Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan

NCT ID: NCT03873116

Last Updated: 2024-07-19

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-12-27

Study Completion Date

2021-07-08

Brief Summary

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This is a phase 3, multi-center, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE who live in Japan.

Detailed Description

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Conditions

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Hereditary Angioedema, HAE

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

PREVENTION

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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BCX7353 110mg once daily

BCX7353 capsules administered orally once daily

Group Type EXPERIMENTAL

BCX7353 capsules

Intervention Type DRUG

BCX7353 capsules administered orally once daily

BCX7353 150mg once daily

BCX7353 capsules administered orally once daily

Group Type EXPERIMENTAL

BCX7353 capsules

Intervention Type DRUG

BCX7353 capsules administered orally once daily

Placebo

Matching placebo oral capsules administered orally once daily

Group Type PLACEBO_COMPARATOR

Placebo oral capsule

Intervention Type DRUG

Matching placebo capsules administered orally once daily

Interventions

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BCX7353 capsules

BCX7353 capsules administered orally once daily

Intervention Type DRUG

BCX7353 capsules

BCX7353 capsules administered orally once daily

Intervention Type DRUG

Placebo oral capsule

Matching placebo capsules administered orally once daily

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* A clinical diagnosis of hereditary angioedema (HAE) Type 1 or Type 2, defined as having a C1-INH functional level and a C4 level below the lower limit of the normal (LLN) reference range, as assessed during the Screening period.
* 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 expert-confirmed attacks during the run-in period of 56 days from the Screening visit.
* 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
* 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
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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Isao Ohsawa

Role: PRINCIPAL_INVESTIGATOR

Saiyu Soka Hospital

Locations

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Study Site

Chiba, , Japan

Site Status

Study Site

Gunma, , Japan

Site Status

Study Site

Hokkaido, , Japan

Site Status

Study Site

Nagoya, , Japan

Site Status

Study Site

Osaka, , Japan

Site Status

Study Center

Saga, , Japan

Site Status

Study Site

Saitama, , Japan

Site Status

Study Site

Shimane, , Japan

Site Status

Study Site

Shizuoka, , Japan

Site Status

Study Site

Tokyo, , Japan

Site Status

Countries

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Japan

References

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Honda D, Hide M, Fukuda T, Koga K, Morita E, Moriwaki S, Sasaki Y, Suzuki Y, Collis P, Johnston DT, Tomita D, Desai B, Ohsawa I. Berotralstat for long-term prophylaxis of hereditary angioedema in Japan: Parts 2 and 3 of the randomized APeX-J Phase III trial. World Allergy Organ J. 2024 Mar 2;17(3):100882. doi: 10.1016/j.waojou.2024.100882. eCollection 2024 Mar.

Reference Type DERIVED
PMID: 38445295 (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)

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.

Reference Type DERIVED
PMID: 36326435 (View on PubMed)

Ohsawa I, Honda D, Suzuki Y, Fukuda T, Kohga K, Morita E, Moriwaki S, Ishikawa O, Sasaki Y, Tago M, Chittick G, Cornpropst M, Murray SC, Dobo SM, Nagy E, Van Dyke S, Reese L, Best JM, Iocca H, Collis P, Sheridan WP, Hide M. Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: A phase 3 randomized trial. Allergy. 2021 Jun;76(6):1789-1799. doi: 10.1111/all.14670. Epub 2020 Dec 23.

Reference Type DERIVED
PMID: 33247955 (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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BCX7353-301

Identifier Type: -

Identifier Source: org_study_id

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