NCT00816660已完成1 期
Recombinant Von Willebrand Factor / Recombinant Factor VIII Complex (rVWF:rFVIII): A Phase 1 Study Evaluating the Pharmacokinetics (PK), Safety, and Tolerability in Type 3 Von Willebrand Disease (VWD)
Baxalta now part of Shire50 个研究点 分布在 6 个国家目标入组 32 人开始时间: 2008年12月1日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 32
- 试验地点
- 50
- 主要终点
- To demonstrate the immediate tolerability and safety after single-dose injections of rVWF:rFVIII at various doses
研究概览
简要总结
The objectives of this study are to evaluate the immediate tolerability and safety of rVWF:rFVIII in subjects with Type 3 Von Willebrand Disease after administration of various dosages of VWF:RCo.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Other
- 盲法
- Single (Participant)
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject has voluntarily given written informed consent (before conduct of any study-related procedures)
- •The subject has hereditary type 3 VWD (<= 3 IU/dL VWF:Ag)or severe type 1 or type 2A VWD (VWF:RCo <= 10% and FVIII:C <20%)
- •The subject has a medical history of at least 25 exposure days to VWF/FVIII coagulation factor concentrates
- •The subject has a Karnofsky score >= 70%
- •The subject is between 18 to 60 years of age (on the day of signing the informed consent)
- •NOT APPLICABLE IN ITALY: Female subjects of child-bearing potential must have a negative pregnancy test and agree to practice contraception using a method of proven reliability from the day of screening until the study completion visit
- •APPLICABLE ONLY IN ITALY: Female subjects of child-bearing potential must have a negative pregnancy test and agree to practice non-hormonal-based contraception using a method of proven reliability (IUD acceptable) from the day of screening until 96 hours after the last investigational drug infusion
- •NOT APPLICABLE IN ITALY: The subject must agree not to be on any therapy (hormone-based contraception acceptable) interfering with coagulation factor pharmacokinetics until 96 hours after the last investigational drug infusion
- •APPLICABLE ONLY IN ITALY: The subject must agree not to be on any therapy interfering with coagulation factor pharmacokinetics until 96 hours after the last investigational drug infusion
排除标准
- •The subject has been diagnosed with a hereditary or acquired coagulation disorder other than VWD (including qualitative and quantitative platelet disorders and/or an international normalized ratio (INR) > 1.4)
- •The subject has been diagnosed with an ADAMTS13 deficiency with less than 10% ADAMTS13 activity
- •The subject has a history or presence of VWF inhibitor
- •The subject has a history or presence of FVIII inhibitor with a titer >= 0.4 BU (by Nijmegen assay) or >= 0.6 BU (by Bethesda assay)
- •The subject has a known hypersensitivity to mouse or hamster proteins
- •The subject has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, food allergies or animal allergies
- •The subject has a medical history of a thromboembolic event
- •The subject is HIV positive with an absolute CD4 count < 200/mm3
- •The subject has been diagnosed with cardiovascular disease (New York Heart Association (NYHA) classes 1-4)
- •The subject has been diagnosed with insulin-dependent diabetes mellitus
- •The subject has an acute illness (e.g. influenza, flu-like syndrome, allergic rhinitis/conjunctivitis)
- •The subject has been diagnosed with liver disease, as evidenced by, but not limited to, any of the following: serum ALT three times the upper limit of normal, hypoalbuminemia, portal vein hypertension (e.g. presence of otherwise unexplained splenomegaly, history of esophageal varices)
- •The subject has been diagnosed with renal disease, with a serum creatinine level >= 2 mg/dL
- •In the judgment of the investigator, the subject has another clinically significant concomitant disease (e.g. uncontrolled hypertension, diabetes type II) that may pose additional risks for the subject
- •The subject has been treated with an immunomodulatory drug, excluding topical treatment (e.g. ointments, nasal sprays) within 30 days before enrollment
- •The subject has been treated with drugs known to induce thrombotic thrombocytopenic purpura (TTP) (e.g. Adenosine diphosphate (ADP) receptor inhibitors (Clopidogrel, Ticlopidine)) within 60 days before enrollment
- •The subject is receiving or anticipates receiving another investigational and/or interventional drug within 30 days before enrollment
- •The subject is a lactating female
- •The subject has a history of drug or alcohol abuse within the last 5 years
- •The subject has a progressive fatal disease and/or life expectancy of less than 3 months
- •The subject is identified by the investigator as being unable or unwilling to cooperate with study procedures
- •The subject suffers from a mental condition rendering him/her unable to understand the nature, scope and possible consequences of the study and/or evidence of an uncooperative attitude
- •Subject is in prison or compulsory detention by regulatory and/or juridical order
结局指标
主要结局
To demonstrate the immediate tolerability and safety after single-dose injections of rVWF:rFVIII at various doses
时间窗: Up to 30 days after the last investigational product infusion
次要结局
未报告次要终点
研究者
研究点 (50)
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