Individualizing Hemophilia Prophylaxis Using Thromboelastography
Condition: Hemophilia A
MeSH terms: Hemophilia A
Study type: Interventional

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.
    

Detailed description: 
      This is a pilot study in children and adults with severe hemophilia A that utilizes the
      TEG/ROTEM to personalize their prophylaxis treatment.

      Primary objectives:

      • Assess the feasibility of TEG/ROTEM-guided modification of the current prophylaxis regimen
      for individuals with severe hemophilia A. Specifically, to:

        -  Test and refine the operational protocol for using TEG/ROTEM to guide factor dosing

        -  Estimate the proportion of hemophilia A patients whose dose will be modified based on
           TEG/ROTEM results

        -  Monitor short term safety of patients whose dose has been modified.

      Secondary objectives

        -  Perform thrombin generation assays to provide additional evidence supporting the
           TEG/ROTEM-guided dosing.

        -  Obtain preliminary data on the economic impact of TEG/ROTEM-guided hemophilia A dosing
           regimens.

        -  Obtain preliminary data on the potential improvement of subject/family burden resulting
           from TEG/ROTEM-guided regimen using generic and specific quality of life tools.

      The study period will consist of screening, pharmacokinetic (PK) study and follow-up. For
      subjects whose prophylaxis treatment regimen is modified, the follow-up period will include 3
      follow-up clinic visits and 4 follow-up phone calls; these patients will remain on study for
      approximately 6 months following completion of the PK-study then will have an end of the
      study visit (visit 9). Subjects whose treatment regimen is not altered will end their study
      participation after Visit 2 and continuing with their current prophylaxis regimen.

      Study endpoints: A safety review will be conducted after the first 6 subjects complete 30
      days with the extended prophylaxis treatment. If no safety issues are identified, the study
      will proceed, and second safety review will be after an additional 8 subjects complete 30
      days of extended treatment. If a subject has two or more spontaneous bleeds in 30 days, the
      subject will be removed from the study. For subjects who did not have bleeds and completed at
      least 6 months of the study, ongoing prophylactic treatment will be decided by their treating
      physician.
    

Intervention name(s): Thromboelastography/Thromboelastometry-Guided Treatment Regimen
Intervention type(s): Other
Intervention description(s): The results of the TEG/ROTEM assay (specifically the R time/CT) will be used to determine the prophylaxis treatment regimen.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Other