NCT01064284已完成4 期
Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) When Exposed to Plasma-derived Von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical Trial
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 303
- 试验地点
- 48
- 主要终点
- To Assess the Immunogenicity of Plasma Derived VWF/FVIII and rFVIII Concentrates by Determining the Frequency of Inhibitor Development in the First 50 EDs or in the First 3 Years From Enrolment, Whichever Comes First in PUPs and MBCTs
研究概览
简要总结
The primary objective of the study is to assess the immunogenicity of VWF/FVIII and of rFVIII concentrates by determining the frequency of inhibitor development in previously untreated patients (PUPs) or minimally blood component-treated (MBCTPs) in the first 50 EDs or in the first 3 years from enrollment, whichever occurs first.
.
详细描述
Patients meeting the enrollment criteria will be consecutively enrolled at each participating centre, randomized to be treated exclusively with a single FVIII product either plasma-derived or recombinant, and followed up until inhibitor development or until 50 exposure days (EDs) or 3 years from enrolment have elapsed, whichever comes first. Study products, belonging to the class of rFVIII concentrates and to the class of plasma-derived VWF/FVIII concentrates, will be provided for free to the patients for all the duration of the study
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 1 Minute 至 6 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male subjects
- •Any ethnicity
- •Age <6 years
- •Severe haemophilia A (FVIII:C <1%), as confirmed at enrolment by the central laboratory.
- •o Those patients diagnosed locally as severe but subsequently found to have FVIII levels >= 1% on testing at the central laboratory will be separately recorded in the screening list.
- •Previously untreated (0 EDs to any FVIII concentrates or blood products) or minimally treated (<5 EDs) with blood components, namely whole blood, fresh frozen plasma, packed red blood cells, platelets or cryoprecipitate.
- •o Patients not meeting these criteria will be separately recorded in the screening list.
- •Negative inhibitor measurement at both local and central laboratory at screening
- •Ability to comply with study requirements
- •Signed informed consent of legal tutors o Patients who will not accept to enter into the study or to be randomized will be separately recorded.
排除标准
- •Previous history of FVIII inhibitor
- •Other congenital or acquired bleeding defects
- •Plasma FVIII level >= 1%, as assayed at the central laboratory
- •o Those patients originally diagnosed locally as severe but subsequently found to have FVIII levels ranging from 1% to 2% on testing at the central laboratory will be separately recorded in the screening list.
- •Concomitant congenital or acquired immunodeficiency
- •Concomitant treatment with systemic immunosuppressive drugs
- •Concomitant treatment with any investigational drug
研究组 & 干预措施
PLASMA DERIVED Factor VIII
Active Comparator
Plasma-derived vWF/FVIII
干预措施: PLASMA DERIVED Factor VIII (Drug)
rFVIII
Active Comparator
Recombinant FVIII
干预措施: Recombinant FVIII (Drug)
结局指标
主要结局
To Assess the Immunogenicity of Plasma Derived VWF/FVIII and rFVIII Concentrates by Determining the Frequency of Inhibitor Development in the First 50 EDs or in the First 3 Years From Enrolment, Whichever Comes First in PUPs and MBCTs
时间窗: During the first 50 exposure days or first 3 years of enrollment, whichever occurs first
Expressed with the numebr of patients for each group who developed FVIII inhibitors. PUPs: Previously Untreated Patients MBCTPs: Minimally Blood Component-Treated Patients
次要结局
- To Evaluate the Anamnestic Response of Inhibitor Patients(During the first 50 exposure days or first 3 years of enrollment, whichever occurs first)
- To Evaluate the Frequency of Transient Inhibitors(In the 6 months after inhibitor development)
- To Evaluate the Modality of Occurrence of Inhibitors (Titre at Onset)(During 6 months of observation, from the inhibitor occurrence)
- To Evaluate the Modality of Occurrence of Inhibitors (Number of EDs)(During the first 50 exposure days or first 3 years of enrollment, whichever occurs first)
- To Evaluate Clinical Factors Potentially Associated to Inhibitor Development(During the first 50 exposure days or first 3 years of enrollment, whichever occurs first)
- To Evaluate Laboratory Factors Potentially Associated to Inhibitor Development(During the first 50 exposure days or first 3 years of enrollment, whichever occurs first)
- To Evaluate the Incidence of All Other Adverse Events Related and Not Related to the Products Used(During the first 50 exposure days or first 3 years of enrollment, whichever occurs first)
研究者
研究点 (48)
Loading locations...
相似试验
已完成
不适用
Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced)Severe Hemophilia ANCT01051076City of Hope Medical Center3
已完成
3 期
Human Cell Line-derived Recombinant Factor VIII (Human-cl-rhFVIII) in Previously Untreated PatientsSevere Hemophilia ANCT01712438Octapharma110
进行中（未招募）
不适用
Development of antibody against FVIII (inhibitors) in Untreated Patients or Minimally Blood Component-Treated Patients when Exposed Factor concentrates which can be made from human blood (plasma-derived) or manufactured in laboratory using genetically engineered cells that carry a human factor gene (recombinant): A non-commercial, international study with lots of sites and it is known which of the two by random assigned drugs will be takeInhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma-derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical TrialMedDRA version: 14.1Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2009-011186-88-DEFONDAZIONE CENTRO EMOFILIA E TROMBOSI ANGELO BIANCHI BONOMI300
进行中（未招募）
不适用
Development of antibody against FVIII (inhibitors) in Untreated Patients or Minimally Blood Component-Treated Patients when Exposed Factor concentrates which can be made from human blood (plasma-derived) or manufactured in laboratory using genetically engineered cells that carry a human factor gene (recombinant): A non-commercial, international study with lots of sites and it is known which of the two by random assigned drugs will be takeInhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma-derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical TrialMedDRA version: 16.1Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 100000004850EUCTR2009-011186-88-SKFONDAZIONE ANGELO BIANCHI BONOMI300
进行中（未招募）
不适用
Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma-derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical TrialInhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma-derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical TrialMedDRA version: 14.1Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 100000004850EUCTR2009-011186-88-PTFONDAZIONE CENTRO EMOFILIA E TROMBOSI ANGELO BIANCHI BONOMI300