A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02571569
• Date of registration: 07/10/2015
• Prospective Registration: Yes
• Primary sponsor: Bayer
• Public title: A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors
• Scientific 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
• Date of first enrolment: October 28, 2015
• Target sample size: 32
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02571569
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Basic Science. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

Bulgaria, Germany, Japan, Ukraine, United Kingdom

Contacts

• Name: Bayer Study Director
• Affiliation: Bayer

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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

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 (clinically relevant) allergies, non-allergic drug reactions, or
multiple drug allergies



Age minimum: 18 Years
Age maximum: 65 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Hemophilia A

Hemophilia B

Intervention(s)

Drug: BAY1093884

Primary Outcome(s)

Plasma levels of anti-BAY1093884 antibodies [Time Frame: Pre-dose, Day 14, 21,28, 43 and 56]

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]

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]

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]

Number of participants (single dose cohors) with adverse events as measure of safety and tolerability [Time Frame: Up to 56 days]

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]

Secondary Outcome(s)

Number of participants (multiple dose cohort) with adverse events as a measure of safety and tolerability [Time Frame: Up to 77 days]

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]

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/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]

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 levels of anti-BAY1093884 antibodies (multiple dose cohort) [Time Frame: Pre-dose, Day 14, 28, 49 and 77]

Tissue factor pathway inhibitor (TFPI) activity [Time Frame: Up to 77 days]

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]

Secondary ID(s)

16144

2014-003283-20

Source(s) of Monetary Support

Please refer to primary and secondary sponsors