Clinical trial NCT02303626: 12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
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
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Hereditary Angioedema
HAE
Keywords: BCX4161
Prophylaxis
Prevention
HAE
Hereditary Angioedema
Lead sponsor: BioCryst Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2014-12-01
Enrollment: 110
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

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.


Eligibility criteria: Key Inclusion Criteria:

1. A clinical diagnosis of HAE type I or II
2. 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
3. Access to acute attack medications
4. Sexually active women of child-bearing potential and sexually active men must utilize highly effective contraception

Key Exclusion Criteria:

1. Women who are pregnant or breast-feeding
2. Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study
3. Use of C1INH or tranexamic acid for prophylaxis of HAE attacks
4. Current participation in any other investigational drug study or within the last 30 days
5. History of or current alcohol or drug abuse
6. Infection with hepatitis B, hepatitis C or HIV

Primary outcomes:
measure: The mean acute angioedema attack rate
timeFrame: 12 weeks
Secondary outcomes:
measure: Number of attack-free days
timeFrame: 12 weeks
measure: Number of subjects who are attack-free
timeFrame: 12 weeks
measure: Disease activity, as measured by the 84-day Angioedema Activity Score
timeFrame: 12 weeks
measure: Quality of Life, as measured by the Angioedema Quality of Life Questionnaire
timeFrame: 12 weeks
measure: Incidence and severity of adverse events and laboratory abnormalities
timeFrame: 12 weeks
measure: Quality of Life, as measured by the EuroQoL five-dimensional, 5-level Questionnaire
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
state: Alabama
city: Scottsdale
country: United States
state: Arizona
city: Granada Hills
country: United States
state: California
city: San Diego
country: United States
state: California
city: San Diego
country: United States
state: California
city: Walnut Creek
country: United States
state: California
city: Colorado Springs
country: United States
state: Colorado
city: Shreveport
country: United States
state: Louisiana
city: Chevy Chase
country: United States
state: Maryland
city: Boston
country: United States
state: Massachusetts
city: Plymouth
country: United States
state: Minnesota
city: St Louis
country: United States
state: Missouri
city: New York
country: United States
state: New York
city: Charlotte
country: United States
state: North Carolina
city: Cincinnati
country: United States
state: Ohio
city: Oklahoma City
country: United States
state: Oklahoma
city: Tulsa
country: United States
state: Oklahoma
city: Lake Oswego
country: United States
state: Oregon
city: Hershey
country: United States
state: Pennsylvania
city: Pittsburg
country: United States
state: Pennsylvania
city: Dallas
country: United States
state: Texas
city: Galveston
country: United States
state: Texas
city: Fairfax
country: United States
state: Virginia
city: Spokane
country: United States
state: Washington
city: Tacoma
country: United States
state: Washington
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
Study officials:
affiliation: UCSD
name: Marc Riedl, MD MS
role: PRINCIPAL_INVESTIGATOR
References:
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.