A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A (PROTECT-VIII)

Bayer

Status and phase: Completed; Phase 3; Phase 2

Conditions: Hemophilia A

Treatments: Biological: BAY94-9027

Study type: Interventional

Funder types: Industry

Identifiers

NCT01580293

13024

2011-005210-11 (EudraCT Number)

Details and patient eligibility

About

Haemophilia A is an inherited disorder in which one of the proteins, Factor VIII, needed to form blood clots is missing or not present in sufficient levels. In a person with haemophilia A, the clotting process is slowed and the person experiences bleeds that can result in serious problems and potential disability.

The current standard treatment for severe haemophilia A is regularly scheduled infusion of FVIII to keep levels high enough to prevent bleeding. Due to the short half-life of FVIII, prophylaxis may require treatment as often as every other day.

In this trial safety and efficacy of a long-acting recombinant factor VIII molecule is evaluated in subjects with severe Hemophilia A.

120-140 patients will receive open label treatment with long-acting rFVIII either on-demand to treat bleeds or prophylactically for 36 weeks in the main trial plus an optional extension to continue treatment for at least 100 total exposure days (ED). Patients on prophylactic treatment will receive study drug at dosing intervals between once and twice a week depending on their observed bleeding. Patients will attend the treatment centre for routine blood samples and be required to keep an electronic diary.

Male patients aged 12-65, with severe hemophilia A, previously treated with FVIII for at least 50 exposure days may be eligible for this study.

Full description

Subjects in prophylactic treatment arms will undergo clinical evaluation at 10 weeks. Those with adequate control of bleeding will undergo randomization to every 5 or 7 day infusion. Those with continued bleeding will remain in treatment arm and have an increase in dose.

Part B-major surgery - optional sub study included to collect information on efficacy of BAY94-9027 in major surgical setting. Due to rarity of surgery in this population, enrollment to this sub-study may be independent of participation in main study.

Enrollment: 145 patients

Sex: Male

Ages: 12 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male; 12-65 years of age
• Subjects with severe hemophilia A
• Previously treated with factor VIII for a minimum of 150 exposure days

Exclusion criteria

• Inhibitors to FVIII (current evidence or history)
• Any other inherited or acquired bleeding disorder in addition to Hemophilia A
• Platelet count < 100,000/mm3
• Creatinine > 2x upper limit of normal or AST/ALT (aspartate aminotransferase/alanine aminotransferase) > 5x upper limit of normal

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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145 participants in 4 patient groups

Arm 1

Experimental group

Description:

On-demand treatment of BAY94-9027 at individual dose and number of infusions based upon location and severity of bleeds

Treatment:

Biological: BAY94-9027

Arm 2

Experimental group

Description:

Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by 2 infusions per week over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED

Treatment:

Biological: BAY94-9027

Arm 3

Experimental group

Description:

Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by infusion every 5 days over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED

Treatment:

Biological: BAY94-9027

Arm 4

Experimental group

Description:

Prophylaxis treatment of BAY94-9027; 2 infusions per week over 10 weeks followed by infusion every 7 days over 26 weeks in the main trial; and at least 1 day per week in the extension for at least 100 ED

Treatment:

Biological: BAY94-9027