2025-523499-22-00招募中3 期
An Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less than 12 Years Old with Hereditary Angioedema [Donidalorsen Treatment in Children with Hereditary Angioedema]
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 4
- 试验地点
- 4
- 主要终点
- 1. Incidence and severity of treatment-emergent adverse events (TEAEs)
研究概览
简要总结
Evaluate the safety, tolerability and pharmacokinetics of donidalorsen in pediatric participants with HAE
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
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: a. Documented clinical history consistent with HAE b. Diagnostic testing results that confirm HAE-1/HAE-2
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
- •Concurrent diagnosis of any other type of recurrent angioedema, including idiopathic angioedema or HAE with normal C1-INH (HAE-nC1-INH or Type III).
- •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.
结局指标
主要结局
1. Incidence and severity of treatment-emergent adverse events (TEAEs)
1. Incidence and severity of treatment-emergent adverse events (TEAEs)
2. Plasma pharmacokinetics and concentrations of donidalorsen
2. Plasma pharmacokinetics and concentrations of donidalorsen
次要结局
- 1. The time-normalized number of investigator-confirmed HAE attacks (per month) over the period of 12 months.
- 3. The percentage of investigator-confirmed HAE attack-free participants over the period of 12 months.
- 4. The time-normalized number of moderate or severe investigatorconfirmed HAE attacks (per month) over the period of 12 months.
- 5. The number of participants with a clinical response defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction from Baseline in investigator-confirmed HAE attack rate over the period of 12 months.
- 6. The number of investigator-confirmed HAE attacks requiring rescue treatment (i.e., acute therapy) over the period of 12 months.
- 7. Change in PKK levels in plasma over the period of 12 months.
- 8. Changes in Pediatrics Quality of Life (PedsQL) scores for participants over the period of 12 months.
研究者
Global Regulatory Affairs
Scientific
Ionis Pharmaceuticals Inc.
研究点 (4)
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