A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Navenibart in Participants With Hereditary Angioedema (HAE)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 157
- 试验地点
- 118
- 主要终点
- Number of time-normalized investigator-confirmed HAE attacks during the 6-month Treatment Period.
研究概览
简要总结
This is a Phase 3 multicenter, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of subcutaneous administration of navenibart in adult and adolescent participants with type 1 or type 2 hereditary angioedema (HAE). The goal of this clinical trial is to evaluate the efficacy and safety of navenibart compared to placebo in preventing HAE attacks in participants with HAE.
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)
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
- •Documented diagnosis of HAE (Type 1 or 2). The following must be met:
- •Documented clinical history consistent with HAE
- •Lab findings consistent with HAE Type 1 or 2
- •Experienced at least 2 HAE attacks during the Run-In period, as confirmed by an investigator based on meeting the protocol-specified definition of an HAE attack.
排除标准
- •Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH (also known as HAE type 3), idiopathic angioedema, or angioedema associated with urticaria.
- •Use of therapies prescribed for the prevention of HAE attacks may not be used during the trial or within the below time frames prior to the Run-In Period (adult participants may be on these medications at the time of the Screening Visit, but will need to washout prior to entering the Run-In Period).
- •Tranexamic acid, oral danazol, oral stanazolol, and oral oxandrolone within 3 days prior to Run-In
- •Plasma-derived C1INH for LTP within 14 days prior to Run-In
- •Berotralstat within 21 days prior to Run-In
- •Lanadelumab within 70 days prior to Run-In
- •Garadacimab within 90 days prior to Run-In
研究组 & 干预措施
Adult Navenibart Dosing Regimen 1
Participants will receive 600 mg of navenibart every 3 months.
干预措施: navenibart (Drug)
Placebo (adult)
Participants will receive placebo every 3 months.
干预措施: Placebo (Drug)
Adolescent Navenibart Dosing Regimen 1
Participants will receive 600 mg of navenibart on Day 1 and 300 mg every 3 months starting at month 3.
干预措施: navenibart (Drug)
Adult Navenibart Dosing Regimen 2
Participants will receive 600 mg of navenibart on Day 1 and 300 mg every 3 months starting at month 3.
干预措施: navenibart (Drug)
Adult Navenibart Dosing Regimen 3
Participants will receive 600 mg of navenibart every 6 months.
干预措施: navenibart (Drug)
结局指标
主要结局
Number of time-normalized investigator-confirmed HAE attacks during the 6-month Treatment Period.
时间窗: Day 1 through Day 181
次要结局
- Number of moderate or severe investigator-confirmed HAE attacks during the 6-month Treatment Period.(Day 1 through Day 181)
- The number of participants responding to treatment, defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction from the Run-In Period in investigator-confirmed HAE attack rate (for adult participants: compared to placebo during the 6-month Treatment Period).(Baseline through Day 181)
- Number of participants with no investigator-confirmed HAE attacks during the 6-month Treatment Period.(Day 1 through Day 181)
- Number of investigator-confirmed HAE attacks that require on-demand treatment during the 6-month Treatment Period.(Day 1 through Day 181)
- Percent reduction in monthly investigator-confirmed HAE attacks in the 6-month Treatment Period versus the Run-In Period.(Baseline through Day 181)
- Time to first investigator-confirmed HAE attack after first and second dose.(Day 1 through Day 181)
- Change from baseline (Day 1) in the Angioedema Quality of Life questionnaire total score.(Day 1 through Day 181)