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)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Bayer
ClinicalTrials.gov Identifier:
NCT01233258
First received: November 2, 2010
Last updated: January 13, 2014
Last verified: January 2014

November 2, 2010
January 13, 2014
January 2011
December 2012   (final data collection date for primary outcome measure)
Annualized Number of All Bleeds [ Time Frame: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design) ] [ Designated as safety issue: No ]
The annualized number of bleeds experienced by participants
Annualized number of all bleeds [ Time Frame: 12 months ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01233258 on ClinicalTrials.gov Archive Site
  • Annualized Number of All Bleeds During CS/EP Period [ Time Frame: Up to 6 months (6 months on CS/EP potency assignment) ] [ Designated as safety issue: No ]
    The annualized number of bleeds experienced by participants while they were taking rFVIII (BAY81-8973) assayed by CS/EP
  • Annualized Number of All Bleeds During CS/ADJ Period [ Time Frame: Up to 6 months (6 months on CS/ADJ potency assignment) ] [ Designated as safety issue: No ]
    The annualized number of bleeds experienced by participants while they were taking rFVIII (BAY81-8973) assayed by CS/ADJ
  • Percentage of Bleeds Per Participant Controlled With ≤ 2 Injections in Participants Treated on Demand With rFVIII (BAY81-8973) [ Time Frame: Up to 12 months (6 months per mode of potency assignment according to the randomized cross-over design) ] [ Designated as safety issue: No ]
    The percentage of bleeds per participant on on-demand treatment that stopped after two or fewer injections
  • Bleeding rates by dose [ Time Frame: 12 months ] [ Designated as safety issue: No ]
  • Response to treatment of bleeds as measured by questionnaire [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Number of Bleeds During Treatment [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    The number of bleeds experienced by each participant
  • Number of Participants With Inhibitory Antibody Formation [ Time Frame: 3, 6, 9 and 12 months after baseline ] [ Designated as safety issue: Yes ]
    A test to ensure that participants have not developed antibodies that will interfere with the action of rFVIII (BAY81-8973)
Not Provided
 
A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A
A Phase II/III, Randomized, Cross-over, Open-label Trial to Demonstrate Superiority of Prophylaxis Over On-demand Therapy in Previously Treated Subjects With Severe Hemophilia A Treated With Plasma Protein-free Recombinant FVIII Formulated With Sucrose (BAY 81-8973)

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.

Not Provided
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Open Label
Primary Purpose: Treatment
Hemophilia A
  • Biological: rFVIII (BAY81-8973) on demand
    Participants received on-demand treatment with rFVIII (BAY81-8973) assayed by CS/EP (Chromogenic Substrate Assay per European Pharmacopoeia) for 6 months and by CS/ADJ (Chromogenic Substrate Assay/label adjusted to one-stage assay) for 6 months, sequence according to randomization
  • Biological: rFVIII (BAY81-8973) prophylaxis low-dose
    Participants received low dose prophylaxis treatment at 20, 25 or 30 IU/kg twice per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.
  • Biological: rFVIII (BAY81-8973) prophylaxis high-dose
    Participants received high dose prophylaxis treatment at 30, 35 or 40 IU/kg 3 times per week with rFVIII (BAY81-8973) assayed by CS/EP for 6 months and by CS/ADJ for 6 months, sequence according to randomization.
  • Experimental: Arm 1: rFVIII on demand first CS/EP then CS/ADJ
    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.
    Intervention: Biological: rFVIII (BAY81-8973) on demand
  • Experimental: Arm 2: rFVIII on demand first CS/ADJ then CS/EP
    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.
    Intervention: Biological: rFVIII (BAY81-8973) on demand
  • Experimental: Arm 3: rFVIII prophylaxis low-dose first CS/EP then CS/ADJ
    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.
    Intervention: Biological: rFVIII (BAY81-8973) prophylaxis low-dose
  • Experimental: Arm 4: rFVIII prophylaxis low-dose first CS/ADJ then CS/EP
    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.
    Intervention: Biological: rFVIII (BAY81-8973) prophylaxis low-dose
  • Experimental: Arm 5: rFVIII prophylaxis high-dose first CS/EP then CS/ADJ
    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.
    Intervention: Biological: rFVIII (BAY81-8973) prophylaxis high-dose
  • Experimental: Arm 6: rFVIII prophylaxis high-dose first CS/ADJ then CS/EP
    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.
    Intervention: Biological: rFVIII (BAY81-8973) prophylaxis high-dose
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
80
December 2012
December 2012   (final data collection date for primary outcome measure)

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
Male
12 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Argentina,   China,   Colombia,   Czech Republic,   India,   Indonesia,   Japan,   Mexico,   Romania,   Russian Federation,   Serbia,   Slovakia,   South Africa,   Taiwan,   Thailand,   Turkey,   Ukraine
 
NCT01233258
14319, 2009-012150-20
Yes
Bayer
Bayer
Not Provided
Study Director: Bayer Study Director Bayer
Bayer
January 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP