Haemophilia A MedDRA version: 8.1 Level: LLT Classification code 10018937 Term: Haemophilia A
Conditions
Interventions
Product Name: BAY 79-4980
Product Code: BAY 79-4980
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: octocog alph
Sponsors
Bayer Healthcare LLC
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: • Males aged 12 to 70 years • Severe hemophilia A ( 20 ED during last year • On-demand treatment for the past year (Subjects having received isolated courses of prophylaxis for 6 consecutive weeks or less in a maximum of 2 occasions in the previous 12 months are also eligible) • At least 20 documented bleeds during the last 12 months (Or equivalent annualized bleeding frequency in no less than the previous 6 months, or documented corresponding infusions > 4 days apart, or if the treating physician confirms this bleeding frequency and this information is documented in the subjects medical records or a combination of them). • No current evidence of inhibitor antibody measured using the Nijmegen modified Bethesda assay (=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Individuals with von Willebrand disease • Any individual with thrombocytopenia (platelets 2.0 mg/dL) • Any individual with active hepatic disease (AST or ALT > 5xULN) • Any individual on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study. (the following drugs are allowed: alpha interferon treatment for HCV, HAART therapy for HIV and/or a total of two courses of pulse treatment with steroids for a maximum of 7 days at 1mg/kg or less) • Any individual with (HIV, HCV or due to another suspicious condition) an absolute CD4 lymphocyte cell count 100 mmHg) which is uncontrollable with proper medication • Any individual with known unstable coronary artery angina and/or with known history of myocardial infarction. • Any individual who is unwilling to comply with study visits or any of the possible treatment regimens (e.g. not willing to receive on-demand treatment for one year) • Planned major surgery (including orthopedic) during the study • Planned radio-isotopic synovectomy during the study • Any individual who is not suitable for participation in this study for any reason, according to the Investigator • Only for subjects participating in the PK substudy: o Any individual receiving any pegylated medication (i.e. PEG interferon) in th e month prior to the PK session o Any subject not willing to or able to comply with fasting requirements (from 10pm previous night until 6 hours post infusion)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to evaluate the effect of a once-a-week prophylaxis regimen with BAY 79-4980 on the frequency of all bleeds compared to once-a-week prophylaxis regimen and to on-demand treatment (OD) with rFVIII-FS in WFI (Kogenate Bayer). ;Secondary Objective: The main secondary objective is to evaluate the effect of a once-a-week prophylaxis regimen with BAY 79-4980 on the frequency of spontaneous bleeds (e.g joints, muscle) compared to once-a-week prophylaxis regimen and on-demand treatment (OD) with rFVIII-FS-WFI (Kogenate Bayer).;Primary end point(s): The primary efficacy variable will be the number of all bleeds (spontaneous and trauma) occurring during 49 weeks of active treatments excluding the cumulative 10 weeks of placebo treatment | — |
Countries
Austria, Belgium, Czech Republic, France, Netherlands, Slovakia, United Kingdom
Outcome results
None listed