EUCTR2018-003453-16-AT进行中（未招募）1 期
A PHASE 3B, PROSPECTIVE, OPEN-LABEL,UNCONTROLLED, MULTICENTER STUDY ON LONGTERMSAFETY AND EFFICACY OF rVWF IN PEDIATRICAND ADULT SUBJECTS WITH SEVERE VONWILLEBRAND DISEASE (VWD) - rVWF Pediatric and Adult Study
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 71
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Inclusion Criteria:
- •Subjects who have completed Study 071301 or 071102 (or subjects who have completed the surgery in Study 071102 and want to continue to receive OD treatment) and are willing to immediately transition into this study, must
- •meet the following 2 criteria to be eligible for this study:
- •1. If female of childbearing potential, has a negative blood/urine pregnancy test at screening and agrees to employ highly effective birth control measures (including sterilization, implant, intra-uterine device (IUD), correct and consistent use of hormonal contraception, and abstinence) for the duration of the study.
- •2. Subject and/or legally authorized representative is willing and able to comply with the requirements of the protocol.
- •New subjects (Cohort 4) who meet the above 2 and ALL the following additional criteria are eligible for this study:
- •3. Subject has a documented diagnosis of severe VWD (baseline VWF:RCo <20 IU/dL) with a history of requiring substitution therapy with rVWF concentrate required to control bleeding:
- •a. Type 1 (VWF:RCo <20 IU/dL) or,
- •b. Type 2A (as verified by multimer pattern), Type 2B (as diagnosed by genotype), Type 2M or,
- •c. Type 3 (VWF:Ag =3 IU/dL).
- •Diagnosis is confirmed by genetic testing and multimer analysis, documented in patient history or at screening.
- •4. Subject has been receiving OD therapy with VWF products for at least 12 months, and prophylactic treatment is recommended by the investigator.
- •5. Subject has =3 documented spontaneous bleeds (not including menorrhagia) requiring VWF treatment during
- •the past 12 months.
- •6. Subject has available records that reliably evaluate type, frequency, and treatment of bleeding episodes for at
- •least 12 months preceding enrollment; up to 24 months of retrospective data should be collected if available.
- •7. Subject is =12 years old at the time of screening and has a body mass index =15 but <40 kg/m2.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 40
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 22
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •The subject will be excluded from the study if any of the following exclusion criteria are met:
- •1. The subject has been diagnosed with Type 2N VWD, pseudo VWD, or another hereditary or acquired coagulation disorder other than VWD (e.g., qualitative and quantitative platelet disorders or elevated
- •prothrombin time/international normalized ratio >1.4).
- •2. The subject has a history or presence of a VWF inhibitor at screening.
- •3. The subject has a history or presence of a FVIII inhibitor with a titer =0.4 Bethesda units (BU) (by Nijmegen modified Bethesda assay) or =0.6 BU (by Bethesda assay).
- •4. The subject has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins.
- •5. The subject has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies, or animal allergies.
- •6. The subject has a medical history of a thromboembolic event.
- •7. The subject is human immunodeficiency virus (HIV) positive with an absolute Helper T cell (CD4) count 200/mm3.
- •8. The subject has been diagnosed with significant liver disease per investigator’s medical assessment of the subject’s current condition or medical history or as evidenced by, but not limited to any of the following:
- •serum alanine aminotransferase (ALT) greater than 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (e.g., presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child-Pugh class B or C.
- •9. The subject has been diagnosed with renal disease, with a serum creatinine (CR) level =2.5 mg/dL.
- •10. The subject has a platelet count <100,000/mL at screening.(for subjects with type 2B VWD, platelet count(s) at screening will be evaluated taking into consideration historical trends in platelet counts and the
- •Investigator’s medical assessment of the patient’s condition).
- •11. The subject has been treated with an immunomodulatory drug, excluding topical treatment (e.g., ointments, nasal sprays), within 30 days prior to signing the informed consent (or assent, if appropriate).
- •12. The subject is pregnant or lactating at the time of enrollment.
- •13. The subject has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
- •14. The subject has participated in another clinical study involving another investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study
- •involving an IP or investigational device during the course of this study.
- •15. The subject has a progressive fatal disease and/or life expectancy of less than 15 months.
- •16. For new OD subjects, the subject is scheduled for a surgical intervention.
- •17. The subject is identified by the investigator as being unable or unwilling to cooperate with study procedures.
- •18. The subject has 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.
- •19. The subject is member of the study team or in a dependent relationship with one of the study team members which includes close relatives (i.e., children, partner/spouse, siblings, and parents) as well as employees.
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