Patients with severe or moderate B Haemophilia, previously treated with rFIX. MedDRA version: 9.1 Level: SOC Classification code 10010331
Conditions
Interventions
Trade Name: BENEFIX
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Nonacog alfa
Concentration unit: IU international unit(s)
Concentration type: equal
Concentr
Sponsors
AZIENDA OSPEDALIERA UNIVERSITARIA CAREGGI
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Severe or moderate B haemophilia (factor IX activity =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patients ongoing treatment with plasmaderived factor IX (pd-FIX); 2. For patients with documented HIV infection, CD4 count 2 mg/dL, seric albumin 1.25 x ULN); 4. Prothrombin Time >1.5 x ULN; 5. Platelet count < 150.000/µL; 6. Patients with current or past inhibitors to factor IX; 7. Patients with history of hypersensitivity to FIX or to eccipients, or to Hamster proteins; 8 Exposure to other experimental drugs, eccept BeneFIX, in the 30 days prior to the sign of the informed consent of this study; 9. Patients with coagulation disorders/diseases different from B haemophilia; 10. Inflammatory disease documented and still present, which could, judjed by the investigator, be confounding the study results (e.g. systemic eritematosus lupus, rheumatoid arthritis, inflammatory bowel disease); 11. Patients showing low compliance to the study procedures, patients with difficult venous access, and all those patients who are judged (by the investigator) to be inadequate for this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary endpoint of the study will be the evaluation of the variability of pharmacokinetic parameters (in vivo recovery, AUC, clearance, MRT, terminal half life, Cmax, elimination k, volume of distribution) of a new formulation of recombinant factor IX in patients with severe or moderate B haemophilia, previously treated with rFIX.;Secondary Objective: Secondary endpoints of the study will be the evaluation and the characterization of: 1) Pharmacokinetic parameters using a simplified pharmacokinetic evaluation model, and validation of the method used against the standard method (Morfini M., Haemophilia 12, Suppl. 4, 2006); 2) Pharmacokinetic parameters in the paediatric population subgroup (6-12); 3) Evaluation of the correlations between pharmacokinetic profile of the single patient and the causative mutation; 4) Efficacy of the drug administration both in prophylaxis and in ?on demand? regimen of the new formulation of recombinant factor IX; 5) Evaluation of adverse events according to the standard method; 6) Evaluation of the variability in the drug consumption of the new formulation of recombinant FIX (International Units/Kg) in the study period, per single patient;;Primary end point(s): The evaluation of the study product will be performed on the base of the following variables: Efficacy The efficacy of the ?on demand? treatment with reformulated rFIX will be evaluated by the number of infusions needed and the total amount of rFIX used for the complete resolution of each bleeding episode The efficacy of the prophylaxis treatment with reformulated rFIX will be evaluated by the number of bleeding episodes elapsed during the treatment, by the time from last infusion, and by the number of infusions needed and the total amount of rFIX used for the complete resolution of each bleeding episode. The efficacy of the treatment with reformulated rFIX in surgery will nbe evaluated by the investigator through the daily detection of Hb levels and | — |
Countries
Italy
Outcome results
None listed