RECRUITING

SAFE Study: Safety of APCC Following Emicizumab Prophylaxis

Conditions

Hemophilia a hemostatic efficacy safety prothrombin complex concentrate

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

APCC and Emicizumab Safety Study in Congenital Hemophilia a Patients with Inhibitors (SAFE Study: Safety of APCC Following Emicizumab Prophylaxis)

Quick Facts

Study Start:2025-04

Study Completion:2025-09

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT04563520

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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)	* 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

Inclusion Criteria

Exclusion 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)

* 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

Contacts and Locations

Study Contact

Robert Sidonio, MD

CONTACT

404-785-1637

robert.sidonio.jr@emory.edu

Principal Investigator

Robert Sidonio, MD

PRINCIPAL_INVESTIGATOR

Emory University

Study Locations (Sites)

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322

United States

Emory University Hospital

Atlanta, Georgia, 30322

United States

Collaborators and Investigators

Sponsor: Emory University

Robert Sidonio, MD, PRINCIPAL_INVESTIGATOR, Emory University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-04

Study Completion Date2025-09

Study Record Updates

Study Start Date2025-04

Study Completion Date2025-09

Terms related to this study

Keywords Provided by Researchers

hemostatic efficacy
safety
prothrombin complex concentrate

Additional Relevant MeSH Terms

Hemophilia a