Hemophilia APhase 1NCT02571569
A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors
A Phase 1 study testing BAY1093884 in people with Hemophilia A and Hemophilia B, run by Bayer at 10 sites in 5 countries.
Trial summary
Investigate the safety, tolerability and pharmacokinetics of BAY1093884 after Intravenous (IV) and subcutaneous (SC) administration of increasing single doses and SC administration of multiple doses. 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?
- BAY1093884
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- 10 sitesin 5 countries
- Study dates
- 28 Oct 2015to 11 Oct 2018
- 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
- Males with severe congenital Hemophilia A or B defined as <1% FVIII or Factor IX (FIX) concentration by measurement at the time of screening or from reliable prior documentation
- For subjects in Cohorts I-IV, I-SC1 and I-SC2; If history of inhibitors is evident, inhibitor titer of ≥5 Bethesda Units (BU) at screening or prior to screening at any time from medical records.
- Age: 18 to 65 years of age at screening
- Body mass index (BMI): 18 to 29.9 kg/m²
Who cannot join
- Subjects with known bleeding disorders (such as von Willebrand factor [vWF] deficiency, FXI deficiency, platelet disorders, or known acquired or inherited thrombophilia etc.) other than congenital Hemophilia A or B with or without inhibitors
- History of angina pectoris or treatment for angina pectoris
- History of coronary and/or peripheral atherosclerotic disease, congestive heart failure, disseminated intravascular coagulopathy, or stage 2 hypertension defined as systolic blood pressure (SBP) ≥160 mmHg or diastolic blood pressure (DBP) ≥100 mmHg even if controlled
- History of thrombophlebitis, venous / arterial thromboembolic diseases (particularly deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction, cerebrovascular accident, ischemic heart disease, transient ischemic attack)
- Known or suspected hypersensitivity of the immune system, history of anaphylactic reaction, known (clinically relevant) allergies, non-allergic drug reactions, or multiple drug allergies
Trial locations and contacts
10 sites in 5 countries.
10 sites in 5 countries
Bulgaria3 sites
Study site
Plovdiv, 4002
Study site
Sofia, 1756
Study site
Varna, 9010
United Kingdom2 sites
Study site
London, NW3 2QG
Study site
Manchester, M13 9WL
About this study
Official title: A Phase 1, First in Man, Multicenter, Open Label, Single Escalating Dose Study of BAY1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors
Investigate the safety, tolerability and pharmacokinetics of BAY1093884 after Intravenous (IV) and subcutaneous (SC) administration of increasing single doses and SC administration of multiple doses.
- Conditions
- Hemophilia A, Hemophilia B
- Being tested
- BAY1093884
- 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.
BAY1093884Drug
Single escalating dose with a starting dose of 0.3 mg/kg for the first cohort. Drug will be administered via IV infusion over 1 hour and SC injection. Based on safety, PK and PD results the doses for the other cohorts will be determined.
BAY1093884Drug
Multiple dose cohort with a single 150-mg SC injection once a week for 6 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Without inhibitors | Experimental | Dose escalation steps for participants without inhibitors - intravenous infusion and subcutaneous injection Receives: Drug: BAY1093884 |
| With inhibitors | Experimental | Dose escalation steps for participants with inhibitors - intravenous infusion and subcutaneous injection Receives: Drug: BAY1093884 |
| Without inhibitors_multiple dose | Experimental | Multiple dose cohort for participants without inhibitors - a single subcutaneous injection once a week for 6 weeks Receives: Drug: BAY1093884 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants (single dose cohors) with adverse events as measure of safety and tolerability
Adverse events including abnormal laboratory findings and local injection site reactions
Measured: Up to 56 days
Plasma levels of anti-BAY1093884 antibodies
Measured: Pre-dose, Day 14, 21,28, 43 and 56
Plasma concentration of BAY1093884 characterized by AUC(0-tlast)
AUC from time 0 to the last data point > LLOQ (lower limit of quantitation)
Measured: Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
Plasma concentration of BAY1093884 characterized by AUC(0-tlast)/D
AUC(0-last) divided by dose
Measured: Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
Plasma concentration of BAY1093884 characterized by Cmax
Maximum observed drug concentration in measured matrix after single dose administration
Measured: Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
Plasma concentration of BAY1093884 characterized by Cmax/D
Cmax divided by dose
Measured: Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
Secondary outcomes (8)
Tissue factor pathway inhibitor (TFPI) activity
Measured: Up to 77 days
Number of participants (multiple dose cohort) with adverse events as a measure of safety and tolerability
Adverse events including abnormal laboratory findings and local injection site reactions
Measured: Up to 77 days
Plasma levels of anti-BAY1093884 antibodies (multiple dose cohort)
Measured: Pre-dose, Day 14, 28, 49 and 77
Plasma concentration of BAY1093884 characterized by AUC(0-7d and AUC(0-tau) (multiple dose cohort)
AUC from time 0 to 7d after first and last dose (AUC(0-tau)
Measured: Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
Plasma concentration of BAY1093884 characterized by AUC(0-7d/D and AUC(0-tau)/D after multiple dose
AUC(0-7d) after first dose and AUC(0-tau) after last dose divided by dose
Measured: Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
Plasma concentration of BAY1093884 characterized by Cmax after first dose and last dose (Cmax,md)
maximum observed drug concentration in measured matrix after first and last dose
Measured: Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
Plasma concentration of BAY1093884 characterized by Cmax/D after first dose and last dose (Cmax,md/D)
Cmax after first and last dose divided by dose
Measured: Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
Accumulation of BAY 1093884 in plasma as defined by ratio for Cmax and AUC (after first and last dose)
Cmax after last dose divided by Cmax after first dose, AUC after last dose divided by AUC after first dose
Measured: Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2015 (estimated)
- Study start28 Oct 2015
- Primary completion9 Jul 2018
- Study completion11 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 32 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
- NCT02571569
- Sponsor's protocol ID
- 16144
- Other IDs
- 2014-003283-20, 2014
- Status last verified
- Oct 2018
- First submitted
- 7 Oct 2015
- Last updated
- 18 Oct 2018
- Results posted
- No
- 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 18 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, First in Man, Multicenter, Open Label, Single Escalating Dose Study of BAY1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors
- ClinicalTrials.gov ID
- NCT02571569
- Acronym / sponsor ID
- 16144
- Conditions
- Hemophilia A, Hemophilia B
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 28 Oct 2015 (actual)
- Primary completion
- 9 Jul 2018 (actual)
- Study completion
- 11 Oct 2018 (actual)
- First posted
- 8 Oct 2015 (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.
