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

Primary Purpose

Hemophilia A and B

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Befovacimab (BAY1093884)
Sponsored by
Bayer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A and B focused on measuring Subcutaneous, Prophylaxis, Non-Inhibitors, Inhibitors

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)MaleDoes not accept 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

Sites / Locations

  • Fiona Stanley Hospital
  • Universitätsklinikum AKH Wien
  • Medical centre Hipokrat - N EOOD
  • SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
  • MHAT Sveta Marina EAD
  • Hôpital Louis Pradel - Bron
  • Hôpital Robert Debré - Reims Cedex
  • Pecsi Tudomanyegyetem Klinikai Kozpont
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
  • Ogikubo Hospital
  • Hiroshima University Hospital
  • Eulji University Hospital
  • Haematology Service, Canterbury Health Laboratories
  • Changhua Christian Hospital
  • University Hospital of Wales
  • Royal Free Hospital
  • Manchester Royal Infirmary

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

BAY1093884 100mg

BAY1093884 225mg

BAY1093884 400mg

Arm Description

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

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

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

Outcomes

Primary Outcome Measures

Number of Participants With Drug-related Treatment-emergent Adverse Events
An adverse event (AE) was any untoward medical occurrence (i.e., any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a participant in the study. Any bleeding event occurring during the study was not documented as an AE because this event was planned to be captured in the assessment of efficacy. AEs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as treatment-emergent AEs (TEAEs). Drug-related TEAEs were TEAEs that had "reasonable causal relationship" to the study treatment decided by the investigators.
Number of Participants With Serious Treatment-emergent Adverse Events
A serious adverse event (SAE) was any untoward medical occurrence that at any dose was resulting in death, was lifethreatening, requires hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity. SAEs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as serious treatment-emergent AEs (TESAEs). Drug-related TESAEs were TESAEs that had "reasonable causal relationship" to the study treatment decided by the investigators.
Number of Participants With Treatment-emergent Adverse Events of Special Interest
Any thromboembolic or thrombotic microangiopathic event or any hypersensitivity reaction was an adverse event of special interest (AESI). AESIs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as treatment-emergent AESIs.
Number of Participants With Clinically Relevant Abnormalities in Laboratory Values
"Clinically relevant "implied the presence of a clinical sign or symptom that required medical action.

Secondary Outcome Measures

Full Information

First Posted
July 13, 2018
Last Updated
November 26, 2020
Sponsor
Bayer
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1. Study Identification

Unique Protocol Identification Number
NCT03597022
Brief Title
Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors
Official Title
Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors
Study Type
Interventional

2. Study Status

Record Verification Date
November 2020
Overall Recruitment Status
Terminated
Why Stopped
Due to thrombosis
Study Start Date
July 24, 2018 (Actual)
Primary Completion Date
October 15, 2019 (Actual)
Study Completion Date
October 15, 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
Yes

5. Study Description

Brief Summary
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.
Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A and B
Keywords
Subcutaneous, Prophylaxis, Non-Inhibitors, Inhibitors

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
24 (Actual)

