Health Condition 1: null- Severe Hemophilia A
Conditions
Interventions
Intervention1: Recombinate Product: Recombinate: Therapeutic Regimen for on demand treatment - Administered through a dose of 40 IU/kg at bleeding occurrence, followed by 20IU/kg at 24 and 72 hours
Pr
Sponsors
Sintesi Research
Max Neeman International
Eligibility
Inclusion criteria
Inclusion criteria: 1. Male subjects 2. Any ethnicity 3. Age 6 years 4. Severe haemophilia A (FVIII:C 1%), as confirmed by the central laboratory 5. A Patient with FVIII levels 1% and 2% will be separately recorded in the screening list 6. Previously untreated (0 EDs to any FVIII concentrate or blood products) or minimally treated (5 EDs) with blood components, namely whole blood, fresh frozen plasma, packed red blood cells, platelets or cryoprecipitate 7. A Patient not meeting these criteria will be separately recorded in the screening list. 8. Negative inhibitor measurement at both local and central laboratory at screening 9. Ability to comply with study requirements 10. Signed informed consent of legal tutors
Exclusion criteria
Exclusion criteria: 1. Plasma FVIII level >=1%, as assayed at the central laboratory 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. 2. Previous history of FVIII inhibitor 3. Other congenital or acquired bleeding defects 4. Concomitant congenital or acquired immunodeficiencies 5. Concomitant treatment with systemic immunosuppressive drugs 6. Concomitant treatment with any investigational drug
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To assess the immunogenicity of VWF/FVIII and of rFVIII concentrates by determining the frequency of inhibitor development in PUPs and MBCTs in the first 50 EDs or in the first 3 years from enrolment, whichever comes first.Timepoint: Enrollment visit,Every 3-4 EDs, Every 10 EDs,Every 3 months, Annual visit,Final visit | — |
Secondary
| Measure | Time frame |
|---|---|
| To evaluate the anamnestic response of inhibitor patients Timepoint: Enrollment visit,Every 3-4 EDs, Every 10 EDs, Every 3 months, Annual visit, final visit.;To evaluate the bleeding history (age at first bleed, bleeding pattern)Timepoint: Enrollment visit,Every 3-4 EDs, Every 10 EDs, Every 3 months, Annual visit, final visit.;To evaluate the frequency of transient inhibitors.Timepoint: Enrollment visit,Every 3-4 EDs, Every 10 EDs, Every 3 months, Annual visit, final visit.;To evaluate the incidence of all other adverse events related and not related to the products used.Timepoint: Enrollment visit,Every 3-4 EDs, Every 10 EDs, Every 3 months, Annual visit, final visit.;To evaluate the modality of occurrence of inhibitors (number of EDs, titre at onset, etc)Timepoint: Enrollment visit,Every 3-4 EDs, Every 10 EDs, Every 3 months, Annual visit, final visit. | — |
Countries
Argentina, Austria, Belgium, Brazil, Chile, Czech Republic, Denmark, Egypt, Fiji, France, Germany, India, Iran (Islamic Republic of), Italy, Mexico, Poland, Portugal, Romania, Saudi Arabia, Slovakia, South Africa, Spain, Switzerland, Turkey, United Kingdom, United States of America, Uruguay
Contacts
Public ContactDr Shariq Anwar
Max Neeman International
Outcome results
None listed