Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE
NCT ID: NCT03485911
Last Updated: 2023-06-26
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
121 participants
INTERVENTIONAL
2018-02-06
2022-04-06
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
PREVENTION
TRIPLE
Study Groups
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BCX7353 110 mg once daily
BCX7353 administered as oral capsules once daily
BCX7353 capsules
BCX7353 oral capsules administered once daily
BCX7353 150 mg once daily
BCX7353 administered as oral capsules once daily
BCX7353 capsules
BCX7353 oral capsules administered once daily
Placebo
Matching placebo administered as oral capsules once daily
Placebo oral capsule
Matching oral capsules administered once daily
Interventions
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BCX7353 capsules
BCX7353 oral capsules administered once daily
Placebo oral capsule
Matching oral capsules administered once daily
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Subject weight of ≥ 40 kg
* Access to and ability to use one or more acute medications approved by the relevant competent authority for the treatment of acute attacks of HAE
* Subjects must be medically appropriate for on-demand treatment as the sole medicinal management for their HAE during the study.
* Subjects must have a specified number of investigator-confirmed attacks during the run-in period of a maximum of 56 days from the Screening visit.
* Acceptable effective contraception
* Written informed consent
Exclusion Criteria
* 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
* Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
* Use of C1-INH within 14 days or use of androgens or tranexamic acid within 28 days prior to the Screening visit for prophylaxis of HAE attacks, or initiation of these drugs during the study
* Current participation in any other investigational drug study or received another investigational drug within 30 days of the Screening visit
* Prior enrollment in a BCX7353 study
12 Years
ALL
No
Sponsors
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BioCryst Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Bruce Zuraw, MD
Role: PRINCIPAL_INVESTIGATOR
UC San Diego School of Medicine, US HAE Angioedema Center
Locations
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Study center
Birmingham, Alabama, United States
Study center
Scottsdale, Arizona, United States
Study center
Little Rock, Arkansas, United States
Study Center
San Diego, California, United States
Study center
Santa Monica, California, United States
Study Center
Walnut Creek, California, United States
Study center
Colorado Springs, Colorado, United States
Study Center
Tampa, Florida, United States
Study center
Chevy Chase, Maryland, United States
Study Center
Boston, Massachusetts, United States
Study Center
Ann Arbor, Michigan, United States
Study center
Plymouth, Minnesota, United States
Study Center
St Louis, Missouri, United States
Study center
Belleville, New Jersey, United States
Study Center
Fair Lawn, New Jersey, United States
Study center
Piscataway, New Jersey, United States
Study Center
New York, New York, United States
Study Site
Charlotte, North Carolina, United States
Study Center
Durham, North Carolina, United States
Study Center
Cincinnati, Ohio, United States
Study center
Columbus, Ohio, United States
Study center
Clackamas, Oregon, United States
Study Center
Hershey, Pennsylvania, United States
Study center
Austin, Texas, United States
Study center
Dallas, Texas, United States
Study Center
San Antonio, Texas, United States
Study Center
Spokane, Washington, United States
Study Center
Vienna, , Austria
Study Center
Ottawa, Ontario, Canada
Study Center
Toronto, Ontario, Canada
Study Center
Québec, , Canada
Study Center
Brno, , Czechia
Study Center
Pilsen, , Czechia
Study Center
Grenoble, , France
Study Center
Paris, , France
Study Center
Berlin, , Germany
Study Center
Frankfurt, , Germany
Study Center
Budapest, , Hungary
Study Center
Skopje, , North Macedonia
Study Center
Sângeorgiu de Mureș, Jud. Mureș, Romania
Study Center
Barcelona, , Spain
Study Center
Madrid, , Spain
Study Center
Seville, , Spain
Study Center
Cambridge, , United Kingdom
Study Center
Frimley, , United Kingdom
Study Center
London, , United Kingdom
Study Center
Plymouth, , United Kingdom
Countries
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References
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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.
Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
Wedner HJ, Aygoren-Pursun E, Bernstein J, Craig T, Gower R, Jacobs JS, Johnston DT, Lumry WR, Zuraw BL, Best JM, Iocca HA, Murray SC, Desai B, Nagy E, Sheridan WP, Kiani-Alikhan S. Randomized Trial of the Efficacy and Safety of Berotralstat (BCX7353) as an Oral Prophylactic Therapy for Hereditary Angioedema: Results of APeX-2 Through 48 Weeks (Part 2). J Allergy Clin Immunol Pract. 2021 Jun;9(6):2305-2314.e4. doi: 10.1016/j.jaip.2021.03.057. Epub 2021 Apr 15.
Zuraw B, Lumry WR, Johnston DT, Aygoren-Pursun E, Banerji A, Bernstein JA, Christiansen SC, Jacobs JS, Sitz KV, Gower RG, Gagnon R, Wedner HJ, Kinaciyan T, Hakl R, Hanzlikova J, Anderson JT, McNeil DL, Fritz SB, Yang WH, Tachdjian R, Busse PJ, Craig TJ, Li HH, Farkas H, Best JM, Clemons D, Cornpropst M, Dobo SM, Iocca HA, Kargl D, Nagy E, Murray SC, Collis P, Sheridan WP, Maurer M, Riedl MA. Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: A randomized, double-blind, placebo-controlled phase 3 trial. J Allergy Clin Immunol. 2021 Jul;148(1):164-172.e9. doi: 10.1016/j.jaci.2020.10.015. Epub 2020 Oct 21.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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BCX7353-302
Identifier Type: -
Identifier Source: org_study_id
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