Severe Haemophilia A MedDRA version: 12.1 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII)
Conditions
Interventions
Product Name: Human cell line recombinant factor VIII
Product Code: human-cl rhFVIII
Pharmaceutical Form: Powder and solvent for solution for injection
Other descriptive name: Human-cl rhFVIII
Concent
Sponsors
Octapharma AG
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: ? Severe haemophilia A (FVIII:C = 2 and 200/ µL) ? HIV negative or respective viral load =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: ? Other coagulation disorder than haemophilia A ? Present or past FVIII inhibitor activity (= 0.6 BU) ? Target joints ? Severe liver or kidney disease (ALT and AST levels >5 times of upper limit of normal, creatinine >120 µmol/L) ? Receipt or scheduled receipt of immuno-modulating drugs (other than anti-retroviral chemotherapy) such as alpha-interferon, prednisone (>10 mg/day), or comparable drugs ? Current participation in another clinical study ? Participation in another interventional clinical study with administration of investigational medical product (IMP) in the course of the past 3 months, except studies investigating already registered FVIII products
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess clinical efficacy of Human-cl rhFVIII in terms of prevention and treatment of (breakthrough) bleeding episodes;Secondary Objective: • to determine pharmacokinetics (in 50% of the subjects included) of Human-cl rhFVIII in terms of the AUC, the in-vivo half-life (T1/2), in-vivo recovery, Cmax, Tmax, MRT, Vss, and CL; the results are compared with those obtained from a PK determination with the previously used FVIII concentrate; • to determine the incremental recovery of Human-cl rhFVIII – also over time; • to investigate the immunogenic potential of Human-cl rhFVIII by assessing the inhibitor titre; • to assess efficacy of Human-cl rhFVIII in surgeries; • to assess safety of Human-cl rhFVIII in terms of adverse event monitoring. ;Primary end point(s): Primary Endpoints: 1.) Efficacy of Prophylactic Treatment: Subjects are all treated prophylactically. The frequency of breakthrough bleeds under prophylactic treatment will be calculated. Study drug consumption data (FVIII IU/kg, extrapolated to monthly and yearly usage) both per subject and in total will be evaluated. The following parameters will be documented: • Dates and times of study product injections. • Details of dose(s) used for prophylactic treatment (in IU FVIII). 2.) Efficacy of On-Demand Treatment of Breakthrough Bleeding Episodes For all BEs occurring in the course of the study period the following parameters will be documented: • Details of the respective BE (see Section 6.1.4). • Efficacy assessment at the end of the BE (see Section 6.1.4.1). • Details of date, time, dose and batch number used to treat BE (in IU FVIII) (see Section 6.1.4.1 of the protocol) | — |
Countries
Austria, Czech Republic, Germany, United Kingdom
Outcome results
None listed