Phase 3
Completed N=121
Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE
Hereditary Angioedema · HAE
Source: ClinicalTrials.gov NCT03485911 ↗
Enrolled (actual)
121
Serious AEs
7.8%
Results posted
Mar 2021
Primary outcomePrimary: Part 1: The Rate of Investigator-confirmed HAE Attacks During Dosing in the Entire 24-week Treatment Period (Day 1 to Day 168) — 1.65; 1.31; 2.35 HAE attack rate per 28 days — p=0.024
◆ Published Evidence
Highly cited
163citations · ~33 / year
Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: A randomized, double-blind, placebo-controlled phase 3 trial.
Summary
This is a phase 3, multicenter, 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.
Linked Publications (4)
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Oral once-daily berotralstat for the prevention of hereditary angioedema attacks: A randomized, double-blind, placebo-controlled phase 3 trial.
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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).
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Interventions for the long-term prevention of hereditary angioedema attacks.
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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 Investigator-confirmed HAE Attacks During Dosing in the Entire 24-week Treatment Period (Day 1 to Day 168) |
1.65; 1.31; 2.35 | 0.024 sig |
| PRIMARY Part 2 & 3: To Evaluate the Long-term Safety and Tolerability of Berotralstat 110 and 150 mg in Subjects With HAE |
22; 13; 27; 12; 67; 4 | — |
| SECONDARY Part 1: Change From Baseline in Angioedema Quality of Life Questionnaire at Week 24 (Total Score) |
-12.46; -14.59; -9.69 | 0.453 |
| SECONDARY Part 1: Proportion of Days With Angioedema Symptoms Through 24 Weeks |
0.134; 0.119; 0.197 | 0.025 sig |
| SECONDARY Part 1: Rate of Expert-confirmed Angioedema Events During Dosing in the Effective Treatment Period |
1.918; 1.552; 2.490 | 0.026 sig |
| SECONDARY Part 2: To Assess the Effectiveness of Berotralstat Over a 24- to 48 Week Period |
-1.383; -1.593; -1.229; -1.903; -1.543; -1.910 | — |
| SECONDARY To Evaluate Angioedema Quality of Life Questionnaire (Total Score) Following Berotralstat Administration for up to 144 Weeks |
-5.85; -9.70; -7.762; -10.58; -13.09; -11.33 | — |
| SECONDARY To Evaluate Treatment Satisfaction Questionnaire for Medication (TSQM) Following Berotralstat Administration for up to 144 Weeks |
-1.3; 2.4; -18.1; 4.3; 2.1; -19.2 | — |
Eligibility Criteria
Key Inclusion Criteria
- A clinical diagnosis of hereditary angioedema 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.
- 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
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 (NCT03485911) 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.