Phase 2
N=75
Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema
Hereditary Angioedema (HAE)
Bottom Line
View on ClinicalTrials.gov: NCT02870972 ↗Enrolled (actual)
75
Serious AEs
1.3%
Results posted
Mar 2021
Primary outcome: Primary: Number of Confirmed HAE Attacks — 0.519; 0.527; 0.249; 0.852 HAE attacks per week — p=<0.001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- BCX7353 (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- BioCryst Pharmaceuticals
- Primary completion
- Aug 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Confirmed HAE Attacks |
0.519; 0.527; 0.249; 0.852; 0.494; 0.952 | <0.001 sig |
| PRIMARY Proportion of Subjects Who Were HAE Attack-free During the Entire Dosing Period |
4; 3; 4; 0; 11; 1 | 0.155 |
| SECONDARY Number of Confirmed Abdominal HAE Attacks |
0.366; 0.324; 0.173; 0.404; 0.309; 0.370 | 0.439 |
| SECONDARY Number of Confirmed Peripheral HAE Attacks |
0.160; 0.143; 0.098; 0.463; 0.179; 0.543 | <0.001 sig |
| SECONDARY HAE Attacks Requiring Treatment |
0.483; 0.449; 0.218; 0.833; 0.450; 0.776 | 0.006 sig |
| SECONDARY HAE Disease Activity - Modified Angioedema Activity Score |
14.070; 9.416; 7.256; 22.559; 18.519 | 0.209 |
| SECONDARY Angioedema Quality of Life (AE-QoL) |
-12.59; -13.39; -28.95; -12.30; -4.47 | 0.135 |
| SECONDARY DASS (Depression, Anxiety and Stress Scales) |
-7.627; -6.429; -10.840; -9.141; 0.643 | 0.067 |
Summary
This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE). In Part 1 of the study, eligible subjects will be randomized to receive oral BCX7353 or placebo for 4 weeks. Assuming successful completion of Part 1, additional subjects will be randomized in Part 2 to one of 2 lower doses of BCX7353 or placebo. Part 3 will enroll additional subjects into one of three doses of BCX7353 or placebo. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical and pharmacologic outcomes, and the safety and tolerability of each dose of BCX7353 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
- Access to acute attack medications
- Sexually active women of child-bearing potential and sexually active men must utilize 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, androgens or tranexamic acid for prophylaxis of HAE attacks
- History of or current alcohol or drug abuse
- Infection with hepatitis B, hepatitis C or HIV
- Participation in any other investigational drug study currently or within the last 30 days
Data sourced from ClinicalTrials.gov (NCT02870972). 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.