Individualizing Hemophilia Bypassing Agent Therapy Utilizing Thromboelastography

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

WITHDRAWN

Clinical Phase

NA

Study Classification

INTERVENTIONAL

Study Start Date

2017-01-31

Study Completion Date

2019-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Individualizing Hemophilia Prophylaxis Using Thromboelastography

NCT02582060

Hemophilia A

COMPLETED NA

Prediction of the Efficacy of Activated Recombinant Human Factor VII in Adult Congenital Haemophilia A or B Patients With Inhibitors by Use of Thromboelastography

NCT01561924

Congenital Bleeding Disorder Haemophilia A With Inhibitors Haemophilia B With Inhibitors

COMPLETED PHASE1

A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on Emicizumab

NCT04205175

Hemophilia A With Inhibitor

ACTIVE_NOT_RECRUITING PHASE4

Thrombin Generation Numerical Models Validation in Haemophilic Case

NCT02300519

Haemophilia B Haemophilia A

COMPLETED

PK Driven Prophylaxis for Hemophilia A

NCT02634424

Hemophilia A

TERMINATED NA

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

See the medical conditions and disease areas that this research is targeting or investigating.

Hemophilia a with Inhibitors

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Severe Hemophilia A subjects w/ inhibitors

Males age 4-70 years diagnosed with severe hemophilia A with inhibitors who are currently treated with prophylaxis or on-demand treatment will be enrolled.

Group Type OTHER

Thromboelastography-Guided Treatment Regimen

Intervention Type OTHER

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.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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.

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Males, 4-60 years old, inclusive on Prophylaxis or On-Demand treatment
2. Diagnosis of Hemophilia with active titer inhibitors (\> 0.6 BU)
3. Willing to alter their treatment regimen per study protocol

Exclusion Criteria

1. Bleeding disorder(s) other than hemophilia A with inhibitors
2. Thrombocytopenia (platelet count \<100,000K/µL)
3. Any concurrent clinically significant major disease that, in the opinion of the investigator, would make the subject unsuitable for enrollment
4. Participation within the past 30 days in a clinical study involving investigational drugs
5. Planned major surgery within 30 days prior to screening or during the study period

Minimum Eligible Age

4 Years

Maximum Eligible Age

70 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No