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
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 110
- 主要终点
- The Mean Acute Angioedema Attack Rate
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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
研究组 & 干预措施
BCX4161 300 mg three times daily
Three BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth
干预措施: BCX4161 (Drug)
BCX4161 300 mg three times daily
Three BCX4161 capsules (100 mg) and two placebo capsules to be taken three times daily by mouth
干预措施: Placebo (Drug)
BCX4161 500 mg three times daily
Five BCX4161 capsules (100 mg) to be taken three times daily by mouth
干预措施: BCX4161 (Drug)
Placebo three times daily
Five placebo capsules to be taken three times daily by mouth
干预措施: Placebo (Drug)
结局指标
主要结局
The Mean Acute Angioedema Attack Rate
时间窗: 12 weeks
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.
次要结局
- Number of Participants Who Are Attack-free(12 weeks)
- Number of Attack-free Days(12 weeks)
- Change From Baseline at Week 12 in Quality of Life, as Measured by the EuroQoL Five-dimensional, 5-level Questionnaire(Baseline (Day 1) and Week 12)
- 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)