A Phase 3 Trial to Evaluate the Long-Term Safety and Efficacy of Navenibart in Participants With Hereditary Angioedema - ORBIT-EXPANSE
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 145
- 试验地点
- 42
- 主要终点
- Incidence of treatment-emergent adverse events (TEAEs)
研究概览
简要总结
This is a Phase 3 multicenter trial in 2 parts to evaluate the long-term safety and efficacy of navenibart in adult and adolescent participants with hereditary angioedema (HAE) who participated in STAR-0215-301 (NCT06842823; ALPHA-ORBIT). Part 1 provides all participants with navenibart in a dose-controlled fashion; Part 2 introduces a personalized dosing option (PDO) for participants based on individual needs.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- None
盲法说明
All participants will knowingly receive navenibart, however participants, care providers, investigators, and outcome assessors will be blinded to their treatment arm for a duration specific to the individual.
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
- •1. Participants from STAR-0215-301 who met one of the following conditions:
- •Completed STAR-0215-301 through the Day 181 visit
- •Withdrew from STAR-0215-301 but met the following criteria:
- •i. Received 2 doses of IP ii. completed ≥ 2 months of trial follow-up after the second dose of IP iii. Met other eligibility criteria as assessed by Investigator
排除标准
- •Participation in an investigational clinical trial other than STAR-0215- 301 in the 30 days or any exposure to an investigational drug (other than navenibart in STAR-0215-301) within 5 half-lives before informed consent/assent
- •Any exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption (such as hormonal contraceptives or hormone replacement therapy) within 30 days before Screening.
- •Known sensitivity to the ingredients in the formulation of IP
研究组 & 干预措施
Adolescent Dosing Regimen 1
Participants will receive 300 mg of navenibart every 3 months.
干预措施: navenibart (Drug)
Adult Dosing Regimen 2
Participants will receive 300 mg of navenibart every 3 months.
干预措施: navenibart (Drug)
Adult Dosing Regimen 1
Participants will receive 600 mg of navenibart every 3 months.
干预措施: navenibart (Drug)
Adult 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
Incidence of treatment-emergent adverse events (TEAEs)
时间窗: Day 1 through Month 48
次要结局
- Time to first investigator-confirmed HAE attack after first dose(Day 1 through Month 48)
- The number of participants responding to treatment, defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction in investigator-confirmed HAE attack rate(Day 1 through Month 48)
- Number of participants with no investigator-confirmed HAE attacks(Day 1 through Month 48)
- Number of time-normalized investigator-confirmed HAE attacks(Day 1 through Month 48)
- Number of moderate or severe investigator-confirmed HAE attacks(Day 1 through Month 48)
- Number of investigator-confirmed HAE attacks that require on-demand treatment(Day 1 through Month 48)
- Percent reduction in monthly investigator-confirmed HAE attacks(Day 1 through Month 48)
- Angioedema Quality of Life questionnaire total score(Day 1 through Month 48)
- Number of time-normalized investigator-confirmed HAE attacks(Day 1 through Month 48)
- Number of moderate or severe investigator-confirmed HAE attacks(Day 1 through Month 48)
- Number of investigator-confirmed HAE attacks that require on-demand treatment(Day 1 through Month 48)
- Percent reduction in monthly investigator-confirmed HAE attacks(Day 1 through Month 48)
- Time to first investigator-confirmed HAE attack after first dose(Day 1 through Month 48)
- The number of participants responding to treatment, defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction in investigator-confirmed HAE attack rate(Day 1 through Month 48)
- Number of participants with no investigator-confirmed HAE attacks(Day 1 through Month 48)
- Angioedema Quality of Life questionnaire total score(Day 1 through Month 48)