A Long Term Safety Study of BCX7353 in Hereditary Angioedema

NCT03472040 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: BCX7353-204
• Study Type: interventional
• Target: 387
• Locations: 19 countries
• Sites: 85

Brief Summary

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).

Conditions

Hereditary Angioedema; HAE; Prophylaxis

Keywords

BCX7353; Hereditary Angioedema; HAE

Outcome Measures

Primary Outcomes (1)

• Safety & Tolerability

  The number and percentage of subjects with treatment-emergent adverse events.

  Up to 96 weeks (US) / 216 weeks (Rest of World (ROW)).

Secondary Outcomes (4)

• Incidence of Acute Attacks of Angioedema in Subjects During Treatment

  Up to 96 weeks (US) / 216 weeks (ROW)

• The Durability of Response to Treatment

  Up to 96 weeks (US) / 216 weeks (ROW)

• Patient Reported Quality of Life (QoL) During Treatment

  Up to 96 weeks (US) / 216 weeks (ROW)

• Patient's Satisfaction With Medication During Long Term Administration of Berotralstat

  Up to 96 weeks (US) / 216 weeks (ROW)

Study Arms (1)

BCX7353 150 mg once daily

EXPERIMENTAL

Drug: BCX7353

Interventions

BCX7353 DRUG

BCX7353 mg oral capsules administered once daily

BCX7353 150 mg once daily

Eligibility Criteria

• Age: 12 Years+
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• BioCryst Pharmaceuticals: lead

Study Sites (87)

Study Center

Birmingham, Alabama, 35209, United States

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Scottsdale, Arizona, 85251, United States

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Bentonville, Arkansas, 72712, United States

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Little Rock, Arkansas, 72205, United States

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San Diego, California, 92123, United States

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Walnut Creek, California, 94598, United States

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Centennial, Colorado, 80112, United States

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Colorado Springs, Colorado, 80907, United States

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Wheat Ridge, Colorado, 80033, United States

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Waterbury, Connecticut, 06708, United States

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Tampa, Florida, 33613, United States

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Marietta, Georgia, 30060, United States

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Normal, Illinois, 61761, United States

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Evansville, Indiana, 47713, United States

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Indianapolis, Indiana, 46202, United States

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Overland Park, Kansas, 66210, United States

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Louisville, Kentucky, 40215, United States

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Chevy Chase, Maryland, 20815, United States

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Boston, Massachusetts, 02114, United States

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Ann Arbor, Michigan, 48106, United States

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Grand Rapids, Michigan, 49506, United States

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Rochester, Minnesota, 55905, United States

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Madison, Mississippi, 39110, United States

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St Louis, Missouri, 63141, United States

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Lincoln, Nebraska, 68505, United States

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Charlotte, North Carolina, 28277, United States

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Durham, North Carolina, 27705, United States

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Cincinnati, Ohio, 45231, United States

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Columbus, Ohio, 43235, United States

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Clackamas, Oregon, 97015, United States

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Happy Valley, Oregon, 97086, United States

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Hershey, Pennsylvania, 17033, United States

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East Providence, Rhode Island, 02914, United States

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Greer, South Carolina, 29651, United States

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Austin, Texas, 78731, United States

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Dallas, Texas, 75231, United States

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Irving, Texas, 75063, United States

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San Antonio, Texas, 78229, United States

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Murray, Utah, 84107, United States

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Seattle, Washington, 98115, United States

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Spokane, Washington, 99202, United States

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Milwaukee, Wisconsin, 53226, 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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Related Publications (6)

• 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.

  PMID: 40490218 DERIVED

• 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.

  PMID: 38020288 DERIVED

• 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.

  PMID: 38006972 DERIVED

• 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.

  PMID: 36408587 DERIVED

• 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.

  PMID: 35820771 DERIVED

• 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.

  PMID: 34161665 DERIVED

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

berotralstat

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Results Point of Contact

• Title: Study Director
• Organization: BioCryst Pharmaceuticals Inc

clinicaltrials@biocryst.com

+1 919-859-1302

Study Officials

• Henriette Farkas, MD

  Semmelweis University, Budapest, Hungary

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 27, 2018
• First Posted: March 21, 2018
• Study Start: February 16, 2018
• Primary Completion: April 27, 2022
• Study Completion: April 27, 2022
• Last Updated: June 18, 2023
• Results First Posted: June 18, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

HK (1); FR (3); SE (2); AU (6); DK (1); SL (1); IL (4); GB (6); KR (4); ES (2); CH (1); US (42); ZA (1); IT (3); HU (1); NZ (2); NO (1); DE (3); PL (1)