Investigate the safety, tolerability and pharmacokinetics of BAY1093884 after Intravenous (IV) and subcutaneous (SC) administration of increasing single doses and SC administration of multiple doses.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Without inhibitors | Experimental | Drug: BAY1093884Dose escalation steps for participants without inhibitors - intravenous infusion and subcutaneous injection |
| With inhibitors | Experimental | Drug: BAY1093884Dose escalation steps for participants with inhibitors - intravenous infusion and subcutaneous injection |
| Without inhibitors_multiple dose | Experimental | Drug: BAY1093884Multiple dose cohort for participants without inhibitors - a single subcutaneous injection once a week for 6 weeks |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Males with severe congenital Hemophilia A or B defined as <1% FVIII or Factor IX (FIX) concentration by measurement at the time of screening or from reliable prior documentation
- For subjects in Cohorts I-IV, I-SC1 and I-SC2; If history of inhibitors is evident, inhibitor titer of ≥5 Bethesda Units (BU) at screening or prior to screening at any time from medical records.
- Age: 18 to 65 years of age at screening
- Body mass index (BMI): 18 to 29.9 kg/m²
Key exclusion criteria (5 listed)
- Subjects with known bleeding disorders (such as von Willebrand factor [vWF] deficiency, FXI deficiency, platelet disorders, or known acquired or inherited thrombophilia etc.) other than congenital Hemophilia A or B with or without inhibitors
- History of angina pectoris or treatment for angina pectoris
- History of coronary and/or peripheral atherosclerotic disease, congestive heart failure, disseminated intravascular coagulopathy, or stage 2 hypertension defined as systolic blood pressure (SBP) ≥160 mmHg or diastolic blood pressure (DBP) ≥100 mmHg even if controlled
- History of thrombophlebitis, venous / arterial thromboembolic diseases (particularly deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction, cerebrovascular accident, ischemic heart disease, transient ischemic attack)
- Known or suspected hypersensitivity of the immune system, history of anaphylactic reaction, known (clinically relevant) allergies, non-allergic drug reactions, or multiple drug allergies
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 |
|---|---|---|
| BAY1093884Single escalating dose with a starting dose of 0.3 mg/kg for the first cohort. Drug will be administered via IV infusion over 1 hour and SC injection. Based on safety, PK and PD results the doses for the other cohorts will be determined. | Drug | With inhibitors, Without inhibitors |
| BAY1093884Multiple dose cohort with a single 150-mg SC injection once a week for 6 weeks. | Drug | Without inhibitors_multiple dose |
Primary outcome
Number of participants (single dose cohors) with adverse events as measure of safety and tolerability
Time frame: Up to 56 days
Adverse events including abnormal laboratory findings and local injection site reactions
Plasma levels of anti-BAY1093884 antibodies
Time frame: Pre-dose, Day 14, 21,28, 43 and 56
Plasma concentration of BAY1093884 characterized by AUC(0-tlast)
Time frame: Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
AUC from time 0 to the last data point > LLOQ (lower limit of quantitation)
Plasma concentration of BAY1093884 characterized by AUC(0-tlast)/D
Time frame: Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
AUC(0-last) divided by dose
Plasma concentration of BAY1093884 characterized by Cmax
Time frame: Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
Maximum observed drug concentration in measured matrix after single dose administration
Plasma concentration of BAY1093884 characterized by Cmax/D
Time frame: Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
Cmax divided by dose
8 secondary outcomes
Tissue factor pathway inhibitor (TFPI) activity
Time frame: Up to 77 days
Number of participants (multiple dose cohort) with adverse events as a measure of safety and tolerability
Time frame: Up to 77 days
Plasma levels of anti-BAY1093884 antibodies (multiple dose cohort)
Time frame: Pre-dose, Day 14, 28, 49 and 77
Plasma concentration of BAY1093884 characterized by AUC(0-7d and AUC(0-tau) (multiple dose cohort)
Time frame: Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
Plasma concentration of BAY1093884 characterized by AUC(0-7d/D and AUC(0-tau)/D after multiple dose
Time frame: Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
Plasma concentration of BAY1093884 characterized by Cmax after first dose and last dose (Cmax,md)
Time frame: Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
Plasma concentration of BAY1093884 characterized by Cmax/D after first dose and last dose (Cmax,md/D)
Time frame: Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
Accumulation of BAY 1093884 in plasma as defined by ratio for Cmax and AUC (after first and last dose)
Time frame: Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
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 18 Oct 2018.
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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
10 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BAY1093884 in people with Hemophilia A and Hemophilia B, run by Bayer at 10 sites in 5 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 5 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 18 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in Bulgaria, Germany, Ukraine, United Kingdom, Japan. 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 (single dose cohors) with adverse events as measure of safety and tolerability (time frame: Up to 56 days). The study lists 6 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 18 Oct 2018.