A Phase 3, Multicenter, Randomized, Single-Blind, Dose-Ranging, Crossover Study to Evaluate the Safety and Efficacy of Intravenous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) for the Prevention of Angioedema Attacks in Children 6 to 11 Years of Age With Hereditary Angioedema
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 12
- 试验地点
- 16
- 主要终点
- Normalized Number of Angioedema Attacks Per Month in a Treatment Period
研究概览
简要总结
Primary Objective - To assess the relative efficacy of two dose levels of CINRYZE (500 Units and 1000 Units) administered by intravenous (IV) injection every 3 or 4 days to prevent angioedema attacks in children 6 to 11 years of age with hereditary angioedema (HAE).
Secondary Objectives - To assess the safety and tolerability, characterize the pharmacokinetics (PK) and pharmacodynamics (PD), and assess the immunogenicity of two dose levels of CINRYZE administered by IV injection in children 6 to 11 years of age with HAE.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Prevention
- 盲法
- Single (Participant)
入排标准
- 年龄范围
- 6 Years 至 11 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Type I or Type II HAE.
- •History of angioedema attacks.
排除标准
- •History of bleeding or clotting abnormality.
- •Diagnosis of acquired angioedema or known to have C1 INH antibodies.
- •History of allergic reaction to C1 esterase inhibitor or other blood products.
- •Receipt of any experimental agents other than those required for prevention or treatment of angioedema attacks within 30 days prior to screening.
结局指标
主要结局
Normalized Number of Angioedema Attacks Per Month in a Treatment Period
时间窗: From start of treatment up to 12 weeks during each treatment period
Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.
次要结局
- Cumulative Daily-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period(From start of treatment up to 12 weeks during each intervention period)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Dose Group(From start of study treatment up to 25 weeks)
- Plasma Concentration of Complement C4(Pre-dose and 1 h post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention period)
- Cumulative Attack-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period(From start of treatment up to 12 weeks during each treatment period)
- Normalized Number of Angioedema Attacks Per Month Requiring Acute Treatment in a Treatment Period(From start of treatment up to 12 weeks during each intervention period)
- Plasma Concentration of C1 Esterase Inhibitor (C1 INH) Antigen(Pre-dose and 1 hour (h) post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention period)
- C1 Esterase Inhibitor (C1 INH) Functional Activity in Plasma(Pre-dose and 1 h post-dose at Week 1 (Dose 1) and Week 6 (Dose 12); Pre-dose, 1, 2, 4 and 8 h post-dose at Week 12 (Dose 24) of each intervention period)
- Number of Participants With C1 Esterase Inhibitor (C1 INH) Antibodies in Plasma(Pre-dose, 1 week post treatment (Week 13, Week 25) and 1 month post treatment follow-up (Week 28))