招募患者:未接受过治疗的儿童血友病A患者中应用turoctocog alfa预防和治疗出血的安全性和有效性临床试验

2015-09-09 MedSci MedSci原创

1.  试验药物简介 Turoctocog alpha(商品名NovoEight)是第三代重组凝血因子VIII,用于A型血友病患者出血事件的预防和治疗。2013年10月FDA批准NovoEight用于A型血友病成人和儿童患者:(1)出血事件的预防和治疗;(2)围手术期管理;(3)常规预防措施,以防止或减少出血发作的频率。   2.  试验目的 评估turoct

1.  试验药物简介
Turoctocog alpha(商品名NovoEight)是第三代重组凝血因子VIII,用于A型血友病患者出血事件的预防和治疗。2013年10月FDA批准NovoEight用于A型血友病成人和儿童患者:(1)出血事件的预防和治疗;(2)围手术期管理;(3)常规预防措施,以防止或减少出血发作的频率。
 
2.  试验目的
评估turoctocog alfa对于患血友病A的儿童中之前未治疗过的患者的安全性。
 
3.  试验设计
试验分类:   安全性和有效性
试验分期:  III期
设计类型:   单臂试验
随机化: 非随机化
盲法:   开放
试验范围:   国际多中心试验
试验人数:   中国7人
 
4.  入选标准(必须全部符合)
1 在进行任何试验相关操作之前,必须签署知情同意书(试验相关操作指任何在患者的常规治疗期间不会进行的操作)
2 男性,诊断为遗传性重型血友病A患者(FVIII水平小于等于1%)
3 年龄<6岁
4 之前没有使用过纯化的凝血因子FVIII产品(以下暴露可以接受:使用血液成分制品,例如冷沉淀物,新鲜的冰冻血浆小于等于5个暴露日)包括已经上市的NovoEight/Novoeight
 
5.  排除标准(必须全部不符合)
1 已知或可疑对仓鼠蛋白过敏或不能耐受试验药物或相关产品
2 之前参加过本试验,定义为在应用试验药物后退出
3 患有血友病A外的其它遗传性或获得性凝血功能异常
4 任何FVIII抑制物史
5 在试验过程中正在应用或计划应用免疫调节剂(例如静脉滴注免疫球蛋白(IVIG)、规律的全身性皮质类固醇治疗)

6. 医院和研究者信息

序号

机构名称

主要研究者

国家

省(州)

城市

1

北京儿童医院

吴润晖

中国

北京

北京

2

中国医学科学院血液病医院

杨仁池

中国

天津

天津

3

南方医科大学南方医院

孙竟

中国

广东

广州

4

中国医学科学院北京协和医院

赵永强

中国

北京

北京

5

浙江大学医学院附属儿童医院

徐卫群

中国

浙江

杭州

6

重庆医科大学附属儿童医院

苏庸春

中国

重庆

重庆

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  1. [GetPortalCommentsPageByObjectIdResponse(id=1862751, encodeId=4bad1862e5175, content=<a href='/topic/show?id=0b69542242' target=_blank style='color:#2F92EE;'>#CTO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=37, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=5422, encryptionId=0b69542242, topicName=CTO)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c66088, createdName=gao_jian4217, createdTime=Sun Sep 20 13:23:00 CST 2015, time=2015-09-20, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1941183, encodeId=1d5b194118366, content=<a href='/topic/show?id=3a862292cd' target=_blank style='color:#2F92EE;'>#ALF#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=51, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=2292, encryptionId=3a862292cd, topicName=ALF)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=84fb152, createdName=xiongliangxl, createdTime=Wed Jan 27 22:23:00 CST 2016, time=2016-01-27, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1976889, encodeId=216d19e6889f2, content=<a href='/topic/show?id=b9f11560065' target=_blank style='color:#2F92EE;'>#ROC#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=40, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=15600, encryptionId=b9f11560065, topicName=ROC)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=d590125, createdName=linlin2312, createdTime=Wed May 11 06:23:00 CST 2016, time=2016-05-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1858309, encodeId=7f141858309c1, content=<a href='/topic/show?id=21311323521' target=_blank style='color:#2F92EE;'>#OCT#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=38, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=13235, encryptionId=21311323521, topicName=OCT)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=19c567, createdName=habb, createdTime=Tue Nov 17 07:23:00 CST 2015, time=2015-11-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2066610, encodeId=c8532066610d1, content=<a href='/topic/show?id=1d3d45e090e' target=_blank style='color:#2F92EE;'>#安全性和有效性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=33, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=45709, encryptionId=1d3d45e090e, topicName=安全性和有效性)], attachment=null, authenticateStatus=null, createdAvatar=https://thirdqq.qlogo.cn/qqapp/1103841572/E33DC517C2EC449E463E3D01D81745BA/100, createdBy=75e12500157, createdName=ms1756883563247400, createdTime=Thu Dec 17 18:23:00 CST 2015, time=2015-12-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1548489, encodeId=49d815484891c, content=<a href='/topic/show?id=6a4756229d9' target=_blank style='color:#2F92EE;'>#招募#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=33, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=56229, encryptionId=6a4756229d9, topicName=招募)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=038c14086626, createdName=ms1260584454294838, createdTime=Fri Sep 11 13:23:00 CST 2015, time=2015-09-11, status=1, ipAttribution=)]
    2015-09-20 gao_jian4217
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    2016-01-27 xiongliangxl
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    2016-05-11 linlin2312
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    2015-11-17 habb
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