Randomized, active-controlled, double-blind, parallel design study to evaluate the efficacy and safety of a once-a-week prophylaxis treatment with BAY 79-4980 compared to three times-per-week prophylaxis with rFVIII-FS in previously treated patients with severe hemophilia A
- Conditions
- Hemophilia A10064477
- Registration Number
- NL-OMON33645
- Lead Sponsor
- Bayer
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- Not specified
- Target Recruitment
- 10
- Males aged 18 to 70 years
- Subjects with severe hemophilia A (< 1% FVIII:C)
- Subjects with equal or greater than 150 EDs with any FVIII in total
- Subjects who have been on-demand treatment (no more than 40% of patients and at least 20% are high-frequency bleeders [>=20 bleeds/year]) with an average of minimum 1 relevant bleed per month or have been on secondary prophylaxis treatment with not more than a 3x/week schedule with the history of minimum 12 relevant bleeds per year prior to be on secondary prophylaxis treatment
- Subjects with bleeding events and/or treatments during the last 6 months prior to study entry which are documented in the subjects medical records.
- Subjects with no measurable inhibitor activity using the Nijmegen-modified Bethesda assay (>0.6BU/mL is considered positive) in two consecutive samples and absence of clinical signs or symptoms of decreased response to FVIII administration.
- Subjects with no history of FVIII inhibitor antibody formation. (>=0.6BU/mL using the Nijmegen-modified Bethesda assay)
- Subjects who complete an EPD (electronic patient diary) device training and demonstrate the ability to correctly use it
- Subjects who are receiving primary prophylaxis (start of prophylaxis before or directly after the first joint bleed without relevant interruptions)
- Subjects on prophylaxis with documented requirements of > 75 IU/kg/week
- Subjects with any other bleeding disease beside hemophilia A (i.e., von Willebrand disease)
- Subjects with thrombocytopenia (platelets < 100,000/mm3)
- Subjects with abnormal renal function (serum creatinine > 2.0 mg/dL)
- Subjects with elevated hepatic transaminases (AST or ALT > 5xULN)
- Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study (the following drugs are allowed: interferon-alpha-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)
- Subjects with an absolute CD4 lymphocyte cell count < 250 cells/mm3
- Subjects with positive Lupus Anticoagulant antibodies or history thereof
- Subjects with known hypersensitivity to the active substance, mouse or hamster protein, liposomes or PEG
- Subjects with severe dyslipidemia of all causes LDL-C >= 190 mg/dl)
- Subjects who are receiving or had received other experimental drugs within 3 months prior to study entry
- Subjects who require any pre-medication for FVIII injections (e.g. anti-histamines)
- Subjects with uncontrollable hypertension (diastolic blood pressure > 100 mmHg)
- Subjects with known unstable coronary artery angina and/or with known history of myocardial infarction.
- Subjects who are unwilling to comply with study visits or the treatment regimens
- Subjects who have planned major surgery (including orthopedic) or radioisotopic synovectomy during the study
- Subjects who are not suitable for participation in this study for any reason, according to the Investigator
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary efficacy variable will be the percentage of subjects with less than<br /><br>9 total bleeds per year. The three weeks run-in phase will not be considered<br /><br>for efficacy analyses.</p><br>
- Secondary Outcome Measures
Name Time Method