An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 16
- 主要终点
- Number of Participants with Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with hereditary angioedema (HAE) Type I (HAE-1) or Type II (HAE-2).
详细描述
This is an open-label study to evaluate the safety, efficacy, and pharmacokinetics (PK) and pharmacodynamics (PD) of donidalorsen in pediatric participants age 2 to less than 12 years old with HAE Type I (HAE-1) or Type II (HAE-2). The study consists of 3 parts: 1) a 3-month Screening Period, 2) a one-year Treatment Period, and 3) a 3-month Post-Treatment Period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must be between the ages of 2 and less than 12 years, inclusive, at the time of informed consent and, as applicable, assent.
- •Must weigh at least 9 kg at the time of informed consent and, as applicable, assent.
- •Documented diagnosis of HAE-1/HAE-2 based upon both of the following:
- •Documented clinical history consistent with HAE (SC or mucosal, non-pruritic swelling episodes without accompanying urticaria).
- •Diagnostic testing results that confirm HAE-1/HAE-2: C1-inhibitor (C1-INH) functional level <50% normal level AND complement factor C4 level below the lower limit of normal (LLN); OR a known pathogenic mutation in the SERPING1 gene.
排除标准
- •Must not have any screening laboratory abnormalities or any other clinically significant abnormalities during screening that would render a participant unsuitable for inclusion.
- •Must not have been treated with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer.
- •Concurrent diagnosis of any other type of recurrent angioedema, including idiopathic angioedema or HAE with normal C1-INH (HAE-nC1-INH or Type III).
- •Note: Other protocol-specified inclusion/exclusion criteria may apply.
研究组 & 干预措施
Donidalorsen: Group 2
Participant weighing greater than or equal to (≥)26 kg to <41 kg, will be administered donidalorsen over the period of one year.
干预措施: Donidalorsen (Drug)
Donidalorsen: Group 1
Participant weighing 9 kilograms (kg) to less than (<)26 kg, will be administered donidalorsen over the period of one year.
干预措施: Donidalorsen (Drug)
Donidalorsen: Group 3
Participant weighing ≥41kg, will receive donidalorsen over the period of one year.
干预措施: Donidalorsen (Drug)
结局指标
主要结局
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
时间窗: over the period of approximately 17 months
Maximum Plasma Concentration (Cmax) of Donidalorsen
时间窗: over the period of approximately 17 months
Maximum Time to Reach Cmax (Tmax) of Donidalorsen
时间窗: over the period of approximately 17 months
Trough Plasma Concentration (Ctrough) of Donidalorsen
时间窗: over the period of approximately 17 months
次要结局
- Time-Normalized Number of Investigator-Confirmed HAE Attacks (per Month)(over the period of 12 months)
- Percentage of Investigator-Confirmed HAE Attack-free Participants(over the period of 12 months)
- Time-Normalized Number of Moderate or Severe Investigator-Confirmed HAE Attacks (per Month)(over the period of 12 months)
- Number of Participants with a Clinical Response Defined as a ≥ 50 Percent (%), ≥ 70% and ≥ 90% Reduction from Baseline in Investigator-Confirmed HAE Attack Rate(over the period of 12 months)
- Change From Baseline in Prekallikrein (PKK) Levels in Plasma(over the period of 12 months)
- Percent Change From Baseline in PKK Levels in Plasma(over the period of 12 months)
- Time-Normalized Number of Investigator-Confirmed HAE Attacks Requiring Rescue Treatment(over the period of 12 months)
- Changes in Pediatrics Quality of Life (PedsQL) Scores for Participants(over the period of 12 months)