NCT03002480

Individualizing Hemophilia Bypassing Agent Therapy Utilizing Thromboelastography

Individualizing Bypassing Agent Therapy Utilizing TEG in Hemophilia Patients with Inhibitors

• Status: Withdrawn
• Phase: N/A
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Children's Hospital Los Angeles · Academic / Other
• Sex: Male
• Age: 4 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This pilot study will investigate the use of thromboelastograph (TEG) to determine the primary bypassing agent for the management of bleeding in children and adults severe hemophilia A patients with inhibitors. The study will evaluate the TEG profile for each of the available bypassing agents (FEIBA and rFVIIa) in each participating patient, which will then determine which agent provides the most robust clot formation as measured by the TEG. This study will consist of screening visit and 2-4 pharmacokinetic studies to determine the "best" bypassing agent based on the TEG results. Patients will then be assigned that bypassing agent and dose for the treatment of their bleeding episodes (Prophylaxis or On-demand). Each patient will be then followed for a period of 6 months to monitor short-term safety of those patients whose bypassing agent was modified.

Detailed description

Currently, there are no laboratory assays that can assess the pharmaceutical activity of the available agents known collectively as bypassing agents, which negatively impacts the treating physician's ability to manage these patients, leaving decisions on the dosing regimen to trial and error. Both agents, FEIBA and recombinant activated factor VII (rFVIIa), are approved for the treatment of bleeding in inhibitor patients with recommended dosing regimens that are based on clinical trials. However, a significant amount of "experimenting" with other dosing regimens is used by physicians and patients. The goal of this project is to demonstrate that TEG is an excellent biomarker for predicting, managing and individualizing the treatment with FEIBA and rFVIIa of this rare and difficult to treat patient population.

Conditions

• Hemophilia a with Inhibitors

Interventions

Type	Name	Description
OTHER	Thromboelastography-Guided Treatment Regimen	The thromboelastograph (TEG) will be used to determine the "best" bypassing agent for the management of subjects with severe hemophilia A with inhibitors. Patients will be assigned that bypassing agent and dose for the treatment of their bleeding episodes and subsequently will be followed for a period of 6 months to determine their annual bleeding rate.

Timeline

• Start date: 2017-01-01
• Primary completion: 2019-01-01
• Completion: 2019-01-01
• First posted: 2016-12-23
• Last updated: 2024-09-26