A Randomized, Double-blind, Placebo-controlled, Dose-ranging, Parallel-group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BCX7353 as a Preventative Treatment to Reduce the Frequency of Attacks in Subjects With Hereditary Angioedema
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 75
- 主要终点
- Number of Confirmed HAE Attacks
研究概览
简要总结
This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE). In Part 1 of the study, eligible subjects will be randomized to receive oral BCX7353 or placebo for 4 weeks. Assuming successful completion of Part 1, additional subjects will be randomized in Part 2 to one of 2 lower doses of BCX7353 or placebo. Part 3 will enroll additional subjects into one of three doses of BCX7353 or placebo. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical and pharmacologic outcomes, and the safety and tolerability of each dose of BCX7353 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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 70 Years（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
- •Access to acute attack medications
- •Sexually active women of child-bearing potential and sexually active men must utilize 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, androgens or tranexamic acid for prophylaxis of HAE attacks
- •History of or current alcohol or drug abuse
- •Infection with hepatitis B, hepatitis C or HIV
- •Participation in any other investigational drug study currently or within the last 30 days
研究组 & 干预措施
Parts 1, 2 and 3: Placebo
Placebo capsules, administered once per day for 28 days
干预措施: Placebo (Drug)
Part 3: BCX7353 62.5 mg once daily
BCX7353 capsules, 62.5 mg dose administered once per day for 28 days
干预措施: BCX7353 (Drug)
Part 1: BCX7353 350 mg once daily
BCX7353 capsules, 350 mg dose administered once per day for 28 days
干预措施: BCX7353 (Drug)
Parts 2 and 3: BCX7353 250 mg once daily
BCX7353 capsules, 250 mg dose administered once per day for 28 days
干预措施: BCX7353 (Drug)
Parts 2 and 3: BCX7353 125 mg once daily
BCX7353 capsules, 125 mg dose administered once per day for 28 days
干预措施: BCX7353 (Drug)
结局指标
主要结局
Number of Confirmed HAE Attacks
时间窗: Investigators collected data from patient diaries from the first day of dosing through to Day 29 or last day of dosing +24 hrs (the effective dosing period).
Efficacy was evaluated by the number of acute angioedema attacks. To ensure that consistent, objective assessments were used in accepting subject-reported attack data, a panel of expert physicians in the treatment of HAE patients adjudicated all subject-reported attacks prior to their inclusion in primary efficacy analyses.
Proportion of Subjects Who Were HAE Attack-free During the Entire Dosing Period
时间窗: Investigators collected data from patient diaries from the first day of dosing through to Day 29 or last day of dosing +24 hrs (the effective dosing period).
Assessment of the proportion of subjects who had no HAE attacks during the entire dosing period
次要结局
- HAE Disease Activity - Modified Angioedema Activity Score(28-day treatment period + 1 day)
- HAE Attacks Requiring Treatment(Investigators collected data from patient diaries from the first day of dosing through to Day 29 or last day of dosing +24 hrs (the effective dosing period).)
- DASS (Depression, Anxiety and Stress Scales)(The DASS was administered at baseline (Day 1), Day 14, and Day 29.)
- Number of Confirmed Abdominal HAE Attacks(Investigators collected data from patient diaries from the first day of dosing through to Day 29 or last day of dosing +24 hrs (the effective dosing period).)
- Number of Confirmed Peripheral HAE Attacks(Investigators collected data from patient diaries from the first day of dosing through to Day 29 or last day of dosing +24 hrs (the effective dosing period).)
- Angioedema Quality of Life (AE-QoL)(The subject-completed AE-QoL was administered at baseline (Day 1) and at Day 29)