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Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

NCT number NCT03002480
Study type Interventional
Source Children's Hospital Los Angeles
Status Withdrawn
Phase N/A
Start date January 2017
Completion date January 2019

See also
  Status Clinical Trial Phase
Completed NCT00231751 - The Malmö International Brother Study (MIBS) N/A
Completed NCT00221195 - Efficacy Study of Activated Prothrombin Complex for Prevention of Bleeds in Hemophilia A With Inhibitors Phase 2/Phase 3
Terminated NCT02484638 - Study of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) for On-demand Treatment of Bleeding Episodes in Patients With Hemophilia A or B With Inhibitors Phase 2/Phase 3
Terminated NCT00212472 - International Immune Tolerance Study N/A
Completed NCT03093480 - A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study) Phase 4
Completed NCT02448680 - A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa Phase 3
Active, not recruiting NCT01105546 - rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors Phase 2
Completed NCT02020369 - A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Patients With Inhibitors to Factor VIII or IX Phase 3