RECRUITINGPhase 3INTERVENTIONAL

SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

aPCC and Emicizumab Safety Study in Congenital Hemophilia A Patients With Inhibitors (SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis)

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 willing to sign a consent form - 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 willing to sign a consent form - 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) Who Should NOT Join This Trial: - 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 ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language

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

Treatments Being Tested

DRUG

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.

DRUG

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.

DRUG

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.

Locations (2)

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Emory University Hospital

Atlanta, Georgia, United States