Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors

Bayer

Status and phase: Terminated; Phase 2

Conditions: Hemophilia A and B

Treatments: Drug: Befovacimab (BAY1093884)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03597022

2017-003324-67 (EudraCT Number)

19580

Details and patient eligibility

About

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.

Full 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.

Enrollment: 24 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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.

Exclusion criteria

• 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 design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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24 participants in 3 patient groups

BAY1093884 100mg

Experimental group

Description:

Subjects received BAY1093884 100 mg once a week until premature termination of the study

Treatment:

Drug: Befovacimab (BAY1093884)

BAY1093884 225mg

Experimental group

Description:

Subjects received BAY1093884 225 mg once a week until premature termination of the study

Treatment:

Drug: Befovacimab (BAY1093884)

BAY1093884 400mg

Experimental group

Description:

Subjects received BAY1093884 400mg once a week until premature termination of the study

Treatment:

Drug: Befovacimab (BAY1093884)