Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
A Randomized, Placebo-controlled, Double Blind Phase II/III Study of the Safety and Efficacy of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
Funding Source - FDA OOPD
研究概览
详细说明
研究类型
注册 (实际的)
阶段
- 阶段2
- 第三阶段
联系人和位置
学习地点
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Leiden、荷兰、2300 AL
- For information on sites please contact Pharming Medical Affairs Department
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Main Inclusion Criteria:
- Clear clinical and laboratory diagnosis of HAE
- Plasma level of functional C1INH of less than 50% of normal
- Acute abdominal, urogenital, peripheral, and/or oro-facial/pharyngeal/laryngeal HAE attack
Main Exclusion Criteria:
- Acquired angioedema
- Pregnancy or breastfeeding
- Treatment with any investigational drug within prior 30 days
- Body weight >120 kg
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:随机化
- 介入模型:并行分配
- 屏蔽:四人间
武器和干预
参与者组/臂 |
干预/治疗 |
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安慰剂比较:盐水
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生理盐水
其他名称:
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实验性的:100 IU/kg rhC1INH
100 IU/kg Recombinant human C1 inhibitor
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IV
其他名称:
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实验性的:50 IU/kg rhC1INH
50 IU/kg Recombinant human C1 inhibitor
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IV
其他名称:
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
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Time to Beginning of Relief of Symptoms
大体时间:up to 48 hours after study drug administration
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The time to beginning of relief of symptoms at the location that showed the first visual analogue scale ("VAS") score decrease of at least 20 mm from baseline score with persistence to the next timepoint, assessment timepoints were taken on pre-scheduled time-points after study drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours.
Time to beginning of relief has been calculated as median time, by using the exact timepoints on which each assessment was performed.
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up to 48 hours after study drug administration
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
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Time to Minimal Symptoms
大体时间:up to 48 hours after study drug administration
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The time to minimal symptoms was the time to minimal symptoms for an attack, assessed using the Visual Analogue Scale ("VAS") score.
Symptoms were said to be minimal when the "VAS" score at all locations was below 20 mm.
Assessment timepoints were: baseline, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours.
Time to minimal symtoms has been calculated by using the exact timepoints on which each assessment was performed.
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up to 48 hours after study drug administration
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合作者和调查者
调查人员
- 研究主任:Anurag Relan, MD、Pharming Group N.V.
出版物和有用的链接
一般刊物
- Zuraw B, Cicardi M, Levy RJ, Nuijens JH, Relan A, Visscher S, Haase G, Kaufman L, Hack CE. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010 Oct;126(4):821-827.e14. doi: 10.1016/j.jaci.2010.07.021.
- Bernstein JA, Relan A, Harper JR, Riedl M. Sustained response of recombinant human C1 esterase inhibitor for acute treatment of hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2017 Apr;118(4):452-455. doi: 10.1016/j.anai.2017.01.029. Epub 2017 Mar 9.
- Riedl MA, Levy RJ, Suez D, Lockey RF, Baker JW, Relan A, Zuraw BL. Efficacy and safety of recombinant C1 inhibitor for the treatment of hereditary angioedema attacks: a North American open-label study. Ann Allergy Asthma Immunol. 2013 Apr;110(4):295-9. doi: 10.1016/j.anai.2013.02.007. Epub 2013 Mar 6.
研究记录日期
研究主要日期
学习开始
初级完成 (实际的)
研究完成 (实际的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计)
研究记录更新
最后更新发布 (估计)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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Mila (bMotion Technologies)完全的
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Universidad Autonoma de MadridCentro Universitario La Salle完全的