Recruiting
Phase 3

aPCC

Sponsor:

Emory University

Code:

NCT04563520

Conditions

Hemophilia A

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Interventions

Emicizumab

FEIBA

rFVIIa

Study Details

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.

Conditions

Hemophilia A

Study ID

NCT04563520

Start date

Sep, 2026

Status verified date

Aug, 2026

Completion date

Mar, 2027

Anticipated

Primary completion date

Mar, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

5 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Experimental treatment

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.

Interventions

Emicizumab

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.

FEIBA

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.

rFVIIa

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.

Primary outcome measure

  • Thrombin generation capacity after aPCC (FEIBA) infusion [ Time Frame: Baseline and up to 30 minutes after infusion ]

Central Contacts and Locations

Central contacts

Robert Sidonio, MD

404-785-1637robert.sidonio.jr@emory.edu

Locations

Children's Healthcare of Atlanta

Recruiting

Atlanta, Georgia, United States, 30322

Contacts

Robert Sidonio, MD

4047851637robert.sidonio.jr@emory.edu

Emory University Hospital

Recruiting

Atlanta, Georgia, United States, 30322

More Information

Sponsor

Emory University

Last update posted

Aug 21, 2026

Last verified

Aug, 2026

Keywords

  • hemostatic efficacy
  • safety
  • prothrombin complex concentrate

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Emory University on 2026-08-21.