A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150
- Conditions
- Hemophilia A, Hemophilia B
- Interventions
- Drug: eptacog alfa [activated]Drug: BAY86-6150
- Registration Number
- NCT01625390
- Lead Sponsor
- Bayer
- Brief Summary
Haemophilia is a disorder, usually genetic, affecting mostly male individuals, in which one of the proteins needed to form blood clots (FVIII) is missing or not present in sufficient levels. In a person with haemophilia, the clotting process is much slower and the person experiences bleeding episodes that can result in serious problems and potential disability.
The current haemophilia standard of care is to maintain FVIII activity level above 1%. Sometimes, patients can develop antibodies (so called "inhibitors") against FVIII and it is no longer effective at controlling bleeds. Bleeds in these patients are currently treated using other proteins involved in the clotting process.
The purpose of this study is to investigate how effectively BAY86-6150 may stop acute bleeds in "inhibitor" patients. This study consists of two parts, A and B. The purpose of part A is to find the most effective yet tolerable out of four doses of BAY86-6150 with regard to efficacy and safety (dose-finding part). Part A is expected to last 9 - 29 months. The purpose of part B is to confirm efficacy and safety of the dose found in part A in all participating patients (confirmatory part). Part B is expected to last 12-32 months.
Approximately 60 male subjects 12 to 62 years-of-age with moderate or severe haemophilia A or B, with inhibitors to FVIII or FIX, who have had 4 or more bleeding episodes in the last 6 months, will participate in this study.
Patient's bleeds will be treated with BAY86-6150 and with a rescue medication if no response is made to BAY86-6150. Patients will attend the treatment centre at regular intervals and be required to keep an electronic diary.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 10
- Male subjects
- 12 to 62 years-of-age
- History of moderate or severe congenital hemophilia A or B with inhibitors to FVIII or FIX
- 4 or more bleeding episodes in the last 6 months before enrollment.
- Clinically relevant coagulation disorder other than congenital hemophilia A or B with inhibitors
- History of coronary and/or peripheral atherosclerotic disease
- Disseminated intravascular coagulopathy, or stage 2 hypertension
- Angina pectoris
- Myocardial infarction
- Transient ischemic attack
- Stroke
- Congestive heart failure
- Thromboembolic event
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- CROSSOVER
- Arm && Interventions
Group Intervention Description Arm 2 eptacog alfa [activated] - Arm 1 BAY86-6150 - Arm 3 BAY86-6150 -
- Primary Outcome Measures
Name Time Method Proportion of successful treatments of bleeding episodes on subject level. 10 hours after each bleed Proportion of successful treatments of bleeding episodes was calculated as number of bleeding episodes treated successfully - without rescue medication - divided by the total number of bleeding episodes on a dose level.
Successful treatments of bleeding episodes. 10 hours after each bleed A bleed was defined as successfully treated, if no administration of rescue medication was required.
- Secondary Outcome Measures
Name Time Method Participant's reported outcome as assessed by Brief Pain Inventory. 7 days after last exposure to BAY86-6150 Participant's reported outcome as assessed by Work Productivity and Activity Impairment Questionaire. 14 days after last exposure to BAY86-6150 Time to stop the bleed 10 hours after each bleed Number of injections needed to stop the bleeding episode. 10 hours after each bleed Participant's reported outcome as assessed by Euro QoL (EQ-5D). 14 days after last exposure to BAY86-6150 Effectiveness of treatment as rated by the subject's assessment (very effective, effective, partially effective, not effective). 10 hours after each bleed