Hemophilia A, Hemophilia B Clinical Trial
Official title:
A Phase 2/3, Multicenter, Open-label Clinical Study to Assess the Safety and Efficacy of BAY86-6150 in Subjects With Hemophilia A or B With Inhibitors, Composed of 2 Parts (A & B). Part A: Sequential Cohorts of Four Dose Levels of the Modified rFVIIa BAY86-6150 Assessed in a Non-controlled Dose Response Design in Acutely Bleeding Subjects and for PK/ PD in an Intra-individual Crossover Design Compared With One Fixed Dose of Eptacog Alfa in Non-bleeding Subjects. Part B: Confirmatory Study to Further Investigate the Efficacy and Safety of BAY86-6150
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.
| Status | Completed |
| Enrollment | 10 |
| Est. completion date | March 2014 |
| Est. primary completion date | March 2014 |
| Accepts healthy volunteers | No |
| Gender | Male |
| Age group | 12 Years to 62 Years |
| Eligibility |
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 |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment
| Country | Name | City | State |
|---|---|---|---|
| n/a | |||
| Lead Sponsor | Collaborator |
|---|---|
| Bayer |
United States, Australia, Brazil, Bulgaria, Chile, China, Colombia, Denmark, France, Germany, Hungary, India, Israel, Italy, Japan, Korea, Republic of, Mexico, Netherlands, New Zealand, Poland, Romania, Russian Federation, Singapore, South Africa, Sweden, Taiwan, Turkey, Ukraine, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Successful treatments of bleeding episodes. | A bleed was defined as successfully treated, if no administration of rescue medication was required. | 10 hours after each bleed | No |
| Primary | Proportion of successful treatments of bleeding episodes on subject level. | 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. | 10 hours after each bleed | No |
| Secondary | Time to stop the bleed | 10 hours after each bleed | No | |
| Secondary | Number of injections needed to stop the bleeding episode. | 10 hours after each bleed | No | |
| Secondary | Effectiveness of treatment as rated by the subject's assessment (very effective, effective, partially effective, not effective). | 10 hours after each bleed | No | |
| Secondary | Participant's reported outcome as assessed by Euro QoL (EQ-5D). | 14 days after last exposure to BAY86-6150 | No | |
| Secondary | Participant's reported outcome as assessed by Brief Pain Inventory. | 7 days after last exposure to BAY86-6150 | No | |
| Secondary | Participant's reported outcome as assessed by Work Productivity and Activity Impairment Questionaire. | 14 days after last exposure to BAY86-6150 | No |