Clinical trial NCT03002480: Individualizing Hemophilia Bypassing Agent Therapy Utilizing Thromboelastography
Official title: Individualizing Bypassing Agent Therapy Utilizing TEG in Hemophilia Patients with Inhibitors
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: NA
Conditions: Hemophilia a with Inhibitors
Lead sponsor: Children's Hospital Los Angeles
Sponsor class: OTHER
First posted: 2016-12-23
Enrollment: 0
Sex: MALE
Minimum age (years): 4
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Eligibility criteria: 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

Primary outcomes:
measure: Elastic properties of a forming clot using thromboelastograph to determine best bypassing agent
timeFrame: 6 months
Secondary outcomes:
measure: Thrombin generation assay measuring the formation of thrombin during clot formation
timeFrame: 6 months
measure: Number of participants with treatment-related adverse events
timeFrame: 1 year
Other outcomes:
Locations:
Study officials:
affiliation: Children's Hospital Los Angeles
name: Guy Young, MD
role: PRINCIPAL_INVESTIGATOR
References: