Phase 3
N=110
12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
Hereditary Angioedema · HAE
Bottom Line
View on ClinicalTrials.gov: NCT02303626 ↗Enrolled (actual)
110
Serious AEs
7.3%
Results posted
Nov 2025
Primary outcome: Primary: The Mean Acute Angioedema Attack Rate — 0.675; 0.589; 0.593 Confirmed attacks per week
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- BCX4161 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- BioCryst Pharmaceuticals
- Primary completion
- Jan 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Mean Acute Angioedema Attack Rate |
0.675; 0.589; 0.593 | — |
| SECONDARY Number of Attack-free Days |
62.8; 66.0; 63.6 | — |
| SECONDARY Number of Participants Who Are Attack-free |
1; 1; 0 | — |
| SECONDARY Disease Activity, as Measured by the 84-day Angioedema Activity Score |
113.9; 88.5; 97.2 | — |
| SECONDARY Change From Baseline at Week 12, in Quality of Life as Measured by the Angioedema Quality of Life Questionnaire |
-9.89; -17.45; -12.14 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events |
31; 35; 32 | — |
| SECONDARY Change From Baseline at Week 12 in Quality of Life, as Measured by the EuroQoL Five-dimensional, 5-level Questionnaire |
3.26; 2.41; 9.92 | — |
Summary
This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in participants with hereditary angioedema (HAE). Eligible participants will be randomized to receive one of two doses of BCX4161 or placebo for 12 weeks. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical outcomes, and the safety and tolerability of each dose of BCX4161 compared to placebo.
Eligibility Criteria
Key Inclusion Criteria
- A clinical diagnosis of HAE type I or II
- Documented HAE attacks within a defined calendar period; in the absence of documented HAE attacks, participants will be required to enter a run-in period to document attacks
- Access to acute attack medications
- Sexually active women of child-bearing potential and sexually active men must utilize highly effective contraception
Key Exclusion Criteria
- Women who are pregnant or breast-feeding
- Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study
- Use of C1INH or tranexamic acid for prophylaxis of HAE attacks
- Current participation in any other investigational drug study or within the last 30 days
- History of or current alcohol or drug abuse
- Infection with hepatitis B, hepatitis C or HIV
Data sourced from ClinicalTrials.gov (NCT02303626). 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.