Back to results12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks (OPuS-2)
Primary Purpose
Hereditary Angioedema, HAE
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
BCX4161
Placebo
Sponsored by
About this trial
This is an interventional prevention trial for Hereditary Angioedema focused on measuring BCX4161, Prophylaxis, Prevention, HAE, Hereditary Angioedema
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, subjects 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
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
BCX4161 300 mg three times daily
BCX4161 500 mg three times daily
Placebo three times daily
Arm Description
Three BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth
Five BCX4161 capsules (100 mg) to be taken three times daily by mouth
Five placebo capsules to be taken three times daily by mouth
Outcomes
Primary Outcome Measures
The mean acute angioedema attack rate
Secondary Outcome Measures
Number of attack-free days
Number of subjects who are attack-free
Disease activity, as measured by the 84-day Angioedema Activity Score
Quality of Life, as measured by the Angioedema Quality of Life Questionnaire
Incidence and severity of adverse events and laboratory abnormalities
Quality of Life, as measured by the EuroQoL five-dimensional, 5-level Questionnaire
Full Information
NCT ID
NCT02303626
First Posted
November 24, 2014
Last Updated
February 15, 2016
Sponsor
BioCryst Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT02303626
Brief Title
12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
Acronym
OPuS-2
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2016
Overall Recruitment Status
Completed
Study Start Date
November 2014 (undefined)
Primary Completion Date
January 2016 (Actual)
Study Completion Date
January 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
BioCryst Pharmaceuticals
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in subjects with hereditary angioedema (HAE). Eligible subjects 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Angioedema, HAE
Keywords
BCX4161, Prophylaxis, Prevention, HAE, Hereditary Angioedema
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
110 (Actual)
8. Arms, Groups, and Interventions
Arm Title
BCX4161 300 mg three times daily
Arm Type
Experimental
Arm Description
Three BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth
Arm Title
BCX4161 500 mg three times daily
Arm Type
Experimental
Arm Description
Five BCX4161 capsules (100 mg) to be taken three times daily by mouth
Arm Title
Placebo three times daily
Arm Type
Placebo Comparator
Arm Description
Five placebo capsules to be taken three times daily by mouth
Intervention Type
Drug
Intervention Name(s)
BCX4161
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
The mean acute angioedema attack rate
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Number of attack-free days
Time Frame
12 weeks
Title
Number of subjects who are attack-free
Time Frame
12 weeks
Title
Disease activity, as measured by the 84-day Angioedema Activity Score
Time Frame
12 weeks
Title
Quality of Life, as measured by the Angioedema Quality of Life Questionnaire
Time Frame
12 weeks
Title
Incidence and severity of adverse events and laboratory abnormalities
Time Frame
12 weeks
Title
Quality of Life, as measured by the EuroQoL five-dimensional, 5-level Questionnaire
Time Frame
12 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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, subjects 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Marc Riedl, MD MS
Organizational Affiliation
UCSD
Official's Role
Principal Investigator
Facility Information:
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35209
Country
United States
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85251
Country
United States
City
Granada Hills
State/Province
California
ZIP/Postal Code
91344
Country
United States
City
San Diego
State/Province
California
ZIP/Postal Code
92093
Country
United States
City
San Diego
State/Province
California
ZIP/Postal Code
92112
Country
United States
City
Walnut Creek
State/Province
California
ZIP/Postal Code
94598
Country
United States
City
Colorado Springs
State/Province
Colorado
ZIP/Postal Code
80907
Country
United States
City
Shreveport
State/Province
Louisiana
ZIP/Postal Code
71106
Country
United States
City
Chevy Chase
State/Province
Maryland
ZIP/Postal Code
20815
Country
United States
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
City
Plymouth
State/Province
Minnesota
ZIP/Postal Code
55446
Country
United States
City
St Louis
State/Province
Missouri
ZIP/Postal Code
63141
Country
United States
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28277
Country
United States
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45267
Country
United States
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73131
Country
United States
City
Tulsa
State/Province
Oklahoma
ZIP/Postal Code
74133
Country
United States
City
Lake Oswego
State/Province
Oregon
ZIP/Postal Code
97035
Country
United States
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
City
Pittsburg
State/Province
Pennsylvania
ZIP/Postal Code
15241
Country
United States
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231
Country
United States
City
Galveston
State/Province
Texas
ZIP/Postal Code
77555
Country
United States
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
63141
Country
United States
City
Spokane
State/Province
Washington
ZIP/Postal Code
99204
Country
United States
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405
Country
United States
City
Leuven
Country
Belgium
City
Ottawa
Country
Canada
City
Grenoble
Country
France
City
Lille
Country
France
City
Paris
Country
France
City
Berlin
Country
Germany
City
Frankfurt
Country
Germany
City
Morfelden-Walldorf
Country
Germany
City
Budapest
Country
Hungary
City
Milano
Country
Italy
City
Birmingham
Country
United Kingdom
City
Bristol
Country
United Kingdom
City
Cardiff
Country
United Kingdom
City
London
Country
United Kingdom
City
Manchester
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
36326435
Citation
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.
Results Reference
derived
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12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
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