A two part randomized, cross-over, open-label trial to evaluate the pharmacokinetics, efficacy, and safety profile of plasma-protein free recombinant FVIII formulated with sucrose (BAY 81-8973) in previously treated subjects with severe hemophilia A under prophylaxis therapy - ND
- Conditions
- Severe Hemophilia-AMedDRA version: 9.1Level: LLTClassification code 10053753MedDRA version: 9.1Level: LLTClassification code 10060612MedDRA version: 9.1Level: LLTClassification code 10060613
- Registration Number
- EUCTR2009-012149-43-IT
- Lead Sponsor
- Bayer HealthCare AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 229
• Male, aged 12 to 65 years • Severe hemophilia A, defined as < 1% FVIII:C as determined by one-stage clotting assay • =150 exposure days (ED) in total with any recombinant FVIII or plasma-derived FVIII only. Cryoprecipitate and fresh frozen plasma treatments are not considered in this total. (as of Amd 1) • Currently receiving on-demand or any type of prophylaxis treatment regimen with any FVIII product. • No current evidence of inhibitor antibody as measured by the Nijmegen modified Bethesda assay [<0.3 Bethesda units (BU/mL)] (as of Amd 5) in 2 consecutive samples and absence of clinical signs or symptoms of decreased response to FVIII administration. (First negative sample can be historical if obtained within 3 months prior to screening. Second negative, confirmatory sample testing must, in all cases, be performed by a central laboratory using the Nijmegen test. If a first recent sample is not available, then testing for 2 negative samples must be performed by the central laboratory at least 1 week apart). Subjects may not receive FVIII within 72 hours (as of Amd 5) prior to the collection of samples for inhibitor testing. • No history of FVIII inhibitor formation, defined as inhibitor antibody < 0.6 BU/mL, by the Nijmegen modified Bethesda assay. However, patients with a maximum historical titer of 1.0 BU (as of Amd 5) with the Classical Bethesda assay on no more than 1 occasion but with at least 3 subsequent (as of Amd 1) successive negative results (<0.6 BU) thereafter are also eligible. • Willingness and ability to complete training in the use of the study electronic patient diary (EPD) by the subject or a surrogate (a caregiver or family member over 18 years of age). (as of Amd 1) • Written informed consent by subject and parent/legal representative, if under age of consent per local regulation.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
• Presence of another bleeding disease that is different from hemophilia A (e.g., von Willebrand disease, hemophilia B) • Thrombocytopenia (platelet count < 100,000/mm3) • Abnormal renal function (serum creatinine > 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 significant portal vein hypertension in the judgment of the Investigator. (as of Amd 5) • 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 5) • 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 protein. • Any subject in Part-A who cannot forego at least 3 days without receiving FVIII before the PK sessions for washout purposes.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method