NCT02670720终止3 期
OPuS-4: An Open-label Study to Evaluate the Long-term Safety of Avoralstat in Subjects With Hereditary Angioedema
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 6
- 主要终点
- Proportion of subjects who discontinue due to a treatment-emergent adverse event, who experience a treatment-emergent serious AE, who experience a Grade 3 or 4 AE and who experience Grade 3 or 4 laboratory abnormalities
研究概览
简要总结
This is an open-label study designed to evaluate the long-term safety of prophylactic avoralstat (500 mg three times daily) when given to approximately 150 patients with hereditary angioedema (HAE) for a duration of up to 72 weeks. The study will also evaluate the long-term efficacy and impact on quality of life of avoralstat prophylactic treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
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
- •Males and non-pregnant, non-lactating females age ≥ 18 years.
- •Provide written, informed consent.
- •Prior completion of an avoralstat therapeutic study OR a confirmed diagnosis of HAE Type 1 or HAE Type
- •Access to appropriate medication for the treatment of acute HAE attacks.
- •Adequate contraception.
排除标准
- •Females who are pregnant or breast feeding.
- •Clinically significant medical condition or medical history.
- •Abnormal screening ECG, laboratory or urinalysis finding that is clinically significant.
- •Investigational drug exposure within 30 days (except avoralstat).
- •History of or current alcohol or drug abuse.
- •HIV or active HBV or HCV infection.
研究组 & 干预措施
avoralstat
Experimental
Five avoralstat capsules (100 mg) to be taken three times daily by mouth
干预措施: avoralstat (Drug)
结局指标
主要结局
Proportion of subjects who discontinue due to a treatment-emergent adverse event, who experience a treatment-emergent serious AE, who experience a Grade 3 or 4 AE and who experience Grade 3 or 4 laboratory abnormalities
时间窗: Up to 72 weeks
次要结局
- Quality of life as determined by the Angioedema Quality of Life Questionnaire(Up to 72 weeks)
- Durability in response (assessed as subject-reported HAE attacks)(change over time through 72 weeks)
- Angioedema attack rate (subject-reported HAE attacks normalized for time on study)(Up to 72 weeks)
- Quality of life as determined by the EQ-5D-5L(Up to 72 weeks)
- Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy(Up to 72 weeks)
研究者
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