Phase 3
Completed N=19
Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan
Hereditary Angioedema, HAE
Source: ClinicalTrials.gov NCT03873116 ↗
Enrolled (actual)
19
Serious AEs
13.0%
Results posted
Mar 2021
Primary outcomePrimary: Part 1: The Rate of Expert-confirmed HAE Attacks During Dosing in the Entire 24-week Treatment Period (Day 1 to Day 168) — 1.961; 1.089; 2.734 Angioedema event rate per 28 days — p=0.181
◆ Published Evidence
Established
48citations · ~10 / year
Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: A phase 3 randomized trial.
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 and Type II HAE who live in Japan.
Linked Publications (4)
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Oral berotralstat for the prophylaxis of hereditary angioedema attacks in patients in Japan: A phase 3 randomized trial.
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Interventions for the long-term prevention of hereditary angioedema attacks.
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Berotralstat for long-term prophylaxis of hereditary angioedema in Japan: Parts 2 and 3 of the randomized APeX-J Phase III trial.
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A review of berotralstat for the treatment of hereditary angioedema.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part 1: The Rate of Expert-confirmed HAE Attacks During Dosing in the Entire 24-week Treatment Period (Day 1 to Day 168) |
1.961; 1.089; 2.734 | 0.181 |
| PRIMARY Part 2: To Evaluate the Long-term Safety and Tolerability of Berotralstat 110 and 150 mg in Subjects With HAE |
6; 1; 7; 2; 6; 2 | — |
| PRIMARY Part 3: To Evaluate the Long-term Safety and Tolerability of Berotralstat Administered QD Over a 52- to up to 104-week Administration Period in Subjects With HAE. |
6; 0; 1; 0; 1; 0 | — |
| SECONDARY Part 1: Proportion of Days With Angioedema Symptoms Through 24 Weeks. |
0.258; 0.118; 0.240 | 0.814 |
| SECONDARY Part 1: Rate of Expert-confirmed Angioedema Events During Dosing in the Effective Treatment Period |
1.988; 1.136; 2.775 | 0.188 |
| SECONDARY Part 1: Change From Baseline in Angioedema Quality of Life (AE-QoL) Questionnaire at Week 24 (Total Score) |
-9.47; -15.82; 3.18 | 0.213 |
| SECONDARY Part 2: To Assess the Effectiveness of Berotralstat Over a 24- to 52-week Period |
-0.487; -0.686; -0.863; -1.386; -1.085; -0.889 | — |
| SECONDARY Part 2: To Evaluate QoL Following Berotralstat Administration Over a 24- to 52-week Period. |
-4.9; -19.1; -19.5; 0; -7.4; -17.2 | — |
| SECONDARY Part 3: To Assess the Effectiveness of Berotralstat Over a 52- to up to 104-week Administration Period |
0.883 | — |
| SECONDARY Part 3: To Evaluate QoL Following Berotralstat Administration Over a 52- to up to 104-week Period |
-11.11; -13.24; -17.38; -11.63; -20.40; -18.93 | — |
| SECONDARY Part 3: To Evaluate Subject's Satisfaction With Berotralstat During 52- to 104-week Administration Period |
11.7; 13.6; 11.7; 18.8; 16.1 | — |
Eligibility Criteria
Key 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
Key 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
Data sourced from ClinicalTrials.gov (NCT03873116) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.