NL-OMON50545已完成3 期
A Phase 3, Prospective, Multicenter, Uncontrolled, Open-Label Clinical Study to Determine the Efficacy, Safety, and Tolerability of rVWF with or without ADVATE in the Treatment and Control of Bleeding Episodes, the Efficacy and Safety of rVWF in Elective and Emergency Surgeries, and the Pharmacokinetics (PK) of rVWF in Children Diagnosed with Severe von Willebrand Disease - BAX 111 rVWF in Pediatrics
Baxalta Inovation GmbH0 个研究点目标入组 1 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 1
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 17（—）
入选标准
- •Subjects who meet ALL of the following criteria are eligible for this
- •1. Diagnosis of severe VWD (defined as VWF:RCo <20%):
- •a. Type 1 (VWF:RCo <20 IU/dL); or
- •b. Type 2A (VWF:RCo <20 IU/dL), Type 2B (as diagnosed by genotype),
- •Type 2N (FVIII:C
- •<10% and historically documented genetics), Type 2M; or
- •c. Type 3 (VWF:Ag *3 IU/dL).
- •2. Age 0 to <18 years at the time of screening
- •3. The subject has provided assent (if appropriate) and legally
- •authorized representative(s) has
- •provided informed consent
- •4. If female of childbearing potential, subject presents with a negative
- •serum pregnancy test
- •5. If applicable, subject agrees to employ adequate birth control
- •measures for the duration of the study
- •6. Subject and/or the legally authorized representative are willing and able to
- •comply with the requirements of the protocol, which should also be confirmed
- •based on a prescreening evaluation held between the Investigator and the
- •Sponsor, to ensure no eminent risk is present that could challenge the
- •subject's compliance with the study requirements.
- •Additional inclusion criteria for previously treated subjects and subjects
- •undergoing surgery are as follows:
- •1. Unable to tolerate or are inadequately responsive to deamino-delta-Darginine
- •vasopressin
- •2. The subject has had a minimum of 1 documented bleed requiring VWF
- •coagulation factor
- •replacement therapy (ie, treatment with a VWF product) during the previous 12
- •months prior to enrollment
- •and overall historically
- •3 or more exposure days (EDs) to plasma-derived VWF.
- •Additional inclusion criterion for previously untreated subjects are as
- •1. The subject has not received prior VWF coagulation factor
- •replacement therapy
排除标准
- •Subjects who meet ANY of the following criteria are not eligible for this
- •1. Diagnosis of pseudo-VWD or another hereditary or acquired
- •coagulation disorder (eg, qualitative
- •and quantitative platelet disorders or elevated prothrombin
- •time/international normalized ratio
- •2. History or presence of a VWF inhibitor at Screening
- •3. History or presence of a FVIII inhibitor with a titer *0.4 Bethesda
- •units (BU) (by Nijmegen assay)
- •or *0.6 BU (by Bethesda assay)
- •4. Documented history of a VWF:RCo half-life <6 hours
- •5. Known hypersensitivity to any of the components of the study drug,
- •such as mouse or hamster
- •6. Medical history of immunological disorders, excluding seasonal
- •allergic rhinitis/ conjunctivitis/
- •asthma, food allergies, or animal allergies
- •7. Medical history of a thromboembolic event
- •8. Human immunodeficiency virus positive, with an absolute CD4 count
- •9. In the judgment of the Investigator, the subject has another clinically
- •significant concomitant
- •disease (eg, uncontrolled hypertension, cancer) that may pose additional
- •risks for the subject
- •10. Diagnosis of significant liver disease, as evidenced by, but not
- •limited to, any of the following:
- •serum alanine aminotransferase 5 times the upper limit of normal;
- •hypoalbuminemia; portal vein
- •hypertension (eg, presence of otherwise unexplained splenomegaly,
- •history of esophageal varices)
- •or liver cirrhosis classified as Child B or C
- •11. Diagnosis of renal disease, with a serum creatinine level *2.5 mg/dL
- •12. Immunomodulatory drug treatment other than anti-retroviral
- •chemotherapy (eg, *-interferon, or
- •corticosteroid agents at a dose equivalent to hydrocortisone greater than
- •10 mg/day (excluding
- •topical treatment [eg, ointments, nasal sprays]), within 30 days prior to
- •signing the informed
- •consent (or assent, if appropriate)
- •13. If female, subject is pregnant or lactating at the time informed
- •consent (or assent, if appropriate) is
- •14. Subject has participated in another clinical study involving an IP,
- •other than rVWF with or
- •without ADVATE, 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
- •15. Subject's legal representative is a family member or employee of the
- •Investigator
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