An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 154
- 试验地点
- 1
- 主要终点
- Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity
研究概览
简要总结
The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).
详细描述
This is a Phase 3, multi-center, open-label, global study with donidalorsen in up to approximately 144 participants with HAE-1 (Type I) and HAE-2 (Type II). There are two groups in this study: 1) participants who roll over from another study of donidalorsen (open-label extension [OLE] participants), and 2) new participants who are not rolling over from another study of donidalorsen and were previously maintained on HAE prophylactic therapy with lanadelumab, berotralstat, or a C1-esterase inhibitor (C1-INH). The length of participation in the study is approximately 70 weeks for OLE participants and 76 weeks for other participants. Participants will receive donidalorsen in an Extended Treatment Period for up to an additional 104 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
OLE Participants
Group 1 and Group 2 participants will be administered donidalorsen by SC injection for up to 157 weeks.
干预措施: Donidalorsen (Drug)
结局指标
主要结局
Percentage of Participants with at Least One Treatment-emergent Adverse Event (TEAE), Graded by Severity
时间窗: Up to approximately 70 weeks, plus 104 weeks for Group 1; up to approximately 76 weeks, plus 104 weeks for Group 2
次要结局
- Time-normalized Number of Investigator-confirmed HAE Attacks (per Month)(Week 1 to Week 157 for Group 1 and Group 2)
- Percentage of Investigator-confirmed HAE Attack-free Participants(Week 1 to Week 157 for Group 1 and Group 2)
- Time-normalized Number of Moderate or Severe Investigator-confirmed HAE Attacks (per Month)(Week 1 to Week 157 for Group 1 and Group 2)
- Number of Investigator-confirmed HAE Attacks Requiring Acute Therapy(Week 1 to Week 157 for Group 1 and Group 2)
- Angioedema Quality of Life (AE-QoL) Questionnaire Total Score(Up to 157 weeks for Group 1 and Group 2)