Hemophilia APhase 1MATCHBOXNCT01921855
Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors
A Phase 1 study testing BAY Factor VII (BAY86-6150) in people with Hemophilia A and Hemophilia B, run by Bayer at 4 sites in 3 countries.
Trial summary
This is the first in humans study of BAY86-6150 (B0189) in non-bleeding subjects with moderate or severe congenital hemophilia A or B with or without inhibitors. This is a randomized, double-blind, placebo-controlled, single-dose, dose escalation study. Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- What is being tested?
- BAY Factor VII (BAY86-6150)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 16 participants
- Where
- 4 sitesin 3 countries
- Study dates
- Jan 2009to Dec 2009
- Phase
- Phase 1
- 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 18 to 65 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- History of moderate or severe congenital hemophilia A or B with or without inhibitors to Factor VIII (FVIII) or Factor IX (FIX)
- Male subjects 18-65 years of age inclusive
- Able to dismiss factor replacement therapy during the course of the study unless required for the treatment of an acute bleeding episode
- Written informed consent
- Willing and able to comply with the requirements of the protocol
- Have adequate venous access
- Willing to use an effective method of contraception until Day 30 of their study participation
Who cannot join
- Received factor replacement therapy or treatment with any other procoagulant therapeutics, or any antifibrinolytic agents, including blood products, at anytime within 5 days prior to administration of investigational medicinal product (IMP)
- Planned administration of factor replacement therapy or treatment with any other procoagulant therapeutics or any antifibrinolytic agents, including blood products, at anytime during the study period
- Acute bleeding episode or any ongoing bleeding episode at any time within 7 days prior to administration IMP
- Clinically relevant coagulation disorder other than congenital hemophilia A or B
- History of angina or receiving treatment for angina
- History of coronary atherosclerotic disease, disseminated intravascular coagulopathy, or stage 2 hypertension defined as systolic blood pressure (SBP) >/= 160 mmHg or diastolic blood pressure (DBP) >/= 90 mmHg
- History of transient ischemic attack, stroke, myocardial infarction, coronary artery disease, congestive heart failure, or thromboembolic event
- Active infection on day of IMP administration or septicemia at any time within 30 days prior to administration of IMP
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
South Africa2 sites
Study site
Bloemfontein, Freestate, 9300
Study site
Johannesburg, Gauteng, 2193
About this study
Official title: A Phase I, Randomized, Double-blind, Placebo Controlled, Single Dose Escalation Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors
This is the first in humans study of BAY86-6150 (B0189) in non-bleeding subjects with moderate or severe congenital hemophilia A or B with or without inhibitors. This is a randomized, double-blind, placebo-controlled, single-dose, dose escalation study. It is designed to investigate the safety, tolerability, potential immunogenicity, pharmacokinetic and pharmacodynamic profile of BAY86-6150 (B0189) and to determine a dose or range of doses to be examined in subsequent studies.
- Conditions
- Hemophilia A, Hemophilia B
- Being tested
- BAY Factor VII (BAY86-6150), Placebo
- 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.
BAY Factor VII (BAY86-6150)Drug
BAY Factor VII (BAY86-6150), 6.5 µg/kg body weight, will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.
BAY Factor VII (BAY86-6150)Drug
BAY Factor VII (BAY86-6150), 20 µg/kg body weight, will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.
BAY Factor VII (BAY86-6150)Drug
BAY Factor VII (BAY86-6150), 50 µg/kg body weight, will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.
BAY Factor VII (BAY86-6150)Drug
BAY Factor VII (BAY86-6150), 90 µg/kg body weight, will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.
