A Long Term Safety Study of BCX7353 in Hereditary Angioedema

NCT ID: NCT03472040

Last Updated: 2023-06-18

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

387 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-02-16

Study Completion Date

2022-04-27

Brief Summary

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This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II Hereditary Angioedema (HAE).

Detailed Description

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Conditions

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Hereditary Angioedema HAE Prophylaxis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

PREVENTION

Blinding Strategy

NONE

Study Groups

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BCX7353 150 mg once daily

Group Type EXPERIMENTAL

BCX7353

Intervention Type DRUG

BCX7353 mg oral capsules administered once daily

Interventions

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BCX7353

BCX7353 mg oral capsules administered once daily

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Subjects with HAE Type I or II who either have participated in a previous BCX7353 study or, in selected countries, in the opinion of the Investigator are expected to derive benefit from an oral treatment for the prevention of angioedema attacks.
* Access to appropriate medication for treatment of acute attacks
* Acceptable effective contraception
* Written informed consent

Exclusion Criteria

* Pregnancy or breast-feeding
* Any clinically significant medical condition or 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
* Any laboratory parameter abnormality that, in the opinion of the Investigator, is clinically significant and relevant for this study
* Discontinuation of study drug due to a hypersensitivity reaction BCX7353 in a prior study
* Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
* Unacceptable noncompliance in a previous BCX7353 study (if applicable) as assessed by the Sponsor or Investigator
* Investigational drug exposure, other than BCX7353, within 30 days prior to the screening visit (or baseline if no screening visit)
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BioCryst Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Henriette Farkas, MD

Role: PRINCIPAL_INVESTIGATOR

Semmelweis University, Budapest, Hungary

Locations

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Study Center

Birmingham, Alabama, United States

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Scottsdale, Arizona, United States

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Bentonville, Arkansas, United States

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Little Rock, Arkansas, United States

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San Diego, California, United States

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Walnut Creek, California, United States

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Centennial, Colorado, United States

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Colorado Springs, Colorado, United States

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Wheat Ridge, Colorado, United States

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Waterbury, Connecticut, United States

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Tampa, Florida, United States

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Marietta, Georgia, United States

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Normal, Illinois, United States

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Evansville, Indiana, United States

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Indianapolis, Indiana, United States

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Overland Park, Kansas, United States

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Louisville, Kentucky, United States

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Chevy Chase, Maryland, United States

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Boston, Massachusetts, United States

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Ann Arbor, Michigan, United States

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Grand Rapids, Michigan, United States

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Rochester, Minnesota, United States

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Madison, Mississippi, United States

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St Louis, Missouri, United States

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Lincoln, Nebraska, United States

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Charlotte, North Carolina, United States

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Durham, North Carolina, United States

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Cincinnati, Ohio, United States

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Columbus, Ohio, United States

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Clackamas, Oregon, United States

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Happy Valley, Oregon, United States

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Hershey, Pennsylvania, United States

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East Providence, Rhode Island, United States

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Greer, South Carolina, United States

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Austin, Texas, United States

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Dallas, Texas, United States

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Irving, Texas, United States

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San Antonio, Texas, United States

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Murray, Utah, United States

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Seattle, Washington, United States

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Spokane, Washington, United States

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Milwaukee, Wisconsin, United States

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Adelaide, , Australia

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Campbelltown, , Australia

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Camperdown, , Australia

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Melbourne, , Australia

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Murdoch, , Australia

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Nedlands, , Australia

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Graz, , Austria

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Vienna, , Austria

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Odense, , Denmark

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Grenoble, , France

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Lille, , France

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Paris, , France

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Berlin, , Germany

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Frankfurt, , Germany

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Ulm, , Germany

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Central, , Hong Kong

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Budapest, , Hungary

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Ashkelon, , Israel

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Haifa, , Israel

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Tel Aviv, , Israel

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Tel Litwinsky, , Israel

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Milan, , Italy

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Padua, , Italy

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Salerno, , Italy

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Auckland, , New Zealand

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Wellington, , New Zealand

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Skopje, , North Macedonia

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Krakow, , Poland

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Belgrade, , Serbia

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Niš, , Serbia

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Martin, , Slovakia

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Cape Town, , South Africa

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Daegu, , South Korea

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Donggu, , South Korea

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Gyeonggi-do, , South Korea

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Seoul, , South Korea

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Barcelona, , Spain

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Madrid, , Spain

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Zurich, , Switzerland

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Birmingham, , United Kingdom

