This clinical trial is to study Immune Tolerance Induction, by Factor VIII concentrate containing von Willebrand factor, in severe or moderate haemophilia A patients with inhibitors.
- Conditions
- Health Condition 1: null- Severe or moderate haemophilia A patients with inhibitors.
- Registration Number
- CTRI/2014/07/004749
- Lead Sponsor
- Sintesi Research
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 6
1.Subjects (his/her parent/legal representative), must have given a written informed consent
2.Male children: age <12 years
3.Severe or moderate hemophilia A (FVIII <2%)
4.High responders (clinical history of inhibitor peak > 5BU)
Or low-responders with potential bleedings, assessed by responsible physicians as not to be treated with high FVIII doses.
5.Any level of inhibitor at study enrollment.
6.Willingness and ability to participate in the study.
7.No other experimental treatments
1.Any clinically relevant abnormality, in hematological, biochemical and urinary routine examinations, or any condition or treatment which in the investigatorâ??s opinion, makes the patient not eligible for the study.
2.Intolerance to active substance or to any of the excipients of FVIII / VWF concentrate.
3.Concomitant systemic treatment with immunosuppressive drugs.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Inhibitor reduction (Details in Brief Summary). <br/ ><br>Timepoint: 6, 12 and 33 months of treatment. <br/ ><br>
- Secondary Outcome Measures
Name Time Method Efficacy: <br/ ><br>Time to achieve IT: Complete or Partial response. <br/ ><br>IT persistence: Absence of relapse, assessed at 12 months from the IT achievement. <br/ ><br>FVIII genetic defect role in IT achievement: Role of FVIII mutations in influencing IT achievement. <br/ ><br>Role of an immediate IT to delayed IT in IT induction: Time elapsing between the onset of the inhibitor and the beginning of treatment. <br/ ><br>Timepoint: 12 months (for IT persistence).;Safety: <br/ ><br>Incidence of AE during the course of prophylactic treatment, with severity, correlation with the IP and final outcome. <br/ ><br>The tolerability of the IP will also be assessed at the different infusion rates of administration. <br/ ><br>Trend of the inhibitor title will be also assessed during the course of prophylactic treatment. <br/ ><br>Timepoint: NA.;Treatment compliance: <br/ ><br>Patientâ??s adherence to the optimal prolonged treatment.Timepoint: NA.