Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE

A Phase 2a Double-Blind Placebo-Controlled 2-Period Crossover Study to Evaluate the Safety and Efficacy of BCX4161 as a Prophylactic Treatment to Reduce the Frequency of Attacks in Subjects With Hereditary Angioedema

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01984788

Acronym

OPuS-1

Enrollment

Registered

2013-11-15

Start date

2013-11-30

Completion date

2014-05-31

Last updated

2014-08-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hereditary Angioedema

Keywords

Oral Prophylaxis

Brief summary

The purpose of this study is to determine whether BCX4161 given as a daily oral prophylactic treatment is safe and effective in reducing the number of acute attacks in patients with hereditary angioedema.

Interventions

DRUGBCX4161

DRUGPlacebo

Study design

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

PREVENTION

Masking

DOUBLE (Subject, Investigator)

Eligibility

Sex/Gender

ALL

Age

18 Years to 65 Years

Healthy volunteers

Inclusion criteria

Key Inclusion Criteria: * Written informed consent * Clinical and laboratory diagnosis of Hereditary Angioedema Type I or II * An average angioedema attack frequency of 1/week * Acceptable birth control measures Key

Exclusion criteria

* Concurrent use of defined treatments for prophylaxis * Pregnancy or breast-feeding * Clinically significant medical condition, laboratory abnormality or medical history

Design outcomes

Primary

Measure	Time frame
Number of acute angioedema attacks	Over 28 days of treatment

Secondary

Measure	Time frame
Incidence and severity of adverse events and laboratory abnormalities	Over 28 days of treatment
Number of attack free days	Over 28 days of treatment
Angioedema Quality of Life scores	Over 28 days of treatment
Angioedema activity scores	Over 28 days of treatment

Countries

Germany, United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026