Natural History Study of Factor IX Treatment and Complications
试验速览
- 阶段
- 不适用
- 入组人数
- 550
- 试验地点
- 1
- 主要终点
- Inhibitory antibodies
研究概览
简要总结
This study will examine two groups of subjects with factor IX (FIX) deficiency: 1) those with a current or history of inhibitors to FIX, and; 2) groups of two or more affected brothers, with or without inhibitors. The overall goal is to characterize the study groups in terms of their medical history, their patterns of bleeding, their care, quality of life, and complications including the development of joint disease, inhibitory antibodies to FIX, use of immune tolerance induction (ITI) and outcome.
详细描述
Hemophilia B, FIX deficiency, is the second most common type of hemophilia, occurring in about one in 25,000 male births. This disease is in some ways more complex than hemophilia A, and is less well understood. Differences include a lower incidence and a greater risk of side effects to treatment, for example, allergic reactions and kidney disease. This study will examine two groups of subjects with FIX deficiency - those with a current or history of inhibitors to FIX, and groups of two or more affected brothers, with or without inhibitors. The overall goal is to characterize the study group in terms of their medical history, their patterns of bleeding, their care, quality of life, and complications including the development of inhibitory antibodies to FIX, allergies, kidney, and joint disease.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •A consent approved by the appropriate Institutional Review Board (IRB)/Independent Ethics Committee (IEC) has been obtained from the subject or his legally acceptable representative
- •Subject has FIX deficiency AND
- •Is part of an affected brother pair/group that will also enroll; AND/OR
- •Has a current or history of inhibitor, defined as >0.6 Bethesda units (BU)
排除标准
- •Subject has another congenital bleeding disorder
- •Subject is a carrier of hemophilia B with factor level >0.40 IU/mL
结局指标
主要结局
Inhibitory antibodies
时间窗: Baseline
Current or history of inhibitors
Hemophilia treatment adherence
时间窗: Baseline
Validated Hemophilia Regimen Treatment Adherence Scale--Prophylaxis (VERITAS-Pro), Validated Hemophilia Regimen Treatment Adherence Scale - PRN (VERITAS-PRN)
Annualized bleeding rate
时间窗: 6 months
Overall and by bleeding site
Joint assessment
时间窗: Baseline
Range of motion
Renal disorders
时间窗: 6 months
Reported subject and family history of renal disease
Non-inhibitory antibodies
时间窗: Baseline
Measured at central laboratory
Health related quality of life
时间窗: Baseline
European Quality of Life - 5 Dimensions (EQ5D)
次要结局
- number of days missed from school or work(6 months)
- Factor IX usage(6 months)
- Number of hospitalizations(6 months)
- Number of surgical procedures(6 months)
研究者
Sharyne M. Donfield, Ph.D.
Principal Investigator, Data Coordinating Center, Rho, Inc.
Skane University Hospital