NCT00851721已完成3 期
FEIBA NF: A Prospective, Open-label, Randomized, Parallel Study to Evaluate the Efficacy and Safety of Prophylactic Versus On-Demand Treatment in Subjects With Hemophilia A or B and a High Titer Inhibitor
Baxalta now part of Shire17 个研究点 分布在 10 个国家目标入组 52 人开始时间: 2009年3月31日最近更新:
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 52
- 试验地点
- 17
- 主要终点
- Reduction in Annualized Bleeding Episode Rate (ABR) Among Participants Receiving Prophylactic Treatment as Compared to Those Treated On-demand
研究概览
简要总结
The purpose of the study was to determine the efficacy, safety, and health-related quality of life benefits with FEIBA NF prophylactic treatment as compared with on-demand treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed and dated informed consent form by the participant or the participant's legally authorized representative
- •The participant is ≥ 4 to ≤ 65 years of age
- •The participant has a Karnofsky performance score of ≥ 60
- •Hemophilia A and B of any severity, with documented history of high-titer inhibitor (> 5 Bethesda unit (BU)) for at least 12 months; or, if inhibitor titer is ≤ 5 BU, and the participant is refractory with increased dosing of either factor VIII (FVIII) or factor IX (FIX), as demonstrated from the participant's medical history
- •Currently being treated on an on-demand basis for treatment of bleeding episodes
- •Adequate venous access, with or without central venous device
- •≥ 12 bleeding episodes requiring treatment with by-passing agents in the past 12 months, based on medical history
- •Competent in-home treatment and infusion therapy
- •Currently using bypassing agents (activated prothrombin complex concentrate (APCC) or recombinant activated factor VII (rFVIIa)) for treatment of bleeding episodes
- •HCV-, either by antibody testing or polymerase chain reaction (PCR); or HCV+ with stable hepatic disease
- •HIV-, or HIV+ with stable disease and CD4 count > 200 cells/mm3 at screening
- •Female participant of childbearing potential, presents with a negative serum pregnancy test, and agrees to employ adequate birth control measures for the duration of the study
排除标准
- •Currently receiving immune tolerance induction (ITI)
- •Currently on regular prophylactic therapy to prevent bleeding episodes
- •Clinically symptomatic liver disease (e.g. diagnosis of cirrhosis [confirmed by liver biopsy], portal vein hypertension, ascites, prothrombin time (PT) 5 seconds above upper limit of normal)
- •Platelet count < 100,000/ml
- •Planned elective surgery during participation in this study
- •Participant is currently participating in another clinical study and has received an investigational product or device within 30 days prior to study entry
- •Planned use of pegylated or non-pegylated alpha-interferon with or without ribavirin for HCV infected participants or planned use of a protease inhibitor for HIV infected participants. Participants currently taking any of these medications for a 30-day course are eligible.
- •Clinically significant increase in D-dimer levels from historical baseline and/or associated with chronic liver disease or clinically evident thromboembolic event
- •Known hypersensitivity to anti-inhibitor coagulant complexes (AICCs)
- •Currently treated with a systemic immunomodulating drug
- •Prior history of thromboembolic event: acute myocardial infarction, deep vein thrombosis, or pulmonary embolism
- •Diagnosis of advanced atherosclerosis, malignancy and/or other diseases that may increase the participant's risk of thromboembolic complications
- •Clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance
研究组 & 干预措施
Prophylaxis arm
Experimental
干预措施: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) (Biological)
On-demand arm
Active Comparator
干预措施: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) (Biological)
结局指标
主要结局
Reduction in Annualized Bleeding Episode Rate (ABR) Among Participants Receiving Prophylactic Treatment as Compared to Those Treated On-demand
时间窗: 12 months ± 14 days
Participants were Randomized to Receive 1 of the 2 Following Treatment Regimens: 1.On-Demand: FEIBA NF dose \& dosing interval as prescribed by treating physician 2.Prophylaxis: 85 ± 15 U/kg of FEIBA NF every other day during 12-month prophylactic period Annualized rate of bleeding episodes was calculated as: (Number of bleeding episodes/observed treatment period in days) \* 365.25
次要结局
- Number of New Target Joints(12 months ± 14 days)
- Total Weight Adjusted Dose to Control a Bleeding Episode(12 months ± 14 days)
- Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old)(Throughout the study period, 12 months ± 14 days)
- Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type(12 months ± 14 days)
- Assessment of Clinical Symptoms - Range of Motion (ROM)(12 months ± 14 days)
- Abnormal D-Dimer Assay Results(Screening visit, Month 3, Month 6, Month 9, and Termination visit)
- Abnormal Fibrin Degradation Products (FDP) Assay Results(Screening visit, Month 3, Month 6, Month 9, and Termination visit)
- Number of Related Thromboembolic Adverse Events (AEs)(12 months ± 14 days)
- Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels(12 months ± 14 days)
- The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding(12 months ± 14 days)
- Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab)(12 months ± 14 days)
- Rate of Related Adverse Events (AEs) During or Within 1 Hour of Infusion Per Year(12 months ± 14 days)
- Annualized Bleeding Rate for New Target Joints(12 months ± 14 days)
- Abnormal Fibrinogen Assay Results(Screening visit, Month 3, Month 6, Month 9, and Termination visit)
- Abnormal Prothrombin Fragment F 1.2 Assay Results(Screening visit, Month 3, Month 6, Month 9, and Termination visit)
- Abnormal Thrombin-Antithrombin III (TAT) Assay Results(Screening visit, Month 3, Month 6, Month 9, and Termination visit)
- Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV](12 months ± 14 days)
- Rate of Related Adverse Events (AEs) Per Year(12 months ± 14 days)
- Pharmacoeconomics: Annual Number of Emergency Room Visits(12 months ± 14 days)
- Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old(Baseline, 6 months and 12 months ± 14 days)
- Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type(12 months ± 14 days)
- Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens: New Target Joints(12 months ± 14 days)
- Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)(12 months ± 14 days)
- Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours(24 ± 1 h post-infusion)
- Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results(Screening visit, Month 3, Month 6, Month 9, and Termination visit)
- Abnormal Prothrombin Time Assay Results(Screening visit, Month 3, Month 6, Month 9, and Termination visit)
- Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours(6 h ± 30 min post-infusion)
- Pharmacoeconomics: Annual Number of Hospitalizations for Indwelling Line(12 months ± 14 days)
- Pharmacoeconomics: Annual Total Number of Days Lost (Work or School)(12 months ± 14 days)
- Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation(12 months ± 14 days)
- Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C(12 months ± 14 days)
- Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV](12 months ± 14 days)
- Pharmacoeconomics: Annual Number of Hospitalizations for Bleeding(12 months ± 14 days)
- Pharmacoeconomics: Annual Total Length of Hospitalization for Indwelling Line(12 months ± 14 days)
- Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation(12 months ± 14 days)
- Pharmacoeconomics: Annual Days Lost Due to Bleeding (Work or School)(12 months ± 14 days)
- Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores(12 months ± 14 days)
- Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old(12 months ± 14 days)
- Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels(12 months ± 14 days)
- Pharmacoeconomics: Annual Number of Physician's Office Visits(12 months ± 14 days)
- Pharmacoeconomics: Annual Total Length of Hospitalization for Bleeding(12 months ± 14 days)
- Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old(Baseline, 6 months and 12 months ± 14 days)
研究者
研究点 (17)
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