8. Arms, Groups, and Interventions

Arm Title
BAY1093884 100mg
Arm Type
Experimental
Arm Description
Subjects received BAY1093884 100 mg once a week until premature termination of the study
Arm Title
BAY1093884 225mg
Arm Type
Experimental
Arm Description
Subjects received BAY1093884 225 mg once a week until premature termination of the study
Arm Title
BAY1093884 400mg
Arm Type
Experimental
Arm Description
Subjects received BAY1093884 400mg once a week until premature termination of the study
Intervention Type
Drug
Intervention Name(s)
Befovacimab (BAY1093884)
Other Intervention Name(s)
Anti-TFPI (Tissue Factor Pathway Inhibitor) monoclonal antibody (immunoglobulin G2; IgG2)
Intervention Description
Once weekly doses until premature termination of the study, subcutaneous injection
Primary Outcome Measure Information:
Title
Number of Participants With Drug-related Treatment-emergent Adverse Events
Description
An adverse event (AE) was any untoward medical occurrence (i.e., any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a participant in the study. Any bleeding event occurring during the study was not documented as an AE because this event was planned to be captured in the assessment of efficacy. AEs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as treatment-emergent AEs (TEAEs). Drug-related TEAEs were TEAEs that had "reasonable causal relationship" to the study treatment decided by the investigators.
Time Frame
After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
Title
Number of Participants With Serious Treatment-emergent Adverse Events
Description
A serious adverse event (SAE) was any untoward medical occurrence that at any dose was resulting in death, was lifethreatening, requires hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity. SAEs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as serious treatment-emergent AEs (TESAEs). Drug-related TESAEs were TESAEs that had "reasonable causal relationship" to the study treatment decided by the investigators.
Time Frame
After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
Title
Number of Participants With Treatment-emergent Adverse Events of Special Interest
Description
Any thromboembolic or thrombotic microangiopathic event or any hypersensitivity reaction was an adverse event of special interest (AESI). AESIs occurring after the first administration of study drug and up to and including 30 days after the last administration of study drug were defined as treatment-emergent AESIs.
Time Frame
After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days
Title
Number of Participants With Clinically Relevant Abnormalities in Laboratory Values
Description
"Clinically relevant "implied the presence of a clinical sign or symptom that required medical action.
Time Frame
After the first administration of study drug and up to and including 30 days after the last administration of study drug, with an average of 183 days

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
Facility Name
Fiona Stanley Hospital
City
Murdoch
State/Province
Western Australia
ZIP/Postal Code
6150
Country
Australia
Facility Name
Universitätsklinikum AKH Wien
City
Wien
ZIP/Postal Code
1090
Country
Austria
Facility Name
Medical centre Hipokrat - N EOOD
City
Plovdiv
ZIP/Postal Code
4000
Country
Bulgaria
Facility Name
SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
City
Sofia
ZIP/Postal Code
1756
Country
Bulgaria
Facility Name
MHAT Sveta Marina EAD
City
Varna
ZIP/Postal Code
9010
Country
Bulgaria
Facility Name
Hôpital Louis Pradel - Bron
City
Bron
ZIP/Postal Code
69500
Country
France
Facility Name
Hôpital Robert Debré - Reims Cedex
City
Reims Cedex
ZIP/Postal Code
51092
Country
France
Facility Name
Pecsi Tudomanyegyetem Klinikai Kozpont
City
Pecs
ZIP/Postal Code
7624
Country
Hungary
Facility Name
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
City
Milano
State/Province
Lombardia
ZIP/Postal Code
20122
Country
Italy
Facility Name
Ogikubo Hospital
City
Suginami
State/Province
Tokyo
ZIP/Postal Code
167-0035
Country
Japan
Facility Name
Hiroshima University Hospital
City
Hiroshima
ZIP/Postal Code
734-8551
Country
Japan
Facility Name
Eulji University Hospital
City
Daejeon
ZIP/Postal Code
35233
Country
Korea, Republic of
Facility Name
Haematology Service, Canterbury Health Laboratories
City
Christchurch
ZIP/Postal Code
8011
Country
New Zealand
Facility Name
Changhua Christian Hospital
City
Changhua
ZIP/Postal Code
50006
Country
Taiwan
Facility Name
University Hospital of Wales
City
Cardiff
ZIP/Postal Code
CF14 4XW
Country
United Kingdom
Facility Name
Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Manchester Royal Infirmary
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom

12. IPD Sharing Statement

Links:
URL
https://clinicaltrials.bayer.com/
Description
Click here to find results for studies related to Bayer products
URL
http://www.clinicaltrialsregister.eu/
Description
Click here to find information about studies related to Bayer Healthcare products conducted in Europe

Learn more about this trial

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

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