Status:

COMPLETED

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

Lead Sponsor:

BioCryst Pharmaceuticals

Conditions:

Hereditary Angioedema, HAE

Eligibility:

All Genders

12+ years

Phase:

PHASE3

Brief Summary

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 a...

Eligibility Criteria

Inclusion

  • Key
  • 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
  • Key

Exclusion

  • 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

Key Trial Info

Start Date :

December 27 2018

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

July 8 2021

Estimated Enrollment :

19 Patients enrolled

Trial Details

Trial ID

NCT03873116

Start Date

December 27 2018

End Date

July 8 2021

Last Update

July 19 2024

Active Locations (10)

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Page 1 of 3 (10 locations)

1

Study Site

Chiba, Japan

2

Study Site

Gunma, Japan

3

Study Site

Hokkaido, Japan

4

Study Site

Nagoya, Japan