Hemophilia A and BPhase 2NCT03597022
Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors
A Phase 2 study testing Befovacimab (BAY1093884) in people with Hemophilia A and B, run by Bayer at 17 sites in 11 countries.
Trial summary
The purpose of this study was to assess the safety and tolerability of multiple doses of a human monoclonal antibody (BAY1093884) given under the skin in subjects with hemophilia A or B. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A and B
- What is being tested?
- Befovacimab (BAY1093884)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 17 sitesin 11 countries
- Study dates
- 24 Jul 2018to 15 Oct 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male severe hemophilic patients with undetectable FVIII activity <1% or FIX activity <2%, with or without inhibitors (any titer) are eligible.
- Subjects with a past history of inhibitors (any inhibitor titer) are eligible.
- Age ≥18 years.
- Documentation of ≥4 bleeding episodes (any type or location of bleeds, treated or not) within the 6 months prior to screening.
- For subjects on prophylaxis: Willingness to interrupt ongoing prophylaxis.
- For subjects on immune tolerance induction (ITI): Willingness to interrupt ongoing ITI.
Who cannot join
- History of any other coagulation disorder (particularly disseminated intravascular coagulopathy or combined FVIII/FV deficiency) or platelet disorder.
- History of diseases related to venous thromboembolic events (e.g., pulmonary embolism, deep vein thrombosis, thrombophlebitis) or thrombotic microangiopathy.
- Risk factors for venous or arterial diseases (e.g., uncontrolled hypertension, uncontrolled diabetes).
- History of cardiac, coronary and/or arterial peripheral atherosclerotic disease
- Platelet count <100,000/μL.
- Human immunodeficiency virus (HIV) infection with a cluster of differentiation 4 (CD4+) lymphocyte count of <200/mm^3
Trial locations and contacts
17 sites in 11 countries.
17 sites in 11 countries
Bulgaria3 sites
Medical centre Hipokrat - N EOOD
Plovdiv, 4000
SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
Sofia, 1756
MHAT Sveta Marina EAD
Varna, 9010
United Kingdom3 sites
University Hospital of Wales
Cardiff, CF14 4XW
Royal Free Hospital
London, NW3 2QG
Manchester Royal Infirmary
Manchester, M13 9WL
France2 sites
Hôpital Louis Pradel - Bron
Bron, 69500
Hôpital Robert Debré - Reims Cedex
Reims, 51092
Japan2 sites
Hiroshima University Hospital
Hiroshima, 734-8551
Ogikubo Hospital
Suginami, Tokyo, 167-0035
Italy1 site
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Lombardy, 20122
New Zealand1 site
Haematology Service, Canterbury Health Laboratories
Christchurch, 8011
About this study
The purpose of this study was to assess the safety and tolerability of multiple doses of a human monoclonal antibody (BAY1093884) given under the skin in subjects with hemophilia A or B. This antibody was intended to protect from bleeds by inhibiting a substance (Tissue Factor Pathway Inhibitor, TFPI) that reduces the ability of the body to form blood clots.
Read the detailed description
The primary objective of the study was to assess the safety and tolerability of multiple subcutaneous injections of BAY1093884 (anti-TFPI monoclonal antibody, immunoglobulin G2, IgG2) in patients with hemophilia A or B with or without inhibitors.
- Conditions
- Hemophilia A and B
- Being tested
- Befovacimab (BAY1093884)
- Keywords
- Subcutaneous, Prophylaxis, Non-Inhibitors, Inhibitors
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Befovacimab (BAY1093884)Drug
Also called Anti-TFPI (Tissue Factor Pathway Inhibitor) monoclonal antibody (immunoglobulin G2; IgG2)
Once weekly doses until premature termination of the study, subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| BAY1093884 100mg | Experimental | Subjects received BAY1093884 100 mg once a week until premature termination of the study Receives: Drug: Befovacimab (BAY1093884) |
| BAY1093884 225mg | Experimental | Subjects received BAY1093884 225 mg once a week until premature termination of the study Receives: Drug: Befovacimab (BAY1093884) |
| BAY1093884 400mg | Experimental | Subjects received BAY1093884 400mg once a week until premature termination of the study Receives: Drug: Befovacimab (BAY1093884) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Drug-related Treatment-emergent Adverse Events
An adverse event (AE) was any untoward medical occurrence (i.e., any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a participant in the study. Any bleeding event occurring during the study was not documented as an AE because this event was planned to be captured in the assessment of efficacy. AEs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as treatment-emergent AEs (TEAEs). Drug-related TEAEs were TEAEs that had "reasonable causal relationship" to the study treatment decided by the investigators.
Measured: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
Number of Participants With Serious Treatment-emergent Adverse Events
A serious adverse event (SAE) was any untoward medical occurrence that at any dose was resulting in death, was lifethreatening, requires hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity. SAEs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as serious treatment-emergent AEs (TESAEs). Drug-related TESAEs were TESAEs that had "reasonable causal relationship" to the study treatment decided by the investigators.
Measured: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
Number of Participants With Treatment-emergent Adverse Events of Special Interest
Any thromboembolic or thrombotic microangiopathic event or any hypersensitivity reaction was an adverse event of special interest (AESI). AESIs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as treatment-emergent AESIs.
