A Phase 2, Randomized, Double-Blind, Multicenter, Dose-Ranging, Crossover Study to Evaluate the Safety and Efficacy of Subcutaneous Administration of CINRYZE® (C1 Esterase Inhibitor [Human]) With Recombinant Human Hyaluronidase (rHuPH20) for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 47
- 试验地点
- 23
- 主要终点
- Normalized Number of Angioedema Attacks During the Treatment Period
研究概览
简要总结
The primary objectives of the study are to evaluate the safety, tolerability, and efficacy of two doses of CINRYZE with recombinant human hyaluronidase (rHuPH20) administered by subcutaneous (SC) injection to prevent angioedema attacks.
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
- 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
- •Be ≥12 years of age.
- •Have a confirmed diagnosis of Hereditary Angioedema.
排除标准
- •Receipt of any C1 inhibitor (C1 INH) therapy or any blood products for treatment or prevention of an angioedema attack within 7 days before the first dose of study drug.
- •Be receiving prophylactic intravenous CINRYZE that exceeds 1000 units every 3 or 4 days (maximum weekly dose 2000 units).
- •Have received any androgen therapy (e.g., danazol, oxandrolone, stanozolol, testosterone) within 7 days prior to the first dose of study drug.
- •If female, have started taking or changed the dose of any hormonal contraceptive regimen or hormone replacement therapy (i.e., estrogen/progestin containing products) within 3 months prior to the first dose of study drug.
- •History of allergic reaction to C1 INH products, including CINRYZE or other blood products.
- •History of abnormal blood clotting.
- •Have a known allergy to hyaluronidase or any other ingredient in the study formulation.
结局指标
主要结局
Normalized Number of Angioedema Attacks During the Treatment Period
时间窗: From Visit 1 (Week 1) up to Visit 16 (Week 8) 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. Participants who were dosed but did not have any attacks in the period were assigned a value of zero. 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 During the Treatment Period(From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period)
- Cumulative Attack-severity During the Treatment Period(From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period)
- Cumulative Symptomatic Days During the Treatment Period(From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period)
- Number of Angioedema Attacks Requiring Acute Treatment During the Treatment Period(From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period)