Study Center
• South Korea
  • Daegu, South Korea
    • Study Center
  • Gwangju, South Korea
    • Study Center
  • Seoul, South Korea
    • Study Center
• Spain
  • Madrid, Spain
    • Study Center
• United Kingdom
  • Bristol, United Kingdom
    • Study Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

• Males and females currently enrolled in BioCryst-sponsored Study 302, 204, or 304
• Participant or parent/legally designated representative (for participants <18 years of age) able to provide written informed consent.
• Would benefit from continued berotralstat treatment
• Acceptable effective contraception

Key Exclusion Criteria:

• Pregnancy or breast-feeding
• Any condition or situation, including medical history that, in the opinion of the investigator or sponsor, would interfere with the subject's safety or ability to participate in the study
• Use of other medications for long-term prophylaxis of HAE attacks at the Baseline visit or any time during the study.
• Use of any other investigational medicinal product at the Baseline visit or any time during the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BCX7353 capsules or granules once daily; Berotralstat (BCX7353) capsules or granules orally administered once daily.	Drug: berotralstat; BCX7353 capsules or granules administered orally once daily; Other Names: Orladeyo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number and proportion of subjects with a treatment-related TEAE	240 weeks
Number and proportion of subjects who experience a serious adverse event (SAE)	240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE	240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality	240 weeks
Number and proportion of subjects who discontinue due to a TEAE	240 weeks

Collaborators and Investigators

• Sponsor: BioCryst Pharmaceuticals
• Investigators: Principal Investigator: Vesna Grivcheva-Panoska, MD, PhD, PHI University Clinic of Dermatology

Study record dates

Study Major Dates

• Study Start (Actual): July 23, 2021
• Primary Completion (Estimated): August 1, 2031
• Study Completion (Estimated): August 1, 2031

Study Registration Dates

• First Submitted: June 14, 2021
• First Submitted That Met QC Criteria: June 14, 2021
• First Posted (Actual): June 22, 2021

Study Record Updates

• Last Update Posted (Estimated): December 23, 2025
• Last Update Submitted That Met QC Criteria: December 19, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: prophylaxis
• Additional Relevant MeSH Terms: Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Immune System Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immunologic Deficiency Syndromes; Skin Diseases; Urticaria; Skin Diseases, Vascular; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin and Connective Tissue Diseases; Angioedema; Angioedemas, Hereditary; Molecular Mechanisms of Pharmacological Action; Protease Inhibitors; Enzyme Inhibitors; Serine Proteinase Inhibitors; berotralstat
• Other Study ID Numbers: BCX7353-312

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes