NCT01434511终止3 期
Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Patients With Congenital Hemophilia A With Factor VIII Inhibitors
Baxalta now part of Shire3 个研究点 分布在 3 个国家目标入组 1 人开始时间: 2011年10月3日最近更新:
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- 入组人数
- 1
- 试验地点
- 3
- 主要终点
- Proportion of Serious Bleeding Episodes Responsive to OBI-1
研究概览
简要总结
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with congenital 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
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent/assent from participant and/or participant's parent or legal representative.
- •Participants with congenital hemophilia A with human factor VIII inhibitor ≤30 BU assessed within 90 days prior to study entry.
- •Has previously or is currently demonstrating suboptimal hemostatic response to bypassing agents for treatment of bleeding episodes; suboptimal response is determined by the investigator , but minimally includes no or minimal evidence of response after at least two doses of bypassing agents, either for the current or a historic bleeding episode.
- •Has an anti-OBI-1 titer ≤ 10 BU
- •Has any serious or life-threatening bleeding episode; or requires a surgical procedure that could lead to a serious bleeding episode if not well controlled.
- •Is willing and able to follow all instructions and attend all study visits.
- •Has no other significant hemostatic abnormality and:
- •Platelets ≥100,000/mm-cubed
- •Prothrombin time < 15 seconds
- •INR < 1.3
- •Participants taking anti-thrombotics (such as clopidogrel, heparin or heparin analogue) may be included provided three half-lives of the agent have elapsed since the last dose of the agent.
排除标准
- •Hemodynamically unstable after blood transfusion, fluid resuscitation and pharmacologic or volume replacement pressor therapy. This hemodynamic instability is characterized by symptomatic hypotension resulting in vital organ dysfunction, such as cardiac ischemia, oliguria (urine volume <0.5 mL/kg in the previous six hours), central nervous system hypoperfusion manifested by mental status change such as confusion (unless head injury or intracranial hemorrhage is present), pulmonary compromise, and/or acidosis (manifested by pH and lactate levels).
- •Bleeding episode assessed likely to resolve on its own if left untreated.
- •Use of hemophilia medication: recombinant factor VIIa within 3 hours prior to OBI-1 administration or activated prothrombin complex concentrate (aPCC) treatment within 6 hours prior to OBI-1 administration
- •Prior history of bleeding disorder other than congenital hemophilia A
- •Known major sensitivity (anaphylactoid reactions) to porcine or hamster products. Examples include therapeutics of porcine origin (e.g. previously marketed porcine factor VIII, Hyate-C®) and recombinant therapeutics prepared from hamster cells (e.g. Humira®, Advate® and Enbrel®).
- •Received any other investigational treatment within 30 days of the first OBI-1 treatment.
- •Anticipated need for treatment or device during the study that may interfere with the evaluation of the safety or efficacy of OBI-1, or whose safety or efficacy may be affected by OBI-
- •Is planning to father a child during the study
- •Has abnormal baseline findings, any other medical condition(s) or laboratory findings that, in the opinion of the investigator, might jeopardize the participant's safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.
- •Inability or unwillingness to comply with the study design, protocol requirements, or the follow-up procedures.
研究组 & 干预措施
OBI-1
Experimental
干预措施: OBI-1 (Biological)
结局指标
主要结局
Proportion of Serious Bleeding Episodes Responsive to OBI-1
时间窗: 24 hours after initiation of treatment
This study was terminated early and only enrolled one participant. Due to concerns that the participant would be at risk of being re-identified, the study results are not posted. The decision to terminate this study was not related to any safety and/or efficacy concern of OBI-1 in the indication described within the OBI-1-302 study (Congenital Hemophilia A).
次要结局
- Total Dose of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes.(Through 90 days ± 7days following final OBI-1 dose)
- Overall Proportion of Serious Bleeding Episodes Successfully Controlled With OBI-1 Therapy, as Assessed by the Investigator.(Through 90 days ± 7days following final OBI-1 dose)
- Correlation Between Response to OBI-1 Therapy at Specified Time Points and Eventual Control of Serious Bleeding Episodes.(Through 90 days ± 7days following final OBI-1 dose)
- Correlation Between the Pre-infusion Anti-OBI-1 Antibody Titers, the Total Dose of OBI-1, the Outcome at 24 Hours and the Eventual Control of the Bleeding Episode.(Frame: Through 90 days ± 7days following final OBI-1 dose)
- Correlation Between the Pre-infusion Anti-OBI-1 Antibody Titers and the Recovery of OBI-1.(Through 90 days ± 7days following final OBI-1 dose)
- Recovery and Elimination Rate Parameters of OBI-1 in Subjects With Inhibitors Treated With OBI-1 Therapy.(Through 90 days ± 7days following final OBI-1 dose)
- Frequency of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes.(Through 90 days ± 7days following final OBI-1 dose)
- Total Number of Infusions of OBI-1 Required to Successfully Control Qualifying Bleeding Episodes.(Through 90 days ± 7days following final OBI-1 dose)
- Anti-OBI-1 Antibody Titer.(Through 90 days ± 7days following final OBI-1 dose)
- Proportion of Bleeding Episodes Responsive to OBI-1 Therapy at Designated Assessment Time Points After the Initiation of Therapy, as Assessed by the Investigator(Through 90 days ± 7days following final OBI-1 dose)
- Efficacy Assessment of OBI-1 in Participants With Anti-human Factor VIII Titers >30 Bethesda Units (BU)(Through 90 days ± 7days following final OBI-1 dose)
- Anti-human Factor VIII Antibody Titer.(Through 90 days ± 7days following final OBI-1 dose)
- Anti-host Cell Protein Baby Hamster Kidney (BHK) Antibody Titer.(Through 90 days ± 7days following final OBI-1 dose)
研究者
研究点 (3)
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