Back to resultsA Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia
Primary Purpose
Hemophilia A; Hemophilia B
Status
Completed
Phase
Phase 1
Locations
Israel
Study Type
Interventional
Intervention
BAY1093884
BAY1093884
Sponsored by
About this trial
This is an interventional basic science trial for Hemophilia A; Hemophilia B
Eligibility Criteria
Inclusion Criteria:
- Males with severe congenital hemophilia A or B defined as <1% FVIII or <2% FIX concentration by measurement at the time of screening or from reliable prior documentation (e.g., measurement in other clinical Bayer trials, or diagnostic genetic testing)
- Male with any inhibitor titer at screening or prior to screening at any time from medical records. Subjects may be receiving a bypassing agent (rFVIIa; NovoSeven and/or aPCC; FEIBA) for treatment.
- Age: 18 to 65 years at screening
- BMI: 18 to 29.9 kg/m2
Exclusion Criteria:
- 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 severe allergies, non-allergic drug reactions, or multiple drug allergies
- Subjects with inhibitors treated with FEIBA, who are not willing to accept rFVIIa (NovoSeven) for the treatment of any bleeds occurring either between screening and dosing or after study drug administration, and until the end of the study.
Sites / Locations
- Chaim Sheba Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
BAY1093884 in subjects with Hemophilia
Arm Description
Single dose of BAY1093884 over 30 minutes administered in subjects with severe congenital Hemophilia A or B, with inhibitors or without inhibitors
Outcomes
Primary Outcome Measures
AUC (0-tlast) of BAY1093884 in plasma
Area under the concentration vs. time curve from time 0 to the last data point > LLOQ
AUC(0-tlast)/D of BAY1093884 in plasma
AUC(0-tlast) divided by dose
Cmax of BAY1093884 in plasma
Maximum observed drug concentration in measured matrix after single dose administration
Cmax/D of BAY1093884 in plasma
Cmax divided by dose
Secondary Outcome Measures
Tissue factor plasma inhibitor activity: effect of BAY1093884 to inhibit the anticoagulatory activity of plasma TFPI as assessed by a chromogenic assay
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03481946
Brief Title
A Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia
Official Title
A Phase 1 Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia
Study Type
Interventional
2. Study Status
Record Verification Date
September 2020
Overall Recruitment Status
Completed
Study Start Date
May 10, 2018 (Actual)
Primary Completion Date
October 17, 2018 (Actual)
Study Completion Date
February 20, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Bayer
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No
5. Study Description
Brief Summary
The primary objective of this study is to assess the pharmacokinetics in patients with severe hemophilia.
The secondary objective is to assess the pharmacodynamics of BAY1093884 based on tissue factor pathway inhibitor activity
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A; Hemophilia B
7. Study Design
Primary Purpose
Basic Science
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
BAY1093884 in subjects with Hemophilia
Arm Type
Experimental
Arm Description
Single dose of BAY1093884 over 30 minutes administered in subjects with severe congenital Hemophilia A or B, with inhibitors or without inhibitors
Intervention Type
Drug
Intervention Name(s)
BAY1093884
Intervention Description
0.3 mg/kg given intravenously
Intervention Type
Drug
Intervention Name(s)
BAY1093884
Intervention Description
1 mg/kg given intravenously
Primary Outcome Measure Information:
Title
AUC (0-tlast) of BAY1093884 in plasma
Description
Area under the concentration vs. time curve from time 0 to the last data point > LLOQ
Time Frame
Up to 15 days after drug administration
Title
AUC(0-tlast)/D of BAY1093884 in plasma
Description
AUC(0-tlast) divided by dose
Time Frame
Up to 15 days after drug administration
Title
Cmax of BAY1093884 in plasma
Description
Maximum observed drug concentration in measured matrix after single dose administration
Time Frame
Up to 15 days after drug administration
Title
Cmax/D of BAY1093884 in plasma
Description
Cmax divided by dose
Time Frame
Up to 15 days after drug administration
Secondary Outcome Measure Information:
Title
Tissue factor plasma inhibitor activity: effect of BAY1093884 to inhibit the anticoagulatory activity of plasma TFPI as assessed by a chromogenic assay
Time Frame
Up to 15 days after drug administration
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Males with severe congenital hemophilia A or B defined as <1% FVIII or <2% FIX concentration by measurement at the time of screening or from reliable prior documentation (e.g., measurement in other clinical Bayer trials, or diagnostic genetic testing)
Male with any inhibitor titer at screening or prior to screening at any time from medical records. Subjects may be receiving a bypassing agent (rFVIIa; NovoSeven and/or aPCC; FEIBA) for treatment.
Age: 18 to 65 years at screening
BMI: 18 to 29.9 kg/m2
Exclusion Criteria:
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 severe allergies, non-allergic drug reactions, or multiple drug allergies
Subjects with inhibitors treated with FEIBA, who are not willing to accept rFVIIa (NovoSeven) for the treatment of any bleeds occurring either between screening and dosing or after study drug administration, and until the end of the study.
Facility Information:
Facility Name
Chaim Sheba Medical Center
City
Ramat Gan
ZIP/Postal Code
5262000
Country
Israel
12. IPD Sharing Statement
Learn more about this trial
A Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia
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