A Phase 3 interventional study of Emicizumab and FEIBA in Hemophilia A, sponsored by Emory University. Recruiting at 2 sites in United States. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-08-21.
Sponsored by Emory University · Phase 3, Interventional, and Treatment
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.
Hemophilia A (HA) is a congenital bleeding disorder caused by deficient or dysfunctional factor VIII (FVIII) which leads to bleeding correlated with factor deficiency severity. Patients with HA develop recurrent bleeds into joints and soft tissues that culminate into debilitating arthropathy and long-term morbidity.
The previous standard of care for high titer antibody eradication in hemophilia A (HA) included a labor-intensive, immune tolerance induction (ITI) regimen administered with concomitant bypassing agent (BPA) prophylaxis, either daily recombinant activated factor VII (rFVIIa) or at least 3 non-consecutive days of activated prothrombin complex concentrate (aPCC) given intravenously (IV) each week.
The overall objective is to determine whether the thrombin generation assay can be used to personalize a dose of aPCC that could be used in a future study during an acute bleeding event and peri-surgical prophylaxis in children and adults with hemophilia A and inhibitors on emicizumab primary prophylaxis.
Exclusion Criteria:
Participants will have a first/baseline thrombin generation assay (TGA) sample (to be processed within 60 minutes), followed by an infusion at 15 U/kg dose of aPCC, and provide a second TGA sample 15-30 minutes after to be processed within 60 minutes. If required, a subsequent TGA sample will be obtained upon a 25 U/kg dose of aPCC to be processed within 60-90 minutes.
Drug: Emicizumab · Drug: FEIBA · Drug: rFVIIa
HEMLIBRA® is a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors.
Also known as: ACE910, Hemlibra and RO5534262
FEIBA™ is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia patients with inhibitors for: control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes. The max dose allowed for aPCC will be 50 U/kg dose given at a single visit.
Also known as: Activated prothrombin complex (aPCC), Anti-Inhibitor Coagulant Complex
rFVIIa is a coagulation factor VIIa concentrate indicated for the treatment and control of bleeding episodes occurring in adults and adolescents with hemophilia with inhibitors.
Also known as: Recombinant activated factor VII
Thrombin generation capacity after aPCC (FEIBA) infusion
Thrombin generation capacity from up to two escalating doses of aPCC will be measured using a thrombin generation assay at baseline and up to 30 minutes after FEIBA infusion.
Time frame: Baseline and up to 30 minutes after infusion
Number of serious adverse events
Number of serious adverse events will be recorded
Time frame: up to 1 year
Number of serious bleeding episodes
Number of serious bleeding episodes will be recorded
Time frame: up to 1 year
Number of episodes of thrombotic events including thrombotic microangiopathy (TMA)
Number of episodes of thrombotic events including thrombotic microangiopathy (TMA) will be recorded
Time frame: up to 1 year
Plan to share: Yes — All of the individual participant data collected during the trial, after de-identification, will be available
Supporting information: Study protocol, Sap, Icf
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Emory University