CTIS2023-509769-18-00招募中1 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 - 071102
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 27
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
- 0 至 17（—）
- 性别
- All
入选标准
- •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)., Age 0 to <18 years at the time of screening., The subject has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent., If female of childbearing potential, subject presents with a negative serum pregnancy test., If applicable, subject agrees to employ adequate birth control measures for the duration of the study., 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 as well as for subjects undergoing surgery: 1. Unable to tolerate or are inadequately responsive to deamino-delta-Darginine vasopressin (DDAVP) 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 VWF replacement therapy., Additional inclusion criterion for previously untreated subjects: The subject has not received prior VWF coagulation factor replacement therapy.
排除标准
- •Diagnosis of pseudo-VWD or another hereditary or acquired coagulation disorder (eg, qualitative and quantitative platelet disorders or elevated prothrombin time/international normalized ratio >1.4), 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., Diagnosis of renal disease, with a serum creatinine level =2.5 mg/dL., Immunomodulatory drug treatment other than anti-retroviral chemotherapy (eg, a-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)., If female, subject is pregnant or lactating at the time informed consent (or assent, if appropriate) is obtained., 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 other than rVWF or investigational device during the course of this study., Subject's legal representative is a family member or employee of the Investigator., History or presence of a VWF inhibitor at Screening., 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.), Documented history of a VWF:RCo half-life <6 hours., Known hypersensitivity to any of the components of the study drug, such as mouse or hamster proteins., Medical history of immunological disorders, excluding seasonal allergic rhinitis/ conjunctivitis/ asthma, food allergies, or animal allergies, Medical history of a thromboembolic event., Human immunodeficiency virus positive, with an absolute CD4 count <200/mm3., 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.
研究者
相似试验
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 Diseasebleeding disorder10018849HemophiliaNL-OMON50545Baxalta Inovation GmbH1
进行中（未招募）
1 期
A study to determine the effectiveness, safety, and tolerability of the Recombinant Von Willebrand Factor administered with or without Advate for children diagnosed with Severe von Willebrand Disease who experience bleeding episodes and/or will undergo major, minor or oral surgery procedures.Hereditary severe von Willebrand Disease in childrenMedDRA version: 20.0Level: LLTClassification code 10055168Term: Von Willebrand's factor deficiencySystem Organ Class: 100000004850EUCTR2016-001477-33-NLBaxalta Innovations GmbH34
进行中（未招募）
1 期
A study to determine the effectiveness, safety, and tolerability of the Recombinant Von Willebrand Factor administered with or without Advate for children diagnosed with Severe von Willebrand Disease who experience bleeding episodes and/or will undergo major, minor or oral surgery procedures.Hereditary severe von Willebrand Disease in childrenMedDRA version: 19.0Level: LLTClassification code 10055168Term: Von Willebrand's factor deficiencySystem Organ Class: 100000004850EUCTR2016-001477-33-GBBaxalta Innovations GmbH40
进行中（未招募）
1 期
A study to determine the effectiveness, safety, and tolerability of the Recombinant Von Willebrand Factor administered with or without Advate for children diagnosed with Severe von Willebrand Disease who experience bleeding episodes and/or will undergo major, minor or oral surgery procedures.EUCTR2016-001477-33-ESBaxalta Innovations GmbH40
进行中（未招募）
1 期
A study to determine the effectiveness, safety, and tolerability of the Recombinant Von Willebrand Factor administered with or without Advate for children diagnosed with Severe von Willebrand Disease who experience bleeding episodes and/or will undergo major, minor or oral surgery procedures.EUCTR2016-001477-33-BEBaxalta Innovations GmbH34