Randomized, Active-controlled, Double-blind, Parallel Design Study to Evaluate the Efficacy and Safety of a Once-a-week Prophylaxis Treatment With BAY79-4980 Compared to Three Times-per-week Prophylaxis With rFVIII-FS in Previously Treated Patients With Severe Hemophilia A
Trial Snapshot
- Phase
- Phase 2
- Status
- Terminated
- Sponsor
- Enrollment
- 143
- Locations
- 97
- Primary Endpoint
- Percentage of Participants With Less Than 9 Total Bleeds Per Year
Study Overview
Brief Summary
A study to assess treatment with a new formulation of recombinant factor VIII reconstituted with liposomes (BAY79-4980) to evaluate whether a once-a-week treatment is safe and can prevent bleeds in subjects with severe haemophilia A.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Eligibility Criteria
- Ages
- 12 Years to 70 Years (Child, Adult, Older Adult)
- Sex
- Male
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Males aged 12 to 70 years
- •Subjects with severe hemophilia A (< 1% factor VIII [FVIII]:C)
- •Subjects with equal or greater than 150 exposure days (EDs) with any FVIII in total
- •Subjects who have been on-demand treatment with a minimum of 1 relevant bleed per month or have been on secondary prophylaxis treatment with not more than a 3x/week schedule
- •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
- •Subjects with no history of FVIII inhibitor antibody formation
- •Written informed consent by subject and parent / legal representative, if < 18 years
Exclusion Criteria
- •Subjects who are receiving primary prophylaxis
- •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 abnormal renal function
- •Subjects with elevated hepatic transaminases
- •Subjects on treatment with immunomodulatory agents within the last 3 months prior to study entry or during the study
- •Subjects with known hypersensitivity to the active substance, mouse or hamster protein, liposomes or polyethyleneglycol (PEG)
- •Subjects who require any pre-medication for FVIII injections
Arms & Interventions
rFVIII-FS/WFI (BAY14-2222)
25 IU/kg body weight of rFVIII-FS 3x/week (employing 1 percent POPC (1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine)-alone liposome (rFVIII-FS-POPC) as blinding agent used for first weekly injection and rFVIII-FS in WFI for 2nd and 3rd injection)
Intervention: rFVIII-FS/WFI (BAY14-2222) (Biological)
rFVIII-FS/pegylated liposomes (BAY79-4980)
35 IU/kg body weight of BAY79-4980 1x/week plus 2 dummy injections/week (dummy = rFVIII (recombinant factor VIII)-FS (formulated with sucrose) excipient reconstituted in WFI (sterile water for injection))
Intervention: rFVIII-FS/pegylated liposomes (BAY79-4980) (Biological)
Outcomes
Primary Outcomes
Percentage of Participants With Less Than 9 Total Bleeds Per Year
Time Frame: up to one year
Bleeds occurring on the same day were counted as one bleeding event. Bleeds occurring within 72 hours into the same location were also counted as one bleeding event.
Secondary Outcomes
- Percentage of Participants With Less Than 5 Joint Bleeds Per Year(up to one year)
- Number of Joint Bleeds Per Participant Per Year in Responders(up to one year)