Severe haemophilia A Haematological Disorders Hereditary factor VIII deficiency
Conditions
Interventions
All patients will be treated in accordance with their needs until the product is registered and launched in the country of conductance. There are no further interventions planned beside the three-mont
Sponsors
Octapharma AG (Switzerland)
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Must have severe haemophilia A (FVIII:C less than 1%; historical value as documented in subject records) 2. Aged greater than 18 years and less than 65 years, male only 3. Body weight 45 kg to 110 kg 4. Previously treated with human-cl rhFVIII (within study GENA-09) 6. Negative for human immunodeficiency virus (HIV) or respective viral load less than 200 particles/µL 7. Freely given written informed consent
Exclusion criteria
Exclusion criteria: 1. Other coagulation disorder than haemophilia A 2. Present or past FVIII inhibitor activity (greater than 0.6 BU) 3. Severe liver or kidney disease (alanine aminotransferase [ALAT] and aspartate aminotransferase [ASAT] levels greater than 5 times of upper limit of normal, creatinine greater than 120 µmol/L) 4. Receiving or scheduled to receive immuno-modulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (equivalent to greater than 10 mg/day), or similar drugs 5. Participation in another clinical study currently or during the past month, except GENA-09
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Immunogenicity: Inhibitor activity will be determined by the modified Bethesda assay (Nijmegen modification) at three months intervals until study completion. At the same time-points the anti-rhFVIII antibodies will be measured. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Clinical tolerability: assessed by monitoring vital signs (blood pressure, heart rate, respiratory rate, and body temperature will be assessed at pre-defined time-points) 2. Laboratory parameters: 2.1. Haematological parameters - red blood cell count, white blood cell count, haemoglobin, haematocrit, and platelet count 2.2. ALAT, ASAT, serum creatinine 3. Adverse events (AEs) 4. Prophylactic treatment: the frequency of bleeds under prophylactic treatment will be calculated. Study drug consumption data (FVIII IU/kg per month, per year) per subject and in total will be evaluated. 5. Treatment of bleeding episodes: efficacy assessment at the end of each BE 6. In-vivo recovery: calculated from the FVIII:C plasma levels measured before infusion and peak level obtained in the 30 or 60 minutes post-infusion sample and the actual potency of Human-cl rhFVIII. FVIII:C in the product and in plasma will be measured both by the chromogenic (CHR) and the one-stage (OS) assay. | — |
Countries
Russian Federation
Outcome results
None listed