Hemophilia A, Hemophilia B
Conditions
Keywords
hemophilia, haemophilia, inhibitor, FVIIa, Factor VII activated, bypassing
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.
Interventions
DRUGBAY86-6150
Four dose levels (6.5 µg/kg, 20 µg/kg, 50 µg/kg and 90 µg/kg) of BAY86-6150 will be studied.
DRUGeptacog alfa [activated]
comparative PK/PD (pharmacokinetics/pharmacodynamics) evaluation
Sponsors
Bayer
Study design
Allocation
NON_RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to 62 Years
Healthy volunteers
No
Inclusion criteria
* 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.
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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. |
Secondary
| Measure | Time frame |
|---|---|
| Effectiveness of treatment as rated by the subject's assessment (very effective, effective, partially effective, not effective). | 10 hours after each bleed |
| Participant's reported outcome as assessed by Euro QoL (EQ-5D). | 14 days after last exposure to BAY86-6150 |
| Time to stop the bleed | 10 hours after each bleed |
| Participant's reported outcome as assessed by Work Productivity and Activity Impairment Questionaire. | 14 days after last exposure to BAY86-6150 |
| Participant's reported outcome as assessed by Brief Pain Inventory. | 7 days after last exposure to BAY86-6150 |
| Number of injections needed to stop the bleeding episode. | 10 hours after each bleed |
Countries
Australia, Brazil, Bulgaria, Chile, China, Colombia, Denmark, France, Germany, Hungary, India, Israel, Italy, Japan, Mexico, Netherlands, New Zealand, Poland, Romania, Russia, Singapore, South Africa, South Korea, Sweden, Taiwan, Turkey (Türkiye), Ukraine, United Kingdom, United States
Outcome results
None listed