Hemophilia A With Inhibitors Clinical Trial
Official title:
Individualizing Bypassing Agent Therapy Utilizing TEG in Hemophilia Patients With Inhibitors
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.
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.
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| 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 |