Phase 3
N=387
A Long Term Safety Study of BCX7353 in Hereditary Angioedema
Hereditary Angioedema · HAE · Prophylaxis
Bottom Line
View on ClinicalTrials.gov: NCT03472040 ↗Enrolled (actual)
387
Serious AEs
11.1%
Results posted
Jun 2023
Primary outcome: Primary: Safety & Tolerability — 94; 240; 58; 119 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- BCX7353 (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- BioCryst Pharmaceuticals
- Primary completion
- Apr 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety & Tolerability |
94; 240; 58; 119; 23; 20 | — |
| SECONDARY Incidence of Acute Attacks of Angioedema in Subjects During Treatment |
95; 231; 87; 214; 59; 99 | — |
| SECONDARY The Durability of Response to Treatment |
1.276; 1.079; 1.005; 0.694; 1.121; 0.591 | — |
| SECONDARY Patient Reported Quality of Life (QoL) During Treatment |
-8.85; -10.38; -11.20; -10.39; -11.92; -11.47 | — |
| SECONDARY Patient's Satisfaction With Medication During Long Term Administration of Berotralstat |
-4.1; -1.3; -0.6; -3.6; 1.5; -0.1 | — |
Summary
This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II Hereditary Angioedema (HAE).
Eligibility Criteria
Key Inclusion Criteria
- Subjects with HAE Type I or II who either have participated in a previous BCX7353 study or, in selected countries, in the opinion of the Investigator are expected to derive benefit from an oral treatment for the prevention of angioedema attacks.
- Access to appropriate medication for treatment of acute attacks
- 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
- Discontinuation of study drug due to a hypersensitivity reaction BCX7353 in a prior study
- Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
- Unacceptable noncompliance in a previous BCX7353 study (if applicable) as assessed by the Sponsor or Investigator
- Investigational drug exposure, other than BCX7353, within 30 days prior to the screening visit (or baseline if no screening visit)
Data sourced from ClinicalTrials.gov (NCT03472040). 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.