BAY79-4980 Compared to rFVIII-FS in Previously Treated Patients With Severe Hemophilia A

Bayer

Status and phase: Terminated; Phase 2

Conditions: Hemophilia A

Treatments: Biological: rFVIII-FS/pegylated liposomes (BAY79-4980); Biological: rFVIII-FS/WFI (BAY14-2222)

Study type: Interventional

Funder types: Industry

Identifiers

NCT00623727

12781

2007-003718-32 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 143 patients

Sex: Male

Ages: 12 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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143 participants in 2 patient groups

rFVIII-FS/pegylated liposomes (BAY79-4980)

Experimental group

Description:

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))

Treatment:

Biological: rFVIII-FS/pegylated liposomes (BAY79-4980)

rFVIII-FS/WFI (BAY14-2222)

Active Comparator group

Description:

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)

Treatment:

Biological: rFVIII-FS/WFI (BAY14-2222)