Measured: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
Number of Participants With Clinically Relevant Abnormalities in Laboratory Values
"Clinically relevant "implied the presence of a clinical sign or symptom that required medical action.
Measured: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jul 2018
- Study start24 Jul 2018
- Primary completion15 Oct 2019
- Study completion15 Oct 2019
Why it stopped: Due to thrombosis
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hemophilia A and 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03597022
- Sponsor's protocol ID
- 19580
- Other IDs
- 2017-003324-67, 2017
- Status last verified
- Nov 2020
- First submitted
- 13 Jul 2018
- Last updated
- 30 Nov 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors
- ClinicalTrials.gov ID
- NCT03597022
- Acronym / sponsor ID
- 19580
- Conditions
- Hemophilia A and B
- Keywords
- Subcutaneous, Prophylaxis, Non-Inhibitors, Inhibitors
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Due to thrombosis
- Enrolment
- 24 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 24 Jul 2018 (actual)
- Primary completion
- 15 Oct 2019 (actual)
- Study completion
- 15 Oct 2019 (actual)
- First posted
- 24 Jul 2018 (actual)
- Results
- Posted 30 Nov 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study was to assess the safety and tolerability of multiple doses of a human monoclonal antibody (BAY1093884) given under the skin in subjects with hemophilia A or B. This antibody was intended to protect from bleeds by inhibiting a substance (Tissue Factor Pathway Inhibitor, TFPI) that reduces the ability of the body to form blood clots.
Detailed description
The primary objective of the study was to assess the safety and tolerability of multiple subcutaneous injections of BAY1093884 (anti-TFPI monoclonal antibody, immunoglobulin G2, IgG2) in patients with hemophilia A or B with or without inhibitors.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| BAY1093884 100mg | Experimental | Drug: Befovacimab (BAY1093884)Subjects received BAY1093884 100 mg once a week until premature termination of the study |
| BAY1093884 225mg | Experimental | Drug: Befovacimab (BAY1093884)Subjects received BAY1093884 225 mg once a week until premature termination of the study |
| BAY1093884 400mg | Experimental | Drug: Befovacimab (BAY1093884)Subjects received BAY1093884 400mg once a week until premature termination of the study |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male severe hemophilic patients with undetectable FVIII activity <1% or FIX activity <2%, with or without inhibitors (any titer) are eligible.
- Subjects with a past history of inhibitors (any inhibitor titer) are eligible.
- Age ≥18 years.
- Documentation of ≥4 bleeding episodes (any type or location of bleeds, treated or not) within the 6 months prior to screening.
- For subjects on prophylaxis: Willingness to interrupt ongoing prophylaxis.
Key exclusion criteria (6 listed)
- History of any other coagulation disorder (particularly disseminated intravascular coagulopathy or combined FVIII/FV deficiency) or platelet disorder.
- History of diseases related to venous thromboembolic events (e.g., pulmonary embolism, deep vein thrombosis, thrombophlebitis) or thrombotic microangiopathy.
- Risk factors for venous or arterial diseases (e.g., uncontrolled hypertension, uncontrolled diabetes).
- History of cardiac, coronary and/or arterial peripheral atherosclerotic disease
- Platelet count <100,000/μL.
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 |
|---|---|---|
| Befovacimab (BAY1093884)Also: Anti-TFPI (Tissue Factor Pathway Inhibitor) monoclonal antibody (immunoglobulin G2; IgG2)Once weekly doses until premature termination of the study, subcutaneous injection | Drug | BAY1093884 100mg, BAY1093884 225mg, BAY1093884 400mg |
Primary outcome
Number of Participants With Drug-related Treatment-emergent Adverse Events
Time frame: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
An adverse event (AE) was any untoward medical occurrence (i.e., any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a participant in the study. Any bleeding event occurring during the study was not documented as an AE because this event was planned to be captured in the assessment of efficacy. AEs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as treatment-emergent AEs (TEAEs). Drug-related TEAEs were TEAEs that had "reasonable causal relationship" to the study treatment decided by the investigators.
Number of Participants With Serious Treatment-emergent Adverse Events
Time frame: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
A serious adverse event (SAE) was any untoward medical occurrence that at any dose was resulting in death, was lifethreatening, requires hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity. SAEs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as serious treatment-emergent AEs (TESAEs). Drug-related TESAEs were TESAEs that had "reasonable causal relationship" to the study treatment decided by the investigators.
Number of Participants With Treatment-emergent Adverse Events of Special Interest
Time frame: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
Any thromboembolic or thrombotic microangiopathic event or any hypersensitivity reaction was an adverse event of special interest (AESI). AESIs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as treatment-emergent AESIs.
Number of Participants With Clinically Relevant Abnormalities in Laboratory Values
Time frame: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
"Clinically relevant "implied the presence of a clinical sign or symptom that required medical action.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Click here to find information about studies related to Bayer Healthcare products conducted in EuropeLink provided by the sponsor.
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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.
The record lists no contact. Use the official record to reach the sponsor.
17 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Befovacimab (BAY1093884) in people with Hemophilia A and B, run by Bayer at 17 sites in 11 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 6 inclusion and 6 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: stopped early, as of the sponsor's last update on 30 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Bulgaria, United Kingdom, France, Japan, Australia and 6 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Nov 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Drug-related Treatment-emergent Adverse Events (time frame: After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days). The study lists 4 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 30 Nov 2020.