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

Phase 1

• Conditions: Hemophilia A or B; MedDRA version: 20.0 Level: LLT Classification code 10053751 Term: Hemophilia A with anti factor VIII System Organ Class: 100000004850; MedDRA version: 20.0 Level: LLT Classification code 10053753 Term: Hemophilia A without inhibitors System Organ Class: 100000004850; MedDRA version: 20.0 Level: LLT Classification code 10053752 Term: Hemophilia B with anti factor IX System Organ Class: 100000004850; MedDRA version: 20.1 Level: LLT Classification code 10053754 Term: Hemophilia B without inhibitors System Organ Class: 100000004850; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2017-003324-67-FR
• Lead Sponsor: Bayer AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-08-10
• Last Update Posted: 10 December 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 24

Inclusion Criteria

1. Male subjects with:
- hemophilia A or hemophilia B (any severity) with inhibitors against FVIII or FIX (any inhibitor titer)
OR:
- severe hemophilia A (FVIII activity <1%) or B (FIX activity <2%) without inhibitors.
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 (signs of liver function impairment, e.g., albumin, vitamin K coagulation factors) and/or alanine aminotransferase (ALT) and aspartate aminotransferase (AST) levels >3 times the upper limit of the normal range and/or total bilirubin >2.0 times the upper limit of the normal range.
11. Subjects with serum creatinine >2.0 times the upper limit of the normal range.
12. Treatment with an investigational drug within 3 months prior to screening visit.
13. Subjects not willing to stop prophylaxis or ITI.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified