LEVP2006-1 CHANGE 2 Trial (C1-Inhibitor in Hereditary Angioedema Nanofiltration Generation Evaluating Efficacy): Open-Label Safety/Efficacy Repeat Exposure Study of C1INH-nf (Human) in the Treatment of Acute HAE Attacks
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Shire
- 入组人数
- 113
- 试验地点
- 58
- 主要终点
- Percent of HAE Attacks With Substantial Relief of the Defining Symptom
研究概览
简要总结
The study objective was to evaluate the safety and efficacy of repeat use of C1INH-nf for the treatment of acute HAE attacks.
详细描述
A total of 113 subjects were enrolled in the study. One-hundred-one (101) subjects received C1INH-nf for the treatment of 1 or more HAE attacks and were analyzed for efficacy. The study design also allowed for short-term prophylaxis with C1INH-nf prior to emergency or non-cosmetic surgical or dental procedures, and an additional 12 subjects received C1INH-nf only for this purpose. All 113 subjects were exposed to C1INH-nf and analyzed for safety.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •This study was open to all subjects who:
- •Completed participation in LEVP2005-1/A (NCT00289211) and were not participating in LEVP2005-1/B (NCT01005888), any time after the 3-day telephone follow-up
- •Completed participation in LEVP2005-1/B any time after the final prophylactic therapy in Part B
- •Were enrolled but not randomized in LEVP2005-1/A after Part A was closed
- •Were excluded from LEVP2005-1 for any of the following reasons:
- •Pregnancy or lactation
- •Age less than 6 years
- •Narcotic addiction
- •Presence of anti-C1INH autoantibodies
- •Were not enrolled in LEVP2005-1 after enrollment in LEVP2005-1 was closed, under the following circumstances:
- •Had a diagnosis of HAE: evidence of a low C4 level plus either a low C1INH antigenic level or a low C1INH functional level, or
- •Had a known HAE-causing C1INH mutation, or
- •Had a diagnosis of HAE based on a strong family history of HAE as determined by the principal investigator
排除标准
- •History of allergic reaction to C1INH or other blood products
- •Participated in any other investigational drug study within the past 30 days
- •Received blood or a blood product in the past 60 days other than C1INH-nf
结局指标
主要结局
Percent of HAE Attacks With Substantial Relief of the Defining Symptom
时间窗: Within 4 hours after initial treatment
Subjects were to assess their symptoms every 15 minutes up to 4 hours after the initial dose or until substantial relief of the defining symptom was achieved. The conservative analysis defined substantial relief as 3 consecutive assessments of improvement of the defining symptom; any attack that did not have 3 consecutive documented reports of improvement was considered a treatment failure. In the less conservative analysis, attacks also were considered to have responded if clinical improvement of the defining symptom occurred but data were incomplete due to cessation of symptom assessments.
Number of Hereditary Angioedema (HAE) Attacks Treated With C1INH-nf
时间窗: Duration of the study (2.5 years)
次要结局
- Antigenic C1 Inhibitor (C1INH) Serum Levels(Pre-infusion to 1 hour post-infusion)
- Functional C1INH Serum Levels(Pre-infusion to 1 hour post-infusion)
- Time to Beginning of Substantial Relief of the Defining Symptom(Within 4 hours after initial treatment)
- Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments(Within 4 hours after initial treatment)
- Complement C4 Serum Levels(Pre-infusion to 1 hour post-infusion)