Severe Hemophilia-A (< 1% FVIII:C) MedDRA version: 13.1 Level: LLT Classification code 10060613 Term: Hemophilia A (Factor VIII) System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 13.1 Level: LLT Classification code 10053753 Term: Hemophilia A without inhibitors System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 13.1 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 10010331 - Congenital
Conditions
Interventions
Product Name: BAY 81-8973 (250 IU/vial)
Product Code: BAY 81-8973
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Octocog alfa
Current Sponsor code: BAY 81-8973
Sponsors
Bayer HealthCare AG
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: • Male, aged 12 to 65 years • Severe hemophilia A, defined as 6 consecutive months in the previous 5 years • No current evidence of inhibitor antibody as measured by the Nijmegen-modified Bethesda assay [=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Presence of another bleeding disease that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B) • Thrombocytopenia (platelet count 2.0 mg/dL) • Presence of active liver disease verified by medical history or persistent and increased alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 5x the upper limit of normal (ULN) or severe liver disease as evidenced by an INR >4, hypoalbuminemia, and portal vein hypertension. • Received treatment with immunomodulatory agents within the last 3 months prior to study entry or requires treatment during the study. [The following drugs are allowed: interferon-a treatment for hepatitis C virus (HCV), highly active antiretroviral therapy (HAART) for human immunodeficiency virus (HIV), and or a total of 2 courses of pulse treatment with steroids for a maximum of 7 days at 1 mg/kg or less]. • Absolute CD4 lymphocyte cell count < 250 cells/?L (as of Amd 1). • Receiving or has received other experimental drugs within 3 months prior to study entry, with the exception of Bayer Kogenate (Bayer factor VIII study drugs) received in studies within 2 weeks prior to study entry. (as of Amd 1) • Requires any pre-medication to tolerate FVIII injections (e.g., antihistamines) • Unwilling to comply with study visits or other protocol requirements or is not suitable for participation in this study for any reason, according to the Investigator • Known hypersensitivity to hamster and/or mouse protein. • Any subject who cannot forego at least 2-3 days without receiving FVIII for washout purposes. (as of Amd 3)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To demonstrate the superiority of prophylaxis over on demand therapy by a clinically significant decrease in bleeding rate following 12 months of treatment with BAY 81-8973.;Secondary Objective: - To demonstrate superiority of prophylaxis versus on-demand treatment with BAY 81-8973 (dose determined by CS/EP) as measured by bleeding rate. - To demonstrate superiority of prophylaxis versus on-demand treatment with BAY 81-8973 (dose determined by CS/ADJ) as measured by bleeding rate. - To demonstrate the non-inferiority of prophylactic treatment with BAY 81 8973 (dose determined by CS/EP versus dose determined by CS/ADJ) as measured by bleeding rate (after combining results of this study with results from Protocol 12954). - To demonstrate the non-inferiority of BAY 81-8973 dose determined by CS/EP versus BAY 81-8973 dose determined by CS/ADJ as measured by the proportion of bleeds controlled by 1 or 2 infusions (among all bleeds) in on-demand patients. (as of Amd 1) - To demonstrate the non-inferiority of prophylactic treatment with BAY 81 8973 (dose determined by CS/EP versus dose determined by CS/ADJ) as measured by in vivo recovery (IVR). ;Primary end point(s): To demonstrate the superiority of prophylaxis over on demand therapy by a clinically significant decrease in bleeding rate following 12 months of treatment with BAY 81-8973. | — |
Countries
Argentina, China, Colombia, Czech Republic, Japan, Mexico, Romania, Russian Federation, Serbia, South Africa, Taiwan, Turkey, Ukraine, United States
Contacts
Public ContactCTP Team / Ref: "EU CTR" / S102 - R
Bayer HealthCare AG
Outcome results
None listed