NCT04933721

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
139

participants targeted

Target at P25-P50 for phase_3

Timeline
64mo left

Started Jul 2021

Longer than P75 for phase_3

Geographic Reach
11 countries

16 active sites

Status
enrolling by invitation

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress48%
Jul 2021Aug 2031

First Submitted

Initial submission to the registry

June 14, 2021

Completed
8 days until next milestone

First Posted

Study publicly available on registry

June 22, 2021

Completed
1 month until next milestone

Study Start

First participant enrolled

July 23, 2021

Completed
10 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2031

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2031

Last Updated

December 23, 2025

Status Verified

December 1, 2025

Enrollment Period

10 years

First QC Date

June 14, 2021

Last Update Submit

December 19, 2025

Conditions

Hereditary AngioedemaHAE

Keywords

prophylaxis

Outcome Measures

Primary Outcomes (5)

  • 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

Study Arms (1)

BCX7353 capsules or granules once daily

EXPERIMENTAL

Berotralstat (BCX7353) capsules or granules orally administered once daily.

Drug: berotralstat

Interventions

berotralstatDRUG

BCX7353 capsules or granules administered orally once daily

Also known as: Orladeyo
BCX7353 capsules or granules once daily

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (16)

Study Center

Ottawa, Canada

Location

Study Center

Brno, Czechia

Location

Study Center

Pilsen, Czechia

Location

Study Center

Grenoble, France

Location

Study Center

Marseille, France

Location

Study Center

Paris, France

Location

Study Center

Frankfurt, Germany

Location

Study Center

Skopje, North Macedonia

Location

Study Center

Krakow, Poland

Location

Study Center

Martin, Slovakia

Location

Study Center

Cape Town, South Africa

Location

Study Center

Daegu, South Korea

Location

Study Center

Gwangju, South Korea

Location

Study Center

Seoul, South Korea

Location

Study Center

Madrid, Spain

Location

Study Center

Bristol, United Kingdom

Location

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

berotralstat

Condition Hierarchy (Ancestors)

AngioedemaVascular DiseasesCardiovascular DiseasesHereditary Complement Deficiency DiseasesPrimary Immunodeficiency DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesUrticariaSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesHypersensitivity, ImmediateHypersensitivityImmune System DiseasesImmunologic Deficiency Syndromes

Study Officials

  • Vesna Grivcheva-Panoska, MD, PhD

    PHI University Clinic of Dermatology

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Masking Details
No masking as this is open-label.
Purpose
PREVENTION
Intervention Model
SINGLE GROUP
Model Details: Open-label berotralstat (BCX7353) orally administered QD.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 14, 2021

First Posted

June 22, 2021

Study Start

July 23, 2021

Primary Completion (Estimated)

August 1, 2031

Study Completion (Estimated)

August 1, 2031

Last Updated

December 23, 2025

Record last verified: 2025-12

Data Sharing

IPD Sharing
Will not share

Locations

GB(1)DE(1)CZ(2)FR(3)CA(1)ES(1)KR(3)SL(1)NO(1)PL(1)ZA(1)