NCT01376700终止3 期
A Phase 3b Clinical Study to Assess Whether Regular Administration of ADVATE in the Absence of Immunological Danger Signals Reduces the Incidence Rate of Inhibitors in Previously Untreated Patients With Hemophilia A
Baxalta now part of Shire19 个研究点 分布在 12 个国家目标入组 22 人开始时间: 2011年8月26日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 22
- 试验地点
- 19
- 主要终点
- Number of Participants With Severe and Moderately Severe Hemophilia A (FVIII ≤ 2%) With Factor VIII (FVIII) Inhibitor Formation Within the First 50 Exposure Days to ADVATE
研究概览
简要总结
The purpose of the study was to assess if a once-weekly prophylactic regimen of 25 IU/kg ADVATE started at or before 1 year of age and before the onset of a severe bleeding phenotype (ie, joint bleeding), together with the minimization of immunological danger signals, can reduce the incidence rate of inhibitor formation in PUPs with severe and moderately severe hemophilia A.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- — 至 1 Year（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participants with severe and moderately severe hemophilia A (FVIII ≤ 2%)
- •Participants < 1 year of age
- •Participants must have ≤ 3 exposure days (EDs) to any FVIII concentrate or FVIII-containing product used for treatment of minor bleeds (bleeds requiring no more than 2 infusions per event), or for preventative or precautionary infusions following possible injury
- •Participants with prior circumcision are allowed to enroll only if bleeding issues related to circumcision were the cause for the original diagnosis of hemophilia A and no more than 2 EDs of FVIII treatment were required
- •Adequate venous access (without need for central venous access device (CVAD)-placement) as determined by the physician
- •Written informed consent from legally authorized representative(s)
排除标准
- •Life-threatening conditions (intracranial hemorrhage, severe trauma) or requirement for surgery at the time of enrollment
- •Evidence of inhibitor ≥ 0.6 Bethesda Unit (BU) in Nijmegen-modified Bethesda Assay at study start (samples may be retested using lupus-insensitive inhibitor tests to reduce the number of false positive inhibitor test results)
- •Inherited or acquired hemostatic defect other than hemophilia A
- •Any clinically significant, chronic disease other than hemophilia A
- •Known hypersensitivity to ADVATE or any of its constituents
- •Any planned elective surgery that cannot be postponed until after the first 20 EDs
- •Participation in the Hemophilia Inhibitor Previously Untreated Patient Study
- •Application of red blood cell, platelet, or leukocyte concentrates, or plasma
- •Administration of any medication affecting coagulation or platelet function
- •Systemic administration of any immunomodulatory drug (eg, chemotherapy, intravenous glucocorticoids)
- •Participation in another clinical study involving an investigational product (IP) or device within 30 days prior to study enrollment or during the course of this study
结局指标
主要结局
Number of Participants With Severe and Moderately Severe Hemophilia A (FVIII ≤ 2%) With Factor VIII (FVIII) Inhibitor Formation Within the First 50 Exposure Days to ADVATE
时间窗: 50 exposure days to ADVATE
Inhibitor testing will be performed in the central laboratory and a non-zero result must be confirmed in the central laboratory as soon as possible, preferably 1 week after inhibitor testing. Confirmed FVIII inhibitor is defined as any FVIII inhibitor assay result equal or greater than 0.6 Bethesda Units (BU)/mL confirmed by the central laboratory on 2 consecutive samples, i.e. at least 2 positive inhibitor results (including the first positive inhibitor test, in accordance with the study protocol) assessed as either: - i. High FVIII inhibitor titer (\> 5 BU/mL) or - ii. Low FVIII inhibitor titer (≥0.6 - ≤5.0 BU/mL).
次要结局
- Correlation of Known Risk Factors to Factor VIII (FVIII) Inhibitor Formation(50 exposure days to ADVATE)
- Number of Exposure Days of Treatment With Advate Prior to First Positive Factor VIII (FVIII) Confirmed Inhibitor Assessment(50 exposure days to ADVATE)
- Number of Participants With Severe Hemophilia A (FVIII ≤ 1%) With Factor VIII (FVIII) Inhibitor Formation Within the First 50 Exposure Days to ADVATE(50 exposure days to ADVATE)
- Number, Type, and Severity of All Bleeds Experienced When Different Prophylactic Dosing Frequencies Are Used (Once or Twice Per Week and Unknown Frequency)(50 exposure days to ADVATE)
- Number of Participants With Low-titer, High-titer, Transient, and All Factor VIII (FVIII) Inhibitors(50 exposure days to ADVATE)
- Total Factor VIII (FVIII) Consumption by Participant(50 exposure days to ADVATE)
- FVIII-Specific Antibody Isotype for All Participants at Study Entry and Every 10 Exposure Days (EDs)(50 exposure days to ADVATE)
- Number of Serious Adverse Events (SAEs) and Non-serious Adverse Events (Non-SAEs) at Least Possibly Related to ADVATE(50 exposure days to ADVATE)
- Number and Type of Surgeries(50 exposure days to ADVATE)
研究者
研究点 (19)
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