Study to Test the Safety of an Investigational Drug Given Repeatedly to Adult Men With Severe Hemophilia

A Multicenter, Non-randomized, Open-label, Multiple Dose Escalation Study to Evaluate the Safety and Tolerability of Subcutaneous BAY1093884 in Males With Severe Hemophilia

The purpose of this study is to investigate the safety of a test drug to treat hemophilia in adult men.

Study Overview

• Status: Withdrawn
• Conditions: Hemophilia
• Intervention / Treatment: Drug: BAY1093884

Detailed Description

The primary objective is to assess the safety of multiple doses of BAY1093884.

• Study Type: Interventional
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

• Participants with hemophilia A and FVIII activity <1% or hemophilia B with FIX activity <2%
• Participants must be currently without inhibitors or with low titer inhibitors (inhibitor titer < 5 Bethesda units) who are receiving current on demand treatment with any FVIII or FIX (recombinant or plasma-derived; modified or unmodified)

Exclusion Criteria:

• History or at risk of developing diseases related to venous thromboembolic events (e.g., pulmonary embolism, deep vein thrombosis or thrombophlebitis)
• History of any other clinically relevant coagulation disorder (particularly disseminated intravascular coagulopathy or combined FVIII/Factor V deficiency) or platelet disorder
• History or at risk of developing cardiac, coronary and/or arterial peripheral atherosclerotic disease and/or arterial thromboembolic events, particularly myocardial infarction, cerebrovascular accident, stroke, transient ischemic attack, congestive heart failure, angina pectoris, treatment for angina pectoris or uncontrolled hypertension
• History or at risk for thrombotic microangiopathy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Hemophilia; Dose escalation starting with 200 mg of BAY1093884	Drug: BAY1093884; Drug administered via subcutaneous injections once weekly for 6 weeks (= 6 doses); Other Names: Human monoclonal immunoglobulin G2 antibody blocking the endogenous tissue factor pathway inhibitor (TFPI)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Frequency of drug-related adverse events		Up to 3 months
Frequency of drug-related serious adverse events		Up to 3 months
Frequency of adverse events of special interest	Adverse events of special interest comprise thromboembolic and thrombotic microangiopathy events, and hypersensitivity reactions.	Up to 3 months

Collaborators and Investigators

• Sponsor: Bayer

Study record dates

Study Major Dates

• Study Start (Anticipated): October 28, 2019
• Primary Completion (Anticipated): May 29, 2020
• Study Completion (Anticipated): July 31, 2020

Study Registration Dates

• First Submitted: June 21, 2019
• First Submitted That Met QC Criteria: June 21, 2019
• First Posted (Actual): June 24, 2019

Study Record Updates

• Last Update Posted (Actual): October 22, 2019
• Last Update Submitted That Met QC Criteria: October 21, 2019
• Last Verified: October 1, 2019

More Information

Terms related to this study

• Keywords: Coagulation Factor VIII (FVIII); Coagulation Factor IX (FIX); Anti-tissue factor pathway inhibitor (aTFPI); Human monoclonal immunoglobulin G2 antibody
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Fibrinolytic Agents; Fibrin Modulating Agents; Immunologic Factors; Protease Inhibitors; Factor Xa Inhibitors; Antithrombins; Serine Proteinase Inhibitors; Anticoagulants; Immunoglobulins; Antibodies, Monoclonal; Immunoglobulin G; Myeloma Proteins; Paraproteins; Lipoprotein-associated coagulation inhibitor
• Other Study ID Numbers: 20414; 2018-004566-34 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

IPD Plan Description

Availability of this study's data will be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access.

As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.

Interested researchers can use www.clinicalstudydatarequest.com to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the Study sponsors section of the portal.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No