Inter Individual Variability in Initiation Pathway Activation and Regulation and Phenotypic Heterogeneity in Patients With Haemophilia A and B (InPath)
2019年3月25日 更新者：Pratima Chowdary、Royal Free Hospital NHS Foundation Trust
Severe haemophilia A and B (SHA, SHB) are X - linked inherited bleeding disorders, characterised by factor VIII and IX levels of <1 IU/dL respectively.
The mainstay of treatment in SHA and SHB is replacement therapy with intravenous infusions of factor VIII and IX.
However, there is significant variability in the bleeding phenotype within severe haemophiliacs with some presenting with minimal bleeding episodes even on less intensive treatment regimens.
A significant contributor to inter-individual variability in the bleeding phenotype is the coagulation phenotype, but there are no established assays in routine clinical practice that can be used to quantify this.
This study aims to study novel assays and characterise the observed phenotypic heterogeneity.
研究概览
研究类型
观察性的
注册 (预期的)
250
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习地点
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London、英国
- 招聘中
- Royal Free Hospital
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首席研究员：
- Pratima Chowdary
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接触：
- Thomas Roberts
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参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
18年 及以上 (成人、年长者)
接受健康志愿者
是的
有资格学习的性别
男性
取样方法
非概率样本
研究人群
The study population will include patients severe or moderate haemophilia A or B who are currently attending study sites for routine follow up visits.
In addition, 10 healthy volunteers will also be recruited to act as controls
描述
Patients
Inclusion Criteria:
- Patients with haemophilia A or B (baseline FVIII/FIX level <30%)
- Age ≥ 18 years
- Written informed consent in accordance with local and institutional guidelines.
Exclusion Criteria:
1. Patients currently enrolled into a clinical trial of investigational medicinal product for haemophilia.
Healthy Volunteers
Inclusion Criteria:
- Currently not receiving any antiplatelet or anticoagulant therapy or other drugs that can affect the coagulation system.
- Age ≥ 18 years
- Written informed consent in accordance with local and institutional guidelines.
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
队列和干预
团体/队列 |
干预/治疗 |
|---|---|
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Haemophilia patients
Persons with haemophilia A or B - 240 to be recruited
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Thrombophilia screen (including antithrombin activity (AT:Ac), protein S antigen (PS:free), protein C activity (PC:Ac) , genetic analysis for FV Leiden and Prothrombin 3'UTR mutations and screening for lupus anticoagulant.
Evaluation of inter-individual variability in regulation of TF.VIIa.Xa.TFPI complex (tissue factor, activated Factor VII, activated factor X, tissue factor pathway inhibitor)
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Healthy volunteers
Healthy volunteers - 10 to be recruited
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Thrombophilia screen (including antithrombin activity (AT:Ac), protein S antigen (PS:free), protein C activity (PC:Ac) , genetic analysis for FV Leiden and Prothrombin 3'UTR mutations and screening for lupus anticoagulant.
Evaluation of inter-individual variability in regulation of TF.VIIa.Xa.TFPI complex (tissue factor, activated Factor VII, activated factor X, tissue factor pathway inhibitor)
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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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Initiation pathway correlation with clinical phenotype
大体时间：Within 18 months of consent
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Correlate lab assays that characterise initiation pathway with clinical phenotype.
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Within 18 months of consent
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次要结果测量
结果测量 |
大体时间 |
|---|---|
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Correlation analysis between FVIII:C/FIX:C levels and whole blood clotting time, thrombin generation in platelet poor plasma.
大体时间：Within 18 months of consent
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Within 18 months of consent
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Evaluation the sensitivity and specificity of global assays for disease severity and clinical phenotype.
大体时间：Within 18 months of consent
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Within 18 months of consent
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Correlation analysis between activation threshold of initiation pathway to thrombin generation and clinical phenotype
大体时间：Within 18 months of consent
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Within 18 months of consent
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
调查人员
- 首席研究员：Pratima Chowdary、Royal Free Hospitals NHS Foundation Trust
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2017年9月19日
初级完成 (预期的)
2019年10月2日
研究完成 (预期的)
2020年10月2日
研究注册日期
首次提交
2017年9月13日
首先提交符合 QC 标准的
2017年9月15日
首次发布 (实际的)
2017年9月19日
研究记录更新
最后更新发布 (实际的)
2019年3月27日
上次提交的符合 QC 标准的更新
2019年3月25日
最后验证
2019年3月1日
更多信息
此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.
Thrombophilia screen的临床试验
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University of WashingtonNational Institute of Mental Health (NIMH); Seattle Children's Hospital完全的