Actively Recruiting
SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
Led by Emory University · Updated on 2026-04-29
5
Participants Needed
2
Research Sites
39 weeks
Total Duration
On this page
Sponsors
E
Emory University
Lead Sponsor
T
Takeda Pharmaceuticals North America, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
The purpose of the aPCC-emicizumab safety study is to investigate the hemostatic efficacy as measured by thrombin generation, of a low personalized dose of aPCC (FEIBA) in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis.
CONDITIONS
Official Title
SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Moderately severe hemophilia A with factor VIII level below 0.05 IU/mL before inhibitor development
- Age 6 years or older at the time of informed consent
- Documented high titer inhibitor greater than 5 Bethesda Units/mL on two occasions within 2 years prior to enrollment
- Parent/guardian or patient has provided written informed consent
- Adequate hematologic function (hemoglobin greater than 8 g/dL and platelet count greater than 100,000 per microliter)
- Adequate liver function (total bilirubin less than or equal to 1.5 times upper limit of normal and AST/ALT less than or equal to 3 times upper limit of normal at screening, excluding known Gilbert's syndrome)
- Adequate kidney function (creatinine clearance greater than or equal to 30 mL/min and renal lab values within 2.5 times upper limit of normal)
You will not qualify if you...
- Inherited or acquired bleeding disorders other than hemophilia A, excluding low von Willebrand factor (greater than 30% VWF:RCo or VWF:GP1bM)
- Active bleeding requiring factor therapy at screening
- Previous or current thromboembolic disease or signs of thromboembolic disease, excluding previously resolved line-associated thrombosis
- Surgical procedure within 14 days before screening
- Conditions increasing risk of bleeding or thrombosis
- Treatment with rFVIIa or aPCC within 7 days before screening
- History of significant hypersensitivity to monoclonal antibody therapies or components of emicizumab
- Current use of medications other than emicizumab that affect coagulation
- Known HIV infection with CD4 count less than 200 cells/microliter within 24 weeks before screening (testing not required if under 35 years old)
- Use of systemic immunomodulators at enrollment or planned during study
- High risk for thrombotic microangiopathy as judged by investigator
- Concurrent disease, treatment, or abnormal lab tests that interfere with study conduct or participant safety
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 2 locations
1
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
Actively Recruiting
2
Emory University Hospital
Atlanta, Georgia, United States, 30322
Actively Recruiting
Research Team
R
Robert Sidonio, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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