A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A (Leopold II)

Bayer

Status and phase: Completed; Phase 3

Conditions: Hemophilia A

Treatments: Biological: rFVIII (BAY81-8973) on demand; Biological: rFVIII (BAY81-8973) prophylaxis low-dose; Biological: rFVIII (BAY81-8973) prophylaxis high-dose

Study type: Interventional

Funder types: Industry

Identifiers

NCT01233258

2009-012150-20 (EudraCT Number)

14319

Details and patient eligibility

About

The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A. The hypothesis is that prophylaxis will result in fewer bleeds than on-demand treatment.

Enrollment: 80 patients

Sex: Male

Ages: 12 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male, aged 12 to 65 years
• Severe hemophilia A
• History of more than 150 exposure days (ED) with clotting factor concentrates
• Currently receiving episodic treatment with FVIII; no regular prophylaxis for more than 6 consecutive months in the past 5 years
• No current Factor VIII inhibitor or history of inhibitor
• Willing to use electronic patient diary

Exclusion criteria

• Presence of another bleeding disease that is different from hemophilia A
• Thrombocytopenia
• Abnormal renal function
• Presence of active liver disease
• Known hypersensitivity to FVIII

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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80 participants in 6 patient groups

Arm 1: rFVIII on demand first CS/EP then CS/ADJ

Experimental group

Description:

Participants received on-demand treatment with recombinant factor VIII (rFVIII, BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months, followed by cross-over to study drug assayed by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months.

Treatment:

Biological: rFVIII (BAY81-8973) on demand

Arm 2: rFVIII on demand first CS/ADJ then CS/EP

Experimental group

Description:

Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/ADJ for 6 months, followed by cross-over to study drug assayed by CS/EP for 6 months.

Treatment:

Biological: rFVIII (BAY81-8973) on demand

Arm 3: rFVIII prophylaxis low-dose first CS/EP then CS/ADJ

Experimental group

Description:

Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII(BAY81-8973) measured by CS/ EP for 6 months then crossed over to study drug measured by CS/ADJ for 6 months.

Treatment:

Biological: rFVIII (BAY81-8973) prophylaxis low-dose

Arm 4: rFVIII prophylaxis low-dose first CS/ADJ then CS/EP

Experimental group

Description:

Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) measured by CS/ADJ for 6 months then crossed over to study drug measured by CS/ EP for 6 months.

Treatment:

Biological: rFVIII (BAY81-8973) prophylaxis low-dose

Arm 5: rFVIII prophylaxis high-dose first CS/EP then CS/ADJ

Experimental group

Description:

Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) measured by CS/ EP for 6 months then crossed over to study drug measured by CS/ADJ for 6 months.

Treatment:

Biological: rFVIII (BAY81-8973) prophylaxis high-dose

Arm 6: rFVIII prophylaxis high-dose first CS/ADJ then CS/EP

Experimental group

Description:

Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII(BAY81-8973) measured by CS/ADJ for 6 months then crossed over to study drug measured by CS/ EP for 6 months.

Treatment:

Biological: rFVIII (BAY81-8973) prophylaxis high-dose