NCT04563520 SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
| NCT ID | NCT04563520 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Emory University |
| Condition | Hemophilia A |
| Study Type | INTERVENTIONAL |
| Enrollment | 5 participants |
| Start Date | 2026-09 |
| Primary Completion | 2027-03 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 5 participants in total. It began in 2026-09 with a primary completion date of 2027-03.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: * Moderately severe hemophilia A, defined as FVIII level \<0.05 IU/mL before development of an inhibitor * Age ≥6 years of age at time of informed consent * Documented on 2 occasions a high titer inhibitor (\>5 BU/mL) with a 72-hour washout within 2 years of enrollment * Parent/guardian (Legally Authorized Representative) or the patient has provided written informed consent * Adequate hematologic function (Hgb \>8 g/dL and platelet count \>100,000 µL) * Adequate hepatic function (total bilirubin ≤1.5 x ULN and both AST/ALT ≤3x ULN at screening (excluding known Gilbert's) * Adequate renal function (≤2.5 x ULN and CrCl ≥30 mL/min) Exclusion Criteria: * Inherited or acquired bleeding disorder other than hemophilia A excluding low VWF (\>30% VWF:RCo or VWF:GP1bm) * Had an active bleed requiring factor therapy at screening * Previous or current treatment for thromboembolic disease or signs of thromboembolic disease (excluding previously resolved line-associated thrombosis) * Had a surgical procedure 14 days before screening * Conditions that may increase the risk of bleeding or thrombosis * If the patient is treated with rFVIIa or aPCC seven days before screening * History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection * Had current use of any medication other than emicizumab that could affect the coagulation system. * Known HIV infection with CD4 count \<200 cells/µL within 24 weeks before screening. Testing is not required if \<35 years of age. * Use of systemic immunomodulators at enrollment or planned use during the study * Participants who are at high risk for TMA (for example, have a previous medical/family history of TMA), in the investigator's judgment * Concurrent disease, treatment, or abnormality in clinical laboratory tests that could interfere with the conduct of the study, may pose an additional risk, or would, in the opinion of the investigator, preclude the participant's safe participation in and completion of the study
Contact & Investigator
Robert Sidonio, MD
PRINCIPAL INVESTIGATOR
Emory University
Frequently Asked Questions
Who can join the NCT04563520 clinical trial?
This trial is open to participants of all sexes, aged 6 Years or older, studying Hemophilia A. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04563520 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 5 participants.
Is NCT04563520 currently recruiting?
Yes, NCT04563520 is actively recruiting participants. Contact the research team at robert.sidonio.jr@emory.edu for enrollment information.
Where is the NCT04563520 trial being conducted?
This trial is being conducted at Atlanta, United States, Atlanta, United States.
Who is sponsoring the NCT04563520 clinical trial?
NCT04563520 is sponsored by Emory University. The principal investigator is Robert Sidonio, MD at Emory University. The trial plans to enroll 5 participants.