Prospective, Single-Arm, Open-Label Use of Hemlibra (Emicizumab) in the Treatment of Mild Hemophilia A
Indiana Hemophilia &Thrombosis Center, Inc.
Summary
This is a single arm, phase 4, prospective, open-label, United States single-center study to determine the hemostatic characteristics of Hemlibra (emicizumab) as measured by coagulation laboratory parameters in the mild hemophilia A male patient population with endogenous altered FVIII (baseline FVIII activity of \>5% to 30%). The safety and hemostatic efficacy of Hemlibra (emicizumab) in this patient population will be investigated. Secondary outcomes will assess changes in joint health and quality of life in treated patients.
Description
This is a single arm, phase 4, prospective, open-label, United States single-center study to develop laboratory data and assess the clinical hemostatic efficacy and safety of Hemlibra (emicizumab) for hemostatic control of mild hemophilia A patients (baseline FVIII activity, \>5 to 30%). Males aged ≥5 years to ≤45 years without inhibitors are eligible for enrollment. Secondary outcomes will assess changes in quality of life and joint health in treated patients. Approximately 20 patients will be enrolled. As much as possible, the patient population will be selected to provide a variety of FVII…
Eligibility
- Age range
- 5–45 years
- Sex
- Male
- Healthy volunteers
- No
Inclusion Criteria: * Signed informed consent form from the subject, parent or guardian * Male sex * Diagnosis of mild congenital hemophilia A (baseline FVIII level of \>5% to 30%) without a current FVIII inhibitor or a history of FVIII inhibitor * Any number of FVIII exposure days, including PUPs * BMI \<30 * Age ≥5 years to ≤45 years * Medical documentation of bleeding events, outcomes and hemostatic product usage for 12 months prior to study enrollment * Willingness and ability to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, including the hea…
Interventions
- DrugEmicizumab
bispecific monoclonal antibody binding to activated Factor IX and Factor X
Location
- Indiana Hemophila @Thrombosis CenterIndianapolis, Indiana