Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A (myPKFiT)
2021年4月12日 更新者:Lund University
Optimizing the Use of Prophylaxis in Patients With Severe Haemophilia A Using PK Measurement (myPKFiT)
MyPKFiT is a web-based application recently developed by Baxalta for the use in patients treated with Advate.
MyPkFit has its basis in Bayesian forecasting, which allows estimation of individual PK parameters by a sparse sampling schedule, where only 2-3 samples are taken between 4 and 48 hours post infusion.
With myPKFiT, it will, therefore, be possible to define an individual PK curve for each patient based on just a few sampling points and hence, taking the bleeding phenotype and the life style into account, potentially adjust the prophylactic treatment accordingly to optimize cost-effectiveness.
研究概览
详细说明
As detailed in brief summary patients will, after accurate information verbally and written according to ethics approval, report bleeding events during previous 6 months.
Blood samples are taken, FVIII analyzed (Advate patients) and the individual PK curve determined using MyPKFit.
The curve will be discussed together with the patient and levels related to bleed events determined.
If needed dose adjustment is done based on the curve.
After a further 6 month follow-up bleeds will again be evaluated as well as factor consumption.
In this way a better personalized treatment based on PK and clinical phenotype will hopefully be achieved.
研究类型
介入性
注册 (实际的)
14
阶段
- 第四阶段
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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Malmo、瑞典、SE-20502
- Malmö Centre for Thrombosis and Haemostasis
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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
1年 至 80年 (孩子、成人、年长者)
接受健康志愿者
不
有资格学习的性别
男性
描述
Inclusion Criteria:
- Children and adults with severe hemophilia A (FVIII:C <1 %), being treated with Advate for more than 50 exposure days (EDs
Exclusion Criteria:
- Current evidence of inhibitor as measured by the Nijmegen-modified Bethesda assay
- Use of another investigational FVIII product in the previous month
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
- 主要用途:其他
- 分配:不适用
- 介入模型:单组作业
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
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其他:Oktokog alpha (Advate)
Personalized treatment according to individual PK using intravenous injection of oktokog alpha with dose and dose interval according to MyPKFIT and phenotypic evaluation.
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adjusting dose and dose interval according to MyPKFIT
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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 |
---|---|---|
PK measurement to individualize replacement therapy
大体时间:3 years
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To evaluate whether PK measurement by myPKFiT and the calculation of an individualized PK profile may allow the treating physician to personalize and optimize the treatment of patients with haemophilia A to minimize the number of bleeds in a cost-effective way without diminishing compliance.
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3 years
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PK measurement to influence FVIII consumption
大体时间:3 years
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2. To calculate whether optimization of treatment by use of MyPKFiT, as described in primary objective 1, result in change of total FVIII consumption and extra doses given before and after visit 1. Measurements are T1/2, area under the curve
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3 years
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Specific pharmacokinetic parameters to analyze
大体时间:3 years
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Biological half-life of infused FVIII product will be measured in hours.
Area under the curve (AUC)will be given in IUxh/dL
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3 years
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
---|---|---|
Signs of hemophilic arthropathy
大体时间:3 years
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1. To identify and characterize and sign of arthropathy by ultrasound sound evaluation of the synovium, cartilage and bone according to the HEAD-US score of ankle, knee and elbows visit 1 and 2 and correlate this to the treatment provided.
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3 years
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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。
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
2016年12月1日
初级完成 (实际的)
2021年4月9日
研究完成 (实际的)
2021年4月9日
研究注册日期
首次提交
2016年12月13日
首先提交符合 QC 标准的
2019年4月11日
首次发布 (实际的)
2019年4月16日
研究记录更新
最后更新发布 (实际的)
2021年4月13日
上次提交的符合 QC 标准的更新
2021年4月12日
最后验证
2021年4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.
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