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Bristol, , United Kingdom

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Cambridge, , United Kingdom

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London, , United Kingdom

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Plymouth, , United Kingdom

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Southampton, , United Kingdom

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Countries

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United States Australia Austria Denmark France Germany Hong Kong Hungary Israel Italy New Zealand North Macedonia Poland Serbia Slovakia South Africa South Korea Spain Switzerland United Kingdom

References

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Farkas H, Peter JG, Stobiecki M, Anderson J, Aygoren-Pursun E, Hagin D, Jesenak M, Kessel A, Kiani-Alikhan S, Kinaciyan T, Manning M, Reshef A, Wu A, Iocca HA, Johnston DT, Noble L, Tomita D, Banerji A. Long-term safety and efficacy of once-daily berotralstat in patients with hereditary angioedema: APeX-S final results. Ann Allergy Asthma Immunol. 2025 Sep;135(3):311-319.e6. doi: 10.1016/j.anai.2025.06.004. Epub 2025 Jun 7.

Reference Type DERIVED
PMID: 40490218 (View on PubMed)

Peter JG, Desai B, Tomita D, Collis P, Stobiecki M. Assessment of HAE prophylaxis transition from androgen therapy to berotralstat: A subset analysis of the APeX-S trial. World Allergy Organ J. 2023 Nov 6;16(11):100841. doi: 10.1016/j.waojou.2023.100841. eCollection 2023 Nov.

Reference Type DERIVED
PMID: 38020288 (View on PubMed)

Riedl MA, Soteres D, Sublett JW, Desai B, Tomita D, Collis P, Bernstein JA; APeX-S Study Investigators. Hereditary angioedema outcomes in US patients switched from injectable long-term prophylactic medication to oral berotralstat. Ann Allergy Asthma Immunol. 2024 Apr;132(4):505-511.e1. doi: 10.1016/j.anai.2023.11.016. Epub 2023 Nov 24.

Reference Type DERIVED
PMID: 38006972 (View on PubMed)

Farkas H, Balla Z. A review of berotralstat for the treatment of hereditary angioedema. Expert Rev Clin Immunol. 2023 Feb;19(2):145-153. doi: 10.1080/1744666X.2023.2150611. Epub 2022 Nov 29.

Reference Type DERIVED
PMID: 36408587 (View on PubMed)

Riedl MA, Neville D, Cloud B, Desai B, Bernstein JA. Shared decision-making in the management of hereditary angioedema: An analysis of patient and physician perspectives. Allergy Asthma Proc. 2022 Sep 1;43(5):397-405. doi: 10.2500/aap.2022.43.220050. Epub 2022 Jul 12.

Reference Type DERIVED
PMID: 35820771 (View on PubMed)

Farkas H, Stobiecki M, Peter J, Kinaciyan T, Maurer M, Aygoren-Pursun E, Kiani-Alikhan S, Wu A, Reshef A, Bygum A, Fain O, Hagin D, Huissoon A, Jesenak M, Lindsay K, Panovska VG, Steiner UC, Zubrinich C, Best JM, Cornpropst M, Dix D, Dobo SM, Iocca HA, Desai B, Murray SC, Nagy E, Sheridan WP. Long-term safety and effectiveness of berotralstat for hereditary angioedema: The open-label APeX-S study. Clin Transl Allergy. 2021 Jun;11(4):e12035. doi: 10.1002/clt2.12035.

Reference Type DERIVED
PMID: 34161665 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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BCX7353-204

Identifier Type: -

Identifier Source: org_study_id

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