Clinical trial NCT03597022: 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
Overall status: TERMINATED
Phase: PHASE2
Conditions: Hemophilia A and B
Keywords: Subcutaneous
Prophylaxis
Non-Inhibitors
Inhibitors
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2018-07-24
Enrollment: 24
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Number of Participants With Drug-related Treatment-emergent Adverse Events
timeFrame: 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
measure: Number of Participants With Serious Treatment-emergent Adverse Events
timeFrame: 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
measure: Number of Participants With Treatment-emergent Adverse Events of Special Interest
timeFrame: 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
measure: Number of Participants With Clinically Relevant Abnormalities in Laboratory Values
timeFrame: 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
Secondary outcomes:
Other outcomes:
Locations:
city: Murdoch
country: Australia
facility: Fiona Stanley Hospital
state: Western Australia
city: Wien
country: Austria
facility: Universitätsklinikum AKH Wien
city: Plovdiv
country: Bulgaria
facility: Medical centre Hipokrat - N EOOD
city: Sofia
country: Bulgaria
facility: SHATHD Spec. Hospi. for Active Treatm. of Haematol. Dis. EAD
city: Varna
country: Bulgaria
facility: MHAT Sveta Marina EAD
city: Bron
country: France
facility: Hôpital Louis Pradel - Bron
city: Reims Cedex
country: France
facility: Hôpital Robert Debré - Reims Cedex
city: Pecs
country: Hungary
facility: Pecsi Tudomanyegyetem Klinikai Kozpont
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
state: Lombardia
city: Suginami
country: Japan
facility: Ogikubo Hospital
state: Tokyo
city: Hiroshima
country: Japan
facility: Hiroshima University Hospital
city: Daejeon
country: Korea, Republic of
facility: Eulji University Hospital
city: Christchurch
country: New Zealand
facility: Haematology Service, Canterbury Health Laboratories
city: Changhua
country: Taiwan
facility: Changhua Christian Hospital
city: Cardiff
country: United Kingdom
facility: University Hospital of Wales
city: London
country: United Kingdom
facility: Royal Free Hospital
city: Manchester
country: United Kingdom
facility: Manchester Royal Infirmary
Study officials:
References: