Multiple escalating dose study of BAY 1093884 in adults with hemophilia A or B with or without inhibitors

Recruitment & Eligibility

Status: ot Recruiting

Sex: Not specified

Target Recruitment: 24

Inclusion Criteria

1. Male severe hemophilic patients with undetectable FVIII activity <1% or FIX activity <2%, with or without inhibitors (any titer) are eligible.
Note: subjects with a past history of inhibitors (any inhibitor titer) are eligible.
2. Age =18 years.
3. Documentation of =4 bleeding episodes (any type or location of bleeds, treated or not) within the 6 months prior to screening.
4. For subjects on prophylaxis: Willingness to interrupt ongoing prophylaxis.
5. For subjects on immune tolerance induction (ITI): Willingness to interrupt ongoing ITI.
6. Signed informed consent.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 22
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

1. History of any other coagulation disorder (particularly disseminated intravascular coagulopathy or combined FVIII/FV deficiency) or platelet disorder.
2. History of diseases related to venous thromboembolic events (e.g., pulmonary embolism, deep vein thrombosis, thrombophlebitis) or thrombotic microangiopathy.
3. Risk factors for venous or arterial diseases (e.g., uncontrolled hypertension, uncontrolled diabetes).
4. History of cardiac, coronary and/or arterial peripheral atherosclerotic disease, particularly myocardial infarction, cerebrovascular accident, stroke, transient ischemic attack, congestive heart failure, angina pectoris or treatment for angina pectoris.
5. Platelet count <100,000/µL.
6. Human immunodeficiency virus (HIV) infection with a cluster of differentiation 4 (CD4+) lymphocyte count of <200/mm3.
7. Any planned major surgical intervention.
8. Subjects with known or suspected hypersensitivity to trial product(s) or related products.
9. Subjects with known autoimmune disease or on treatment with immune-modulatory drugs.
10. Subjects with advanced liver disease (Child Pugh Grade B and C) or with signs of liver function impairment (e.g., alanine aminotransferase [ALT] and aspartate aminotransferase [AST] levels >3 x the upper limit of the normal range and/or total bilirubin >2.0 x the upper limit of the normal range).
11. Subjects with serum creatinine >2.0 x the upper limit of the normal range.
12. Treatment with an investigational drug within 3 months prior to enrolment.
13. Subjects not willing to stop prophylaxis or ITI.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the safety and tolerability of multiple subcutaneous doses of BAY 1093884 in subjects with hemophilia A or B with or without inhibitors. ;<br> Secondary Objective: There are no secondary objectives in the trial.<br> <br> ;<br> Primary end point(s): The primary endpoint is safety as defined by the frequency of drug-related AEs, SAEs, AESIs and clinically relevant abnormal laboratory values.<br> Another safety endpoint will be the frequency of binding and neutralizing antibodies against BAY 1093884. <br> ;Timepoint(s) of evaluation of this end point: Throughout the conduct of the study.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): There is no secondary endpoint;Timepoint(s) of evaluation of this end point: n/a

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