Hemophilia A, Hemophilia BPhase 2/3NCT01625390
A Phase 2/ 3 Trial to Evaluate the Efficacy and Safety of BAY86-6150
A Phase 2/3 study testing BAY86-6150 and eptacog alfa [activated] in people with Hemophilia A, Hemophilia B, run by Bayer at 61 sites in 29 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 62 years, men only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- What is being tested?
- BAY86-6150 and eptacog alfa [activated]
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 61 sitesin 29 countries
- Study dates
- Jun 2012to Mar 2014
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 62 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 9Show fewer
- Stroke
- Congestive heart failure
- Thromboembolic event
Trial locations and contacts
61 sites in 29 countries.
61 sites in 29 countries
India4 sites
Study site
Bangalore, 34
Study site
Hyderabad, Andhra Pradesh, 500034
Study site
Ludhiana, Punjab, 141008
Study site
Pune, 411004
Mexico4 sites
Study site
Guadalajara, Jalisco
Study site
México D. F., 04530
Study site
Oaxaca City, Oaxaca, 68000
Study site
San Luis Potosí City, San Luis Potosí, 78216
Russia4 sites
Study site
Khabarovsk, 680009
Study site
Saint Petersburg, 191186
Study site
Samara, 443079
Study site
Yekaterinburg, 620149
Brazil3 sites
Study site
Rio de Janeiro, Rio de Janeiro, 20211030
Study site
São Paulo, São Paulo, 01401901
Study site
São Paulo, São Paulo, 04023-061
China3 sites
Study site
Beijing, 100730
Study site
Guangzhou, Guangdong, 510515
Study site
Tianjin
Japan3 sites
Study site
Kashihara, Nara, 634-8522
Study site
Shinjuku-ku, Tokyo, 160-0023
Study site
Suginami, Tokyo, 167-0035
Romania3 sites
Study site
Bucharest, 022328
Study site
Bucharest, 11026
Study site
Timișoara, Timiș County, 300011
South Africa3 sites
Study site
Bloemfontein, Freestate
Study site
Johannesburg, Gauteng, 2132
Study site
Pretoria, Gauteng, 0001
Taiwan3 sites
Study site
Changhua, 500
Study site
Taipei, Taipei, 10016
Study site
Taipei, 110
Ukraine3 sites
Study site
Donetsk, 83045
Study site
Lviv, 79044
Study site
Odesa, 65025
United States2 sites
Study site
Chattanooga, Tennessee, 37403
Study site
Sacramento, California, 95817
Germany2 sites
Study site
Mainz, Rhineland-Palatinate, 55131
Study site
Villingen-Schwenningen, Baden-Wurttemberg, 78050
Turkey (Türkiye)2 sites
Study site
Istanbul, 34098
Study site
Izmir, 35100
United Kingdom2 sites
Study site
London, SE1 7EH
Study site
Truro, TR1 3LJ
About this study
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.
Read more
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.
- Conditions
- Hemophilia A, Hemophilia B
- Being tested
- BAY86-6150, eptacog alfa [activated]
- Keywords
- hemophilia, haemophilia, inhibitor, FVIIa, Factor VII activated, bypassing
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BAY86-6150Drug
Four dose levels (6.5 µg/kg, 20 µg/kg, 50 µg/kg and 90 µg/kg) of BAY86-6150 will be studied.
eptacog alfa [activated]Drug
comparative PK/PD (pharmacokinetics/pharmacodynamics) evaluation
BAY86-6150Drug
Confirmation of recommended dose of BAY86-6150 to be evaluated further as determined in Part A.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 | Experimental | Not described Receives: Drug: BAY86-6150 |
| Arm 2 | Active comparator | Not described Receives: Drug: eptacog alfa [activated] |
| Arm 3 | Experimental | Not described Receives: Drug: BAY86-6150 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Successful treatments of bleeding episodes.
A bleed was defined as successfully treated, if no administration of rescue medication was required.
Measured: 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.
Measured: 10 hours after each bleed
Secondary outcomes (6)
Time to stop the bleed
Measured: 10 hours after each bleed
Number of injections needed to stop the bleeding episode.
Measured: 10 hours after each bleed
Effectiveness of treatment as rated by the subject's assessment (very effective, effective, partially effective, not effective).
Measured: 10 hours after each bleed
Participant's reported outcome as assessed by Euro QoL (EQ-5D).
Measured: 14 days after last exposure to BAY86-6150
Participant's reported outcome as assessed by Brief Pain Inventory.
Measured: 7 days after last exposure to BAY86-6150
Participant's reported outcome as assessed by Work Productivity and Activity Impairment Questionaire.
Measured: 14 days after last exposure to BAY86-6150
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2012 (estimated)
- Study startJun 2012
- Primary completionMar 2014
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A, Hemophilia BStudies of the same condition, open and under way.
- More trials run by BayerThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bayer Study Director, Study director, Bayer
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01625390
- Sponsor's protocol ID
- 15534
- Other IDs
- 2011-000323-33, U1111-1133-2156
- Status last verified
- Jun 2015
- First submitted
- 19 Jun 2012
- Last updated
- 1 Jul 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 1 Jul 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01625390
- Acronym / sponsor ID
- 15534
- Conditions
- Hemophilia A, Hemophilia B
- Keywords
- hemophilia, haemophilia, inhibitor, FVIIa, Factor VII activated, bypassing
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- Jun 2012
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 21 Jun 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Arm 1 | Experimental | Drug: BAY86-6150 |
| Arm 2 | Active comparator | Drug: eptacog alfa [activated] |
| Arm 3 | Experimental | Drug: BAY86-6150 |
Who may qualify
- Ages
- 12 to 62 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BAY86-6150Four dose levels (6.5 µg/kg, 20 µg/kg, 50 µg/kg and 90 µg/kg) of BAY86-6150 will be studied. | Drug | Arm 1 |
| eptacog alfa [activated]comparative PK/PD (pharmacokinetics/pharmacodynamics) evaluation | Drug | Arm 2 |
| BAY86-6150Confirmation of recommended dose of BAY86-6150 to be evaluated further as determined in Part A. | Drug | Arm 3 |
Primary outcome
Successful treatments of bleeding episodes.
Time frame: 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.
Time frame: 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.
6 secondary outcomes
Time to stop the bleed
Time frame: 10 hours after each bleed
Number of injections needed to stop the bleeding episode.
Time frame: 10 hours after each bleed
Effectiveness of treatment as rated by the subject's assessment (very effective, effective, partially effective, not effective).
Time frame: 10 hours after each bleed
Participant's reported outcome as assessed by Euro QoL (EQ-5D).
Time frame: 14 days after last exposure to BAY86-6150
Participant's reported outcome as assessed by Brief Pain Inventory.
Time frame: 7 days after last exposure to BAY86-6150
Participant's reported outcome as assessed by Work Productivity and Activity Impairment Questionaire.
Time frame: 14 days after last exposure to BAY86-6150
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bayer Study Director study director
Bayer
61 sites in 29 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing BAY86-6150 and eptacog alfa [activated] in people with Hemophilia A, Hemophilia B, run by Bayer at 61 sites in 29 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 Jul 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 61 sites in India, Mexico, Russia, Brazil, China and 24 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Successful treatments of bleeding episodes. (time frame: 10 hours after each bleed). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2015.