PlaceboDrug
Placebo will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1.
| Study group | Role | What they receive |
|---|---|---|
| BAY Factor VII (6.5 µg/kg) / Placebo | Experimental | n = 4, randomized 3:1; 6.5 µg/kg BAY 86-6150 (B0189):Placebo Receives: Drug: BAY Factor VII (BAY86-6150); Drug: Placebo |
| BAY Factor VII (20 µg/kg) / Placebo | Experimental | n = 4, randomized 3:1; 20 µg/kg BAY 86-6150 (B0189):Placebo Receives: Drug: BAY Factor VII (BAY86-6150); Drug: Placebo |
| BAY Factor VII (50 µg/kg) / Placebo | Experimental | n = 4, randomized 3:1; 50 µg/kg BAY 86-6150 (B0189):Placebo Receives: Drug: BAY Factor VII (BAY86-6150); Drug: Placebo |
| BAY Factor VII (90 µg/kg) / Placebo | Experimental | n = 4, randomized 3:1; 90 µg/kg BAY 86-6150 (B0189):Placebo Receives: Drug: BAY Factor VII (BAY86-6150); Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with adverse events as a measure of safety and tolerability
Measured: Up to Day 50
Secondary outcomes (3)
Pharmacokinetic assessment, based on plasma concentration of BAY86-6150
Measured: 9 time points from pre-dosing on Day 1 up to 48 hours post-dosing
Pharmacodynamic assessment, based on plasma hemostasis marker level
Measured: 9 time points from pre-dosing on Day 1 up to 48 hours post-dosing
Immunogenicity assessment, based on anti-BAY86-6150 binding antibody levels
Measured: 3 time points from pre-dosing on Day 1 up to Day 50
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2013 (estimated)
- Study startJan 2009
- Primary completionDec 2009
- Study completionDec 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia AStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 16 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
- NCT01921855
- Sponsor's protocol ID
- 13787
- Other IDs
- 2008-000117-29, 2008
- Acronym
- MATCHBOX
- Status last verified
- Aug 2014
- First submitted
- 10 Jul 2013
- Last updated
- 21 Aug 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Mahlangu JN, Coetzee MJ, Laffan M, Windyga J, Yee TT, Schroeder J, Haaning J, Siegel JE, Lemm G. Phase I, randomized, double-blind, placebo-controlled, single-dose escalation study of the recombinant factor VIIa variant BAY 86-6150 in hemophilia. J Thromb Haemost. 2012 May;10(5):773-80. doi: 10.1111/j.1538-7836.2012.04667.x.
PubMed 22353395
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Randomized, Double-blind, Placebo Controlled, Single Dose Escalation Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors
- ClinicalTrials.gov ID
- NCT01921855
- Acronym / sponsor ID
- MATCHBOX / 13787
- Conditions
- Hemophilia A, Hemophilia B
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- Jan 2009
- Primary completion
- Dec 2009 (actual)
- Study completion
- Dec 2009 (actual)
- First posted
- 13 Aug 2013 (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.
This is the first in humans study of BAY86-6150 (B0189) in non-bleeding subjects with moderate or severe congenital hemophilia A or B with or without inhibitors. This is a randomized, double-blind, placebo-controlled, single-dose, dose escalation study. It is designed to investigate the safety, tolerability, potential immunogenicity, pharmacokinetic and pharmacodynamic profile of BAY86-6150 (B0189) and to determine a dose or range of doses to be examined in subsequent studies.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| BAY Factor VII (6.5 µg/kg) / Placebo | Experimental | Drug: BAY Factor VII (BAY86-6150), Drug: Placebon = 4, randomized 3:1; 6.5 µg/kg BAY 86-6150 (B0189):Placebo |
| BAY Factor VII (20 µg/kg) / Placebo | Experimental | Drug: BAY Factor VII (BAY86-6150), Drug: Placebon = 4, randomized 3:1; 20 µg/kg BAY 86-6150 (B0189):Placebo |
| BAY Factor VII (50 µg/kg) / Placebo | Experimental | Drug: BAY Factor VII (BAY86-6150), Drug: Placebon = 4, randomized 3:1; 50 µg/kg BAY 86-6150 (B0189):Placebo |
| BAY Factor VII (90 µg/kg) / Placebo | Experimental | Drug: BAY Factor VII (BAY86-6150), Drug: Placebon = 4, randomized 3:1; 90 µg/kg BAY 86-6150 (B0189):Placebo |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- History of moderate or severe congenital hemophilia A or B with or without inhibitors to Factor VIII (FVIII) or Factor IX (FIX)
