Individualizing Hemophilia Prophylaxis Using Thromboelastography

NCT02582060 | Completed DMC

• 1 other identifier: 14-00030
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

Currently dosing for prophylaxis is not individualized, and the general approach is to use a dose of 25-40 units/kg given 3 times per week or every other day. One of the issues with weight-based dosing is the possible over-treatment. This is likely due to the fact that laboratory tests are not sensitive enough at the low levels to support decision-making. The Thromboelastograph (TEG®) and Thromboelastometry (ROTEM®) are coagulation devices, which assess the dynamics of clot formation over time and have several characteristics which suggest they may provide important information for individualized prophylaxis treatment for our patients.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (3)

• Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by estimated proportion of patients whose dose is modified.

  A total of 60 patients will be enrolled on this study. It is expected that less than 25% of these patients will be eligible for dose/schedule modification based on TEG/ROTEM.

  Ongoing while patients are on study (~ 6 months)

• Monitoring short-term safety of patients whose dose has been modified, assessed by monitoring number, type, and severity of bleeds.

  The primary endpoint for subject safety will be the occurrence of two serious spontaneous bleeding episodes within 28 days of each other.

  Ongoing while patients are on study (~ 6 months)

• Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by testing and refining operational protocol for using TEG/ROTEM to guide factor dosing.

  Ongoing while patients are on study (~ 6 months)

Secondary Outcomes (4)

• Thrombin generation assay will be performed to provide additional evidence supporting the TEG/ROTEM-guided dosing.

  6 months

• Assessment of direct costs for all subjects whose treatment was modified using factor consumption 6 months before, and the 6 months during study participation.

  1 year

• Assessment of indirect costs for all subjects whose treatment was modified using factor consumption 6 months before, and the 6 months during study participation.

  6 months

• Improvement of subject/family burden as assessed by the Health-Related Quality of Life.

  6 months

Study Arms (1)

Severe Hemophilia A Subjects

OTHER

The results of the TEG/ROTEM assay (specifically the R time/CT) will be used to determine the prophylaxis treatment regimen.

Other: Thromboelastography/Thromboelastometry-Guided Treatment Regimen

Interventions

Thromboelastography/Thromboelastometry-Guided Treatment Regimen OTHER

The results of the TEG/ROTEM assay (specifically the R time/CT) will be used to determine the prophylaxis treatment regimen.

Severe Hemophilia A Subjects

Eligibility Criteria

• Age: 5 Years - 70 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Males, 5-70 years, inclusive
• Plasma FVIII activity \<1% documented (Laboratory result or MD documentation of Severe Hemophilia A diagnosis)
• Currently prescribed prophylaxis treatment regimen infusing ≥3 times a week
• Willing to alter their prophylaxis treatment regimen per study protocol

You may not qualify if:

• Bleeding disorder(s) other than Hemophilia A
• Current inhibitor (\>0.6BU)
• Thrombocytopenia (platelet count \<100,000K/µL since it can alter TEG®/ROTEM® results)
• Creatinine \>2x the upper limit of normal (indicating potential platelet dysfunction)
• Prothrombin time \>3 seconds above the upper limit of normal (indicating potential liver dysfunction)
• Any concurrent clinically significant major disease, frequent bleeding pattern or history of non-compliance that, in the opinion of the investigator, would make the subject unsuitable for enrollment
• Participation within the past 30 days in any other clinical study involving investigational drugs
• Planned major surgery within 30 days prior to screening or during the study period
• Current use of any medication known to have effects on the coagulation system

Sponsors & Collaborators

• Children's Hospital Los Angeles: lead

Study Sites (1)

CHLA

Los Angeles, California, 90027, United States

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Thrombelastography

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Tests; Hematologic Tests; Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Guy Young, MD

Study Record Dates

• First Submitted: October 9, 2015
• First Posted: October 21, 2015
• Study Start: May 20, 2014
• Primary Completion: December 31, 2017
• Study Completion: December 31, 2017
• Last Updated: February 18, 2019

Record last verified: 2019-02

Locations

US (1)