NCT02895945终止3 期
A Phase 3, Multicenter, Open-label Study of the Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (BAX 802) in Subjects With Congenital Hemophilia A With Factor VIII Inhibitors Undergoing Surgical or Other Invasive Procedures
Baxalta now part of Shire47 个研究点 分布在 12 个国家目标入组 8 人开始时间: 2016年12月22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
- 8
- 试验地点
- 47
- 主要终点
- Percentage of Surgeries With a "Good" or "Excellent" Response as Measured by the Global Hemostatic Efficacy Assessment (GHEA) Score
研究概览
简要总结
The purpose of this study is to evaluate the efficacy and safety of BAX 802 in males with congenital hemophilia A (CHA) with inhibitors who are undergoing major or minor elective surgical, dental, or other invasive procedures.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 75 Years（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Participant requires a major or minor elective surgical, dental or other invasive procedure
- •Participant is male and ≥ 12 to ≤ 75 years old at the time of screening
- •Participant has provided signed informed consent (and assent for adolescent participants, as applicable) in accordance with local regulatory requirements
- •Participant has severe (factor VIII (FVIII) level < 1%) or moderately severe (FVIII level ≤ 2%) congenital hemophilia A (CHA) with inhibitors to human factor VIII (hFVIII) of ≥ 0.6 Bethesda units (BU), as tested at screening at the central laboratory
- •Participant is not currently receiving or has recently received (< 30 days) immune tolerance induction (ITI) therapy
- •Participant has a Karnofsky performance score of ≥ 60 at screening
- •Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3 at screening
- •Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing; or HCV+ with chronic stable hepatitis disease. Positive serologies will be confirmed by PCR testing.
- •Participant is willing and able to comply with the requirements of the protocol.
排除标准
- •The participant requires emergency surgery
- •Severe chronic liver dysfunction or disease (e.g., ≥ 5 × upper limit of normal [ULN] alanine aminotransferase [ALT], as confirmed by central laboratory at screening or a documented prothrombin time/international normalized ratio [PT/INR] > 1.5)
- •Clinically symptomatic renal disease (serum creatinine > 2.0 mg/dL), as confirmed by central laboratory at screening
- •Anti-porcine factor VIII (pFVIII) inhibitor > 10 BU prior to surgery
- •Platelet count < 100,000/μL at screening
- •Participant has another active coagulation disorder, other than hemophilia A, as per the medical history
- •Planned use of α-interferon with or without ribavirin for HCV infected patients or planned use of a protease inhibitor for HIV infected patients. Patients currently taking any of these medications for ≥ 30 days are eligible
- •Known hypersensitivity to recombinant porcine factor VIII (rpFVIII), or hamster or murine proteins
- •Participant has an ongoing or recent (within 3 months of screening) thrombo-embolic disease, fibrinolysis or disseminated intravascular coagulation (DIC)
- •Participant has been exposed to an IP within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an investigational product (IP) or investigational device during the course of this study
- •Participant is unable to tolerate quantity of blood to be drawn for protocol procedures
- •Participant is a family member or employee of the Investigator.
结局指标
主要结局
Percentage of Surgeries With a "Good" or "Excellent" Response as Measured by the Global Hemostatic Efficacy Assessment (GHEA) Score
时间窗: Day 1 up to discharge or Day 14 (whichever was earlier)
GHEA score consisted of 3 individual rating scales: (1) Intra-operative Efficacy Assessment Scale, (2) Post-operative Efficacy Assessment Scale, and (3) Overall Peri-operative Efficacy Assessment Scale. Scales 1 and 2 was performed by the operating surgeon on Day 1, and Scale 3 was performed by the investigator on Day 14. Each rating scale was based on 4 points scale ranging from: 3 (Excellent), 2 (Good), 1 (Fair), and 0 (None). Total score ranged from 0 to 9, where scores evaluated as: excellent (7 to 9), good (5 to 7), fair (3 to 4), and none (0 to 2). The scores of 3 individual ratings scales were added together to form a GHEA score. For a GHEA score of 7 to be rated "excellent" with no individual assessment scores less than (\<) 2 and at least 1 assessment score equal to (=) 3; otherwise a score of 7 was rated "good". Percentage of Surgeries With a "Good" or "Excellent" response as measured by the GHEA score were reported.
次要结局
- Total Weight-adjusted Dose of BAX 802 Per Participant During Pre-operative, Intra-operative and Post-operative Period(Pre-operative: before surgery, Intra-operative: up to completion of surgery (Day 1), Post-operative: from completion of surgical procedure till discharge or 14 days post surgery (whichever was earlier))
- Ratio of Actual Blood Loss and Expected Maximum Blood Loss During Intra-operative, Post-operative and Peri-operative Period(Intra-operative: up to completion of surgery (Day 1), Post-operative: at 24 hours post-surgery, and Peri-operative: at discharge or Day 14 (whichever was earlier))
- Percentage of Major Surgeries With Good or Excellent Hemostatic Score(Day 1 up to discharge or Day 14 (whichever was earlier))
- Number of Participants With De Novo Inhibitors(Baseline up end of study (EOS) (up to 44 months))
- Mean Change From Baseline up to EOS in Inhibitory and Binding Antibodies to pFVIII(Baseline up to EOS (up to 44 months))
- Number of Participants With Clinically Significant Change From Baseline in Binding Antibodies to Baby Hamster Kidney (BHK) Proteins(Baseline up to EOS (up to 44 months))
- Ratio of Actual Blood Loss and Estimated Volume of Expected Average Blood Loss During Intra-operative, Post-operative and Peri-operative Period(Intra-operative: up to completion of surgery (Day 1), Post-operative: at 24 hours post-surgery, and Peri-operative: at discharge or Day 14 (whichever was earlier))
- Number of Participants With Investigational Product (IP) Related Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs(Baseline up to EOS (up to 44 months))
- Actual Blood Loss, Estimated Volume of Expected Average Blood Loss and Expected Maximum Blood Loss During Intra-operative, Post-operative and Peri-operative Period(Intra-operative: up to completion of surgery (Day 1), Post-operative: at 24 hours post-surgery, and Peri-operative: at discharge or Day 14 (whichever was earlier))
- Number of Participants With Anamnestic Reactions(Baseline up to EOS (up to 44 months))
- Number of Participants With Thromboembolic Events(Baseline up to EOS (up to 44 months))
- Average Daily Weight-adjusted Dose of BAX 802 Per Participant During Pre-operative, Intra-operative and Post-operative Period(Pre-operative: before surgery, Intra-operative: up to completion of surgery (Day 1), Post-operative: from completion of surgical procedure till discharge or 14 days post surgery (whichever was earlier))
- Volume of Blood Products Transfused(From initiation of the surgery up to discharge or Day 14 (whichever came earlier))
- Mean Change From Baseline up to EOS in Inhibitory and Binding Antibodies to hFVIII(Baseline up to EOS (up to 44 months))
- Number of Participants With Severe Allergic Reactions(Baseline up to EOS (up to 44 months))
- Number of Participants With Clinically Significant Change From Baseline in Clinical Laboratory Values(Baseline up to EOS (up to 44 months))
- Number of Participants With Clinically Significant Change From Baseline in Vital Sign(Baseline up to EOS (up to 44 months))
研究者
研究点 (47)
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