Brief Title
A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A
Official Title
A Phase II/III, Multicenter, Partially Randomized, Open Label Trial Investigating Safety and Efficacy of On-demand and Prophylactic Treatment With BAY94-9027 in Severe Hemophilia A
Brief Summary
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.
Detailed 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.
Study Phase
Phase 2/Phase 3
Study Type
Interventional
Primary Outcome
Annualized Number of Total Bleeds in On-demand Treatment Arm (Weeks 0 -36) and in Each Prophylaxis Arm (Weeks 10 - 36, Excluding Rescue Bleeds) - Part A, Main Trial
Secondary Outcome
Annualized Number of Joint Bleeds, Trauma, Spontaneous Bleeds in On-demand Treatment Arm (Weeks 0 -36) and in Each Prophylaxis Arm (Weeks 10 - 36, Excluding Rescue Bleeds) - Part A
Condition
Hemophilia A
Intervention
BAY94-9027
Study Arms / Comparison Groups
Arm 1
Description: On-demand treatment of BAY94-9027 at individual dose and number of infusions based upon location and severity of bleeds
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
145
Start Date
April 23, 2012
Completion Date
November 21, 2019
Primary Completion Date
June 13, 2014
Eligibility Criteria
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
Gender
Male
Ages
12 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Bayer Study Director, ,
Location Countries
Austria
Location Countries
Austria
Administrative Informations
NCT ID
NCT01580293
Organization ID
13024
Secondary IDs
2011-005210-11
Responsible Party
Sponsor
Study Sponsor
Bayer
Study Sponsor
Bayer Study Director, Study Director, Bayer
Verification Date
November 2020