This is a prospective, randomized control trial in which each patient will be randomly assigned to receive either extended half-life factor VIII based replacement therapy or non-FVIII based replacement therapy, which are both standard of care treatment for persons with Hemophilia A.
This is a research study to find out if there is a difference in the way children with hemophilia A form a clot and also evaluate if they develop tiny bleeds within the joint and subsequently early joint changes when receiving extended half-life factor VIII based replacement therapy vs non-FVIII based replacement as part of their hemophilia treatment to prevent spontaneous joint bleeds. Both therapies are standard of care therapies for patients with hemophilia A. The only experimental/research procedures as part of this study include the thrombin generation assay and ultrasound. All other procedures are standard of care.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Eloctate is FDA-approved to treat patients with Hemophilia A. This drug will be used for extended half-life factor VIII-based replacement therapy.
Adynovate is FDA-approved to treat patients with Hemophilia A. This drug will be used for extended half-life factor VIII-based replacement therapy.
Emicizumab is FDA-approved to treat patients with Hemophilia A. This drug will be used for non-factor VIII-based replacement therapy.
The University of Texas Southwestern Medical Center
Dallas, Texas, United States
Changes in hemostatic potential
Evaluate differences in hemostatic potential in persons with hemophilia A without evidence of hemophilic target joints who are receiving prophylactic FVIII-based replacement therapy versus non-FVIII replacement therapy by measuring thrombin generation potential via a plasma-based assay
Time frame: Baseline, 1 month, 6 months, and 12 months from start of study
Changes in early joint-health markers
Identify early joint-health markers associated with bleeding in persons with hemophilia A without evidence of hemophilic target joints who are receiving prophylactic FVIII-based replacement therapy versus non-FVIII replacement by measuring soft tissue and osteochondral changes among 6 joints (bilateral elbows, knees, and ankles) using point of care ultrasound between both treatment groups
Time frame: Baseline, 1 month, 6 months, and 12 months from the start of study
Changes in bleeding rates
Measure bleeding rates in persons with hemophilia A without evidence of hemophilic target joints on prophylactic FVIII-based replacement therapy versus non-FVIII replacement therapy by measuring the difference in annualized bleeding rates between the two treatment groups, as well as other surrogate markers of bleeding, or indirect causes of bleeding, which include FVIII inhibitor level, hemoglobin, and serum ferritin
Time frame: Baseline and 12 months after start of study
Changes in health-related quality of life and physical activity
Evaluate health-related quality of life and physical activity in persons with hemophilia A without evidence of hemophilic target joints on prophylactic FVIII-based replacement therapy versus non-FVIII replacement therapy using validated questionnaires
Time frame: Baseline, 1 month, 6 months, and 12 months from the start of study
Changes in joint health
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Assess joint health in persons with hemophilia A without evidence of hemophilic target joints on prophylactic FVIII-based replacement therapy versus non-FVIII replacement therapy using validated outcome tool (Hemophilia Joint Health Score)
Time frame: Baseline, 1 month, 6 months, and 12 months from the start of study