Open Label, Phase III, Multicenter study on Pharmacokinetic, Efficacy and Safety evaluation of Kedrion Factor VIII concentrate (EMOCLOT) in 50 previously treated Haemophilia A Children.
试验速览
- 阶段
- 3 期
- 状态
- Other
- 入组人数
- 60
- 试验地点
- 5
- 主要终点
- 1.)Efficacy: Evaluation of the haemostatic efficacy of Kedrion human plasma-derived factor VIII EMOCLOT in the management of acute bleeding events.
研究概览
简要总结
Note: There was no recruitment in the study and it was never initiated. The study has also been withdrawn; notification regarding the same has also been submitted to DCGI.
Protocol Title: Open Label, Phase III, Multicenter study on Pharmacokinetic, Efficacy and Safety evaluation of Kedrion Factor VIII concentrate (EMOCLOT) in 50 previously treated Haemophilia A Children.
Objective:
- Evaluation of the Pharmacokinetics, safety & efficacy assessment of the EMOCLOT.
Efficacy:
· Evaluation of the haemostatic efficacy of Kedrion human plasma-derived factor VIII EMOCLOT in the management of acute bleeding events
Safety:
· Assessment of the risk of inhibitor (neutralizing anti-factor VIII antibodies) development in conjunction with infusion of the IMP throughout the study period.
· Evaluation of short & medium term safety of the IMP when administered to severe previously treated hemophilia A subjects.
Evaluation of immediate and delayed local tolerability
研究设计
- 研究类型
- Interventional
- 分配方式
- Not Applicable
- 盲法
- Not Applicable
入排标准
- 年龄范围
- 0.00 Year(s) 至 12.00 Year(s)（—）
- 性别
- All
入选标准
- •1.Previously treated patients aged <12 years.
- •2.Prior to study entry, subjects should have a history of at least 50 exposure days for all types of factor VIII containing products, including fresh frozen plasma (FFP).
- •This minimum exposure rate shall be estimated by the Heamophilia treating physician.
- •3.Subjects should have severe or moderately severe Heamophilia A as defined by a baseline factor VIII level <1%, documented at pre-enrolment screening on the basis of anamnesis data (i.e., at heamophilia diagnosis).
- •4.If subjects are HIV-1 seropositive they should have a CD4+ lymphocyte count ≥ 200/µl documented on 2 consecutive occasions over a 12 months period prior to study entry.
- •The latest CD4+ lymphocyte count qualifying the patient for enrolment (i.e. ≥ 200/µl) should be within 6 months before enrolment in the study.
- •5.Legally authorized representatives of the subjects should be informed of the nature of the study, agree to its provision, and sign and date an informed consent and authorization for the participation and data handling approved by the IRB/IEC.
- •6.Subjects who will be available for the duration of the study will be included.
排除标准
- •1.Subjects with a detectable inhibitor to factor VIII at the time of enrolment or a history of inhibitor to factor VIII ( 0.6 BU).
- •2.Subjects HIV positive who are treated with highly active anti-retroviral therapy (HAART) regimen.
- •3.Subjects with any condition which, in the opinion of the Investigator, might interfere with the evaluation of the study objectives, or participation in this trial.
- •4.Informed Consent and/or the Authorization for Data Handling approved by the EC not signed by parents/legal guardians.
- •5.Subjects who have participated in another clinical trial within 1 month before study initiation, i.e. they have received any test drug within 30 days prior the study.
结局指标
主要结局
1.)Efficacy: Evaluation of the haemostatic efficacy of Kedrion human plasma-derived factor VIII EMOCLOT in the management of acute bleeding events.
时间窗: At 10-15 EDs or Every 3 Months | At 25±2 EDs and at 50 EDs | For PK study participating subject safety and efficacy assessment also will be done at pre infusion and 30 Minutes, 1-3 hours, 4-6 hours, 10-14 hours, 20-26 hours & 32-48 hours post infusion.
2.)Safety: Assessment of the risk of inhibitor (neutralizing anti-factor VIII antibodies) development in conjunction with infusion of the IMP throughout the study period.
时间窗: At 10-15 EDs or Every 3 Months | At 25±2 EDs and at 50 EDs | For PK study participating subject safety and efficacy assessment also will be done at pre infusion and 30 Minutes, 1-3 hours, 4-6 hours, 10-14 hours, 20-26 hours & 32-48 hours post infusion.
次要结局
- NA(NA)