STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 90
- 试验地点
- 83
- 主要终点
- Efficacy of ADX-324 in preventing HAE attacks
研究概览
简要总结
This study will evaluate the efficacy and safety of ADX-324 in participants with Type 1 or Type 2 hereditary angioedema. The study will also evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and health-related quality of life measures.
详细描述
This Phase 3 study will evaluate the efficacy of 2 doses and regimens of ADX-324 in preventing HAE attacks compared with placebo in participants with Type I and Type II HAE. The study will also evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and health-related quality of life (HRQoL) measures.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Double blinded
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
- •Age ≥18 years at the time of signing informed consent.
- •Have a documented diagnosis of HAE-1/HAE-2 (Type I or II)
- •Experience ≥1 Investigator-confirmed HAE attack in the first 4 weeks of Screening or ≥2 Investigator-confirmed HAE attacks in 8 weeks of Screening
- •Able to use at least one acute therapy to treat HAE attacks (such as a plasma-derived or recombinant C1-INH concentrate or a BK2-receptor antagonist)
排除标准
- •Concurrent diagnosis of another form of recurrent angioedema (such as acquired angioedema, HAE with normal C1-INH (previously known as HAE Type III), idiopathic angioedema, or recurrent angioedema associated with urticaria).
- •Any clinically significant renal disease
- •Any clinically significant hepatic disease
- •Have used any of the following for long-term prevention of HAE attacks:
- •C1-INH agent (CINRYZE, HAEGARDA, RUCONEST) within 2 weeks prior to Screening.
- •Berotralstat (ORLADEYO) within 3 weeks prior to Screening.
- •Lanadelumab (TAKHZYRO) within 8 weeks prior to the Screening.
- •Androgen use within 12 weeks prior to Screening.
- •Received prior treatment with any RNA/DNA-based therapy for HAE or intolerant to any prior RNA/DNA-based therapy for any condition, excluding vaccines.
研究组 & 干预措施
ADX-324 Dose Level 1
干预措施: ADX-324 (Drug)
ADX-324 Dose Level 2
干预措施: ADX-324 (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Efficacy of ADX-324 in preventing HAE attacks
时间窗: Day 22 to Week 25
The time-normalized number of Investigator-confirmed HAE attacks per month for ADX-324 Dose Level 1 vs placebo
次要结局
- Efficacy of ADX-324 in preventing HAE attacks(Day 22 to Week 25)
- Evaluate the effects of ADX-324 on the quality and pattern of HAE attacks(Day 22 to Week 25)