- Male subjects 18-65 years of age inclusive
- Able to dismiss factor replacement therapy during the course of the study unless required for the treatment of an acute bleeding episode
- Written informed consent
- Willing and able to comply with the requirements of the protocol
Key exclusion criteria (8 listed)
- Received factor replacement therapy or treatment with any other procoagulant therapeutics, or any antifibrinolytic agents, including blood products, at anytime within 5 days prior to administration of investigational medicinal product (IMP)
- Planned administration of factor replacement therapy or treatment with any other procoagulant therapeutics or any antifibrinolytic agents, including blood products, at anytime during the study period
- Acute bleeding episode or any ongoing bleeding episode at any time within 7 days prior to administration IMP
- Clinically relevant coagulation disorder other than congenital hemophilia A or B
- History of angina or receiving treatment for angina
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 |
|---|---|---|
| BAY Factor VII (BAY86-6150)BAY Factor VII (BAY86-6150), 6.5 µg/kg body weight, will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1. | Drug | BAY Factor VII (6.5 µg/kg) / Placebo |
| BAY Factor VII (BAY86-6150)BAY Factor VII (BAY86-6150), 20 µg/kg body weight, will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1. | Drug | BAY Factor VII (20 µg/kg) / Placebo |
| BAY Factor VII (BAY86-6150)BAY Factor VII (BAY86-6150), 50 µg/kg body weight, will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1. | Drug | BAY Factor VII (50 µg/kg) / Placebo |
| BAY Factor VII (BAY86-6150)BAY Factor VII (BAY86-6150), 90 µg/kg body weight, will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1. | Drug | BAY Factor VII (90 µg/kg) / Placebo |
| PlaceboPlacebo will be administered as a slow intravenous (i.v.) administration over a period of 2-5 minutes (min) on Study Day 1. | Drug | BAY Factor VII (20 µg/kg) / Placebo, BAY Factor VII (50 µg/kg) / Placebo, BAY Factor VII (6.5 µg/kg) / Placebo, BAY Factor VII (90 µg/kg) / Placebo |
Primary outcome
Number of participants with adverse events as a measure of safety and tolerability
Time frame: Up to Day 50
3 secondary outcomes
Pharmacokinetic assessment, based on plasma concentration of BAY86-6150
Time frame: 9 time points from pre-dosing on Day 1 up to 48 hours post-dosing
Pharmacodynamic assessment, based on plasma hemostasis marker level
Time frame: 9 time points from pre-dosing on Day 1 up to 48 hours post-dosing
Immunogenicity assessment, based on anti-BAY86-6150 binding antibody levels
Time frame: 3 time points from pre-dosing on Day 1 up to Day 50
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 21 Aug 2014.
- Results publication: PubMed 22353395Mahlangu JN, Coetzee MJ, Laffan M, Windyga J, Yee TT, Schroeder J, Haaning J, Siegel JE, Lemm G. Phase I, randomized, double-blind, placebo-controlled, single-dose escalation study of the recombinant factor VIIa variant BAY 86-6150 in hemophilia. J Thromb Haemost. 2012 May;10(5):773-80. doi: 10.1111/j.1538-7836.2012.04667.x.
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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
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BAY Factor VII (BAY86-6150) in people with Hemophilia A and Hemophilia B, run by Bayer at 4 sites in 3 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 7 inclusion and 8 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 21 Aug 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in South Africa, Poland, United Kingdom. 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 phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Number of participants with adverse events as a measure of safety and tolerability (time frame: Up to Day 50). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01921855, which also lists 1 linked publication.
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 21 Aug 2014.