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

Bayer

Status and phase: Completed; Phase 1

Conditions: Hemophilia B; Hemophilia A

Treatments: Drug: BAY1093884

Study type: Interventional

Funder types: Industry

Identifiers

NCT02571569

2014-003283-20 (EudraCT Number)

16144

Details and patient eligibility

About

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.

Enrollment: 32 patients

Sex: Male

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Basic Science

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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32 participants in 3 patient groups

Without inhibitors

Experimental group

Description:

Dose escalation steps for participants without inhibitors - intravenous infusion and subcutaneous injection

Treatment:

Drug: BAY1093884

Drug: BAY1093884

With inhibitors

Experimental group

Description:

Dose escalation steps for participants with inhibitors - intravenous infusion and subcutaneous injection

Treatment:

Drug: BAY1093884

Drug: BAY1093884

Without inhibitors_multiple dose

Experimental group

Description:

Multiple dose cohort for participants without inhibitors - a single subcutaneous injection once a week for 6 weeks

Treatment:

Drug: BAY1093884

Drug: BAY1093884