Individualizing Hemophilia Bypassing Agent Therapy Utilizing Thromboelastography

NCT03002480 | Withdrawn

• 1 other identifier: CHLA-16-00501
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Hemophilia a with Inhibitors

Outcome Measures

Primary Outcomes (1)

• Elastic properties of a forming clot using thromboelastograph to determine best bypassing agent

  This study will assess the feasibility of TEG-guided individualization of bypassing agent treatment of bleeding for severe hemophilia A patients with inhibitors.

  6 months

Secondary Outcomes (2)

• Thrombin generation assay measuring the formation of thrombin during clot formation

  6 months

• Number of participants with treatment-related adverse events

  1 year

Study Arms (1)

Severe Hemophilia A subjects w/ inhibitors

OTHER

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.

Other: Thromboelastography-Guided Treatment Regimen

Interventions

Thromboelastography-Guided Treatment Regimen 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.

Severe Hemophilia A subjects w/ inhibitors

Eligibility Criteria

• Age: 4 Years - 70 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Children's Hospital Los Angeles: lead

Study Officials

• Guy Young, MD

  Children's Hospital Los Angeles

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director of Thrombosis and Hemostasis

Study Record Dates

• First Submitted: December 19, 2016
• First Posted: December 23, 2016
• Study Start: January 1, 2017
• Primary Completion: January 1, 2019
• Study Completion: January 1, 2019
• Last Updated: September 26, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share