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Individualizing Hemophilia Prophylaxis Using Thromboelastography

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ClinicalTrials.gov Identifier: NCT02582060
Recruitment Status : Completed
First Posted : October 21, 2015
Last Update Posted : February 18, 2019
Sponsor:
Information provided by (Responsible Party):
Guy Young, Children's Hospital Los Angeles

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.

Condition or disease Intervention/treatment Phase
Hemophilia A Other: Thromboelastography/Thromboelastometry-Guided Treatment Regimen Not Applicable

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.


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Individualizing Hemophilia Prophylaxis Using Thromboelastography
Actual Study Start Date : May 20, 2014
Actual Primary Completion Date : December 31, 2017
Actual Study Completion Date : December 31, 2017

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
Severe Hemophilia A Subjects
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
The results of the TEG/ROTEM assay (specifically the R time/CT) will be used to determine the prophylaxis treatment regimen.




Primary Outcome Measures :
  1. Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by estimated proportion of patients whose dose is modified. [ Time Frame: Ongoing while patients are on study (~ 6 months) ]
    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.

  2. Monitoring short-term safety of patients whose dose has been modified, assessed by monitoring number, type, and severity of bleeds. [ Time Frame: Ongoing while patients are on study (~ 6 months) ]
    The primary endpoint for subject safety will be the occurrence of two serious spontaneous bleeding episodes within 28 days of each other.

  3. Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by testing and refining operational protocol for using TEG/ROTEM to guide factor dosing. [ Time Frame: Ongoing while patients are on study (~ 6 months) ]

Secondary Outcome Measures :
  1. Thrombin generation assay will be performed to provide additional evidence supporting the TEG/ROTEM-guided dosing. [ Time Frame: 6 months ]
  2. Assessment of direct costs for all subjects whose treatment was modified using factor consumption 6 months before, and the 6 months during study participation. [ Time Frame: 1 year ]
    Analysis for those subjects who had a modified dosing regimen to determine the per capita reduction in consumption when a modified dosing regimen is achieved.

  3. Assessment of indirect costs for all subjects whose treatment was modified using factor consumption 6 months before, and the 6 months during study participation. [ Time Frame: 6 months ]
    Questionnaire collecting data on time spend on hemophilia-related care per week, caregiver support related to hemophilia, and number of infusions per week.

  4. Improvement of subject/family burden as assessed by the Health-Related Quality of Life. [ Time Frame: 6 months ]
    (HRQoL) Assessments/Analysis



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Ages Eligible for Study:   5 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02582060


Locations
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United States, California
CHLA
Los Angeles, California, United States, 90027
Sponsors and Collaborators
Children's Hospital Los Angeles

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Responsible Party: Guy Young, Guy Young, MD, Children's Hospital Los Angeles
ClinicalTrials.gov Identifier: NCT02582060     History of Changes
Other Study ID Numbers: 14-00030
First Posted: October 21, 2015    Key Record Dates
Last Update Posted: February 18, 2019
Last Verified: February 2019

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn