12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
OPuS-2
OPuS-2: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Two Dose Levels of BCX4161 for 12 Weeks as an Oral Prophylaxis Treatment for Attacks of Hereditary Angioedema
1 other identifier
interventional
110
8 countries
40
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Dec 2014
Shorter than P25 for phase_2
40 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 24, 2014
CompletedFirst Posted
Study publicly available on registry
December 1, 2014
CompletedStudy Start
First participant enrolled
December 17, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2016
CompletedStudy Completion
Last participant's last visit for all outcomes
January 1, 2016
CompletedResults Posted
Study results publicly available
November 17, 2025
CompletedNovember 17, 2025
November 1, 2025
1 year
November 24, 2014
January 31, 2017
November 3, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The Mean Acute Angioedema Attack Rate
An angioedema attack was defined as swelling at any location reported by participant, which had no swelling earlier. Total number of confirmed attacks during the treatment period standardized to a weekly attack-rate to adjust for the total duration of treatment. The attack rate was derived for each participant by treatment period. The weekly attack rate was equal to the total number of confirmed attacks during a treatment period divided by the duration of the treatment (in days) times 7 days.
12 weeks
Secondary Outcomes (6)
Number of Attack-free Days
12 weeks
Number of Participants Who Are Attack-free
12 weeks
Disease Activity, as Measured by the 84-day Angioedema Activity Score
12 weeks
Change From Baseline at Week 12, in Quality of Life as Measured by the Angioedema Quality of Life Questionnaire
Baseline (Day 1) and Week 12
Number of Participants With Treatment Emergent Adverse Events
From first dose up to 14 weeks
- +1 more secondary outcomes
Study Arms (3)
BCX4161 300 mg three times daily
EXPERIMENTALThree BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth
BCX4161 500 mg three times daily
EXPERIMENTALFive BCX4161 capsules (100 mg) to be taken three times daily by mouth
Placebo three times daily
PLACEBO COMPARATORFive placebo capsules to be taken three times daily by mouth
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (40)
Unknown Facility
Birmingham, Alabama, 35209, United States
Unknown Facility
Scottsdale, Arizona, 85251, United States
Unknown Facility
Granada Hills, California, 91344, United States
Unknown Facility
San Diego, California, 92093, United States
Unknown Facility
San Diego, California, 92112, United States
Unknown Facility
Walnut Creek, California, 94598, United States
Unknown Facility
Colorado Springs, Colorado, 80907, United States
Unknown Facility
Shreveport, Louisiana, 71106, United States
Unknown Facility
Chevy Chase, Maryland, 20815, United States
Unknown Facility
Boston, Massachusetts, 02114, United States
Unknown Facility
Plymouth, Minnesota, 55446, United States
Unknown Facility
St Louis, Missouri, 63141, United States
Unknown Facility
New York, New York, 10029, United States
Unknown Facility
Charlotte, North Carolina, 28277, United States
Unknown Facility
Cincinnati, Ohio, 45267, United States
Unknown Facility
Oklahoma City, Oklahoma, 73131, United States
Unknown Facility
Tulsa, Oklahoma, 74133, United States
Unknown Facility
Lake Oswego, Oregon, 97035, United States
Unknown Facility
Hershey, Pennsylvania, 17033, United States
Unknown Facility
Pittsburgh, Pennsylvania, 15241, United States
Unknown Facility
Dallas, Texas, 75231, United States
Unknown Facility
Galveston, Texas, 77555, United States
Unknown Facility
Fairfax, Virginia, 63141, United States
Unknown Facility
Spokane, Washington, 99204, United States
Unknown Facility
Tacoma, Washington, 98405, United States
Unknown Facility
Leuven, Belgium
Unknown Facility
Ottawa, Canada
Unknown Facility
Grenoble, France
Unknown Facility
Lille, France
Unknown Facility
Paris, France
Unknown Facility
Berlin, Germany
Unknown Facility
Frankfurt, Germany
Unknown Facility
Mörfelden-Walldorf, Germany
Unknown Facility
Budapest, Hungary
Unknown Facility
Milan, Italy
Unknown Facility
Birmingham, United Kingdom
Unknown Facility
Bristol, United Kingdom
Unknown Facility
Cardiff, United Kingdom
Unknown Facility
London, United Kingdom
Unknown Facility
Manchester, United Kingdom
Related Publications (1)
Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.
PMID: 36326435DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- VP Clinical Development
- Organization
- BioCryst Pharmaceuticals, Inc
Study Officials
- PRINCIPAL INVESTIGATOR
Marc Riedl, MD MS
UCSD
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 24, 2014
First Posted
December 1, 2014
Study Start
December 17, 2014
Primary Completion
January 1, 2016
Study Completion
January 1, 2016
Last Updated
November 17, 2025
Results First Posted
November 17, 2025
Record last verified: 2025-11