A Study to Assess Pharmacokinetics and Pharmacodynamics Following Administration of BAY1093884 in Patients With Severe Hemophilia

NCT03481946 | Completed Phase 1 FDA Drug

• 1 other identifier: 19592
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objective of this study is to assess the pharmacokinetics in patients with severe hemophilia. The secondary objective is to assess the pharmacodynamics of BAY1093884 based on tissue factor pathway inhibitor activity

Conditions

Hemophilia A; Hemophilia B

Outcome Measures

Primary Outcomes (4)

• AUC (0-tlast) of BAY1093884 in plasma

  Area under the concentration vs. time curve from time 0 to the last data point \> LLOQ

  Up to 15 days after drug administration

• AUC(0-tlast)/D of BAY1093884 in plasma

  AUC(0-tlast) divided by dose

  Up to 15 days after drug administration

• Cmax of BAY1093884 in plasma

  Maximum observed drug concentration in measured matrix after single dose administration

  Up to 15 days after drug administration

• Cmax/D of BAY1093884 in plasma

  Cmax divided by dose

  Up to 15 days after drug administration

Secondary Outcomes (1)

• Tissue factor plasma inhibitor activity: effect of BAY1093884 to inhibit the anticoagulatory activity of plasma TFPI as assessed by a chromogenic assay

  Up to 15 days after drug administration

Study Arms (1)

BAY1093884 in subjects with Hemophilia

EXPERIMENTAL

Single dose of BAY1093884 over 30 minutes administered in subjects with severe congenital Hemophilia A or B, with inhibitors or without inhibitors

Drug: BAY1093884

Interventions

BAY1093884 DRUG

0.3 mg/kg given intravenously

BAY1093884 in subjects with Hemophilia

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males with severe congenital hemophilia A or B defined as \<1% FVIII or \<2% FIX concentration by measurement at the time of screening or from reliable prior documentation (e.g., measurement in other clinical Bayer trials, or diagnostic genetic testing)
• Male with any inhibitor titer at screening or prior to screening at any time from medical records. Subjects may be receiving a bypassing agent (rFVIIa; NovoSeven and/or aPCC; FEIBA) for treatment.
• Age: 18 to 65 years at screening
• BMI: 18 to 29.9 kg/m2

You may not qualify if:

• Subjects with known bleeding disorders (such as von Willebrand factor \[vWF\] deficiency, FXI deficiency, platelet disorders, or known acquired or inherited thrombophilia etc.) other than congenital Hemophilia A or B with or without inhibitors
• History of angina pectoris or treatment for angina pectoris
• History of coronary and/or peripheral atherosclerotic disease, congestive heart failure, disseminated intravascular coagulopathy, or stage 2 hypertension defined as systolic blood pressure (SBP) ≥160 mmHg or diastolic blood pressure (DBP) ≥100 mmHg even if controlled
• History of thrombophlebitis, venous/arterial thromboembolic diseases (particularly deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction, cerebrovascular accident, ischemic heart disease, transient ischemic attack)
• Known or suspected hypersensitivity of the immune system, history of anaphylactic reaction, known severe allergies, non-allergic drug reactions, or multiple drug allergies
• Subjects with inhibitors treated with FEIBA, who are not willing to accept rFVIIa (NovoSeven) for the treatment of any bleeds occurring either between screening and dosing or after study drug administration, and until the end of the study.

Sponsors & Collaborators

• Bayer: lead

Study Sites (1)

Chaim Sheba Medical Center

Ramat Gan, 5262000, Israel

Location

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 12, 2018
• First Posted: March 29, 2018
• Study Start: May 10, 2018
• Primary Completion: October 17, 2018
• Study Completion: February 20, 2019
• Last Updated: September 16, 2020

Record last verified: 2020-09

Locations

IL (1)