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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03597022




Registration number
NCT03597022
Ethics application status
Date submitted
13/07/2018
Date registered
24/07/2018
Date last updated
30/11/2020

Titles & IDs
Public title
Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors
Scientific title
Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors
Secondary ID [1] 0 0
2017-003324-67
Secondary ID [2] 0 0
19580
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A and B 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Befovacimab (BAY1093884)

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

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

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


Treatment: Drugs: Befovacimab (BAY1093884)
Once weekly doses until premature termination of the study, subcutaneous injection
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Drug-related Treatment-emergent Adverse Events
Timepoint [1] 0 0
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
Primary outcome [2] 0 0
Number of Participants With Serious Treatment-emergent Adverse Events
Timepoint [2] 0 0
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
Primary outcome [3] 0 0
Number of Participants With Treatment-emergent Adverse Events of Special Interest
Timepoint [3] 0 0
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
Primary outcome [4] 0 0
Number of Participants With Clinically Relevant Abnormalities in Laboratory Values
Timepoint [4] 0 0
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

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/07/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
15/10/2019
Sample size
Target
Accrual to date
Final
24
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
Austria
State/province [1] 0 0
Wien
Country [2] 0 0
Bulgaria
State/province [2] 0 0
Plovdiv
Country [3] 0 0
Bulgaria
State/province [3] 0 0
Sofia
Country [4] 0 0
Bulgaria
State/province [4] 0 0
Varna
Country [5] 0 0
France
State/province [5] 0 0
Bron
Country [6] 0 0
France
State/province [6] 0 0
Reims Cedex
Country [7] 0 0
Hungary
State/province [7] 0 0
Pecs
Country [8] 0 0
Italy
State/province [8] 0 0
Lombardia
Country [9] 0 0
Japan
State/province [9] 0 0
Tokyo
Country [10] 0 0
Japan
State/province [10] 0 0
Hiroshima
Country [11] 0 0
Korea, Republic of
State/province [11] 0 0
Daejeon
Country [12] 0 0
New Zealand
State/province [12] 0 0
Christchurch
Country [13] 0 0
Taiwan
State/province [13] 0 0
Changhua
Country [14] 0 0
United Kingdom
State/province [14] 0 0
Cardiff
Country [15] 0 0
United Kingdom
State/province [15] 0 0
London
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Bayer
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT03597022
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03597022