Health TrialsbyHealthPanel

SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

ConditionHemophilia A
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age6+
SponsorEmory University

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • Emicizumab

    Drug

    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

    Drug

    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

    Drug

    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.

Treatment groups

5 Participants
are divided into 1 treatment group
Group A: Experimental treatmentExperimental treatment 3 interventions

Trial outcomes

Primary outcomes

1

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

Secondary outcomes

1

Number of serious adverse events

Number of serious adverse events will be recorded

Time frame
up to 1 year
2

Number of serious bleeding episodes

Number of serious bleeding episodes will be recorded

Time frame
up to 1 year
3

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Emory University

Lead sponsor

Takeda Pharmaceuticals North America, Inc.

Collaborator

Source ClinicalTrials.gov
Apply now