A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150
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
3 other identifiers
interventional
10
29 countries
61
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jun 2012
61 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
June 1, 2012
CompletedFirst Submitted
Initial submission to the registry
June 19, 2012
CompletedFirst Posted
Study publicly available on registry
June 21, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2014
CompletedJuly 1, 2015
June 1, 2015
1.7 years
June 19, 2012
June 4, 2015
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
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
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
Secondary Outcomes (6)
Time to stop the bleed
10 hours after each bleed
Number of injections needed to stop the bleeding episode.
10 hours after each bleed
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
Participant's reported outcome as assessed by Brief Pain Inventory.
7 days after last exposure to BAY86-6150
- +1 more secondary outcomes
Study Arms (3)
Arm 1
EXPERIMENTALArm 2
ACTIVE COMPARATORArm 3
EXPERIMENTALInterventions
Four dose levels (6.5 µg/kg, 20 µg/kg, 50 µg/kg and 90 µg/kg) of BAY86-6150 will be studied.
Eligibility Criteria
You may qualify if:
- Male subjects
- to 62 years-of-age
- History of moderate or severe congenital hemophilia A or B with inhibitors to FVIII or FIX
- or more bleeding episodes in the last 6 months before enrollment.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Bayerlead
Study Sites (61)
Unknown Facility
Sacramento, California, 95817, United States
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Chattanooga, Tennessee, 37403, United States
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Melbourne, Victoria, Australia
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Rio de Janeiro, Rio de Janeiro, 20211030, Brazil
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São Paulo, São Paulo, 01401901, Brazil
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São Paulo, São Paulo, 04023-061, Brazil
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Sofia, 1756, Bulgaria
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Santiago, 836-0156, Chile
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Guangzhou, Guangdong, 510515, China
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Beijing, 100730, China
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Tianjin, China
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Barranquilla, Atlántico, Colombia
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Bogotá, Colombia
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Aarhus N, 8200, Denmark
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Lyon, 69437, France
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Tours, 37044, France
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Villingen-Schwenningen, Baden-Wurttemberg, 78050, Germany
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Mainz, Rhineland-Palatinate, 55131, Germany
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Budapest, 1134, Hungary
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Debrecen, 4032, Hungary
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Hyderabad, Andhra Pradesh, 500034, India
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Ludhiana, Punjab, 141008, India
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Bangalore, 34, India
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Pune, 411004, India
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Tel Litwinsky, 5262000, Israel
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Florence, 50134, Italy
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Milan, 20122, Italy
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Kashihara, Nara, 634-8522, Japan
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Shinjuku-ku, Tokyo, 160-0023, Japan
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Suginami, Tokyo, 167-0035, Japan
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Guadalajara, Jalisco, Mexico
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Oaxaca City, Oaxaca, 68000, Mexico
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San Luis Potosí City, San Luis Potosí, 78216, Mexico
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México D. F., 04530, Mexico
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Utrecht, 3508 GA, Netherlands
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Christchurch, 8011, New Zealand
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Warsaw, 02-776, Poland
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Timișoara, Timiș County, 300011, Romania
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Bucharest, 022328, Romania
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Bucharest, 11026, Romania
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Khabarovsk, 680009, Russia
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Saint Petersburg, 191186, Russia
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Samara, 443079, Russia
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Yekaterinburg, 620149, Russia
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Singapore, 119228, Singapore
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Singapore, 169608, Singapore
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Bloemfontein, Freestate, South Africa
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Johannesburg, Gauteng, 2132, South Africa
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Pretoria, Gauteng, 0001, South Africa
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Seoul, 134-727, South Korea
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Gothenburg, 413 45, Sweden
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Taipei, Taipei, 10016, Taiwan
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Changhua, 500, Taiwan
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Taipei, 110, Taiwan
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Istanbul, 34098, Turkey (Türkiye)
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Izmir, 35100, Turkey (Türkiye)
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Donetsk, 83045, Ukraine
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Lviv, 79044, Ukraine
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Odesa, 65025, Ukraine
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London, SE1 7EH, United Kingdom
Unknown Facility
Truro, TR1 3LJ, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Bayer Study Director
Bayer
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
June 19, 2012
First Posted
June 21, 2012
Study Start
June 1, 2012
Primary Completion
March 1, 2014
Study Completion
March 1, 2014
Last Updated
July 1, 2015
Record last verified: 2015-06