Prediction of the Efficacy of Activated Recombinant Human Factor VII in Adult Congenital Haemophilia A or B Patients With Inhibitors by Use of Thromboelastography

Study Overview
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Brief Summary
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This trial is conducted in Europe and the United States of America (USA). The aim of this trial is to evaluate the basal and spiked TEG® (Thromboelastography) or ROTEM® (Thromboelastometry) profiles of frequently bleeding haemophilia subjects with inhibitors in a non-bleeding state.

Detailed Description
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The TEG parameters are: R time (Reaction Time), K time (K Time (arbitrary measurement)), a (a angle), MA (Maximum Amplitude) and LY30 (Lysis 30 min after MA) while the ROTEM parameters are: CT (Clotting Time), CFT (Clot Formation Time), a (a angle), MCF (Maximum Clot Firmness) and LI60 (Lysis index 60 min after CT)

Official Title
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An Exploratory Non-interventional Study of Ex-vivo Spiking Followed by Thromboelastography as a Method for Predicting the Efficacy of Recombinant Activated Human FVII (rFVIIa, NovoSeven®) in Adult Congenital Haemophilia A or B Patients With Inhibitors

Conditions
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Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors

Intervention / Treatment
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* Drug: eptacog alfa (activated)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed diagnosis of congenital haemophilia A or B with a FVIII:C clot activity or FIX:C clot activity, respectively, less than 5% Presently use of activated recombinant human factor VII (NovoSeven®) as haemostatic agent for preventive treatment or treatment of bleeding episodes A documented historical or present record for the presence of inhibitors to factor VIII or IX, respectively A documented history of 2 or more joint bleeding episodes during the preceding 12 months Subjects must be in a non-bleeding state (i.e. no clinical manifestations of active bleeds) Exclusion Criteria: Subjects who have received any haemostatic treatment for a bleeding episode within the last 7 days prior to this trial Immune tolerance therapy within the last 30 days prior to this trial Clinically relevant coagulation disorders other than congenital haemophilia A or B Thrombocytopenia (platelet count below 60,000 platelets/mcl) Prophylactic haemostatic treatment within 3 days prior to this trial

Ages Eligible for Study
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Minimum Age: 16 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Diagnostic
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Ex vivo<br> | Drug: eptacog alfa (activated)<br>* Subjects will be called to attend the clinic in a non-bleeding state. Blood samples will be drawn for thromboelastogram profiling and will be in-vitro spiked with different doses of activatated recombinant human factor VII.<br>* Other names: activated recombinant human factor VII;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| TEG parameters obtained at baseline and with activated recombinant human factor VII |  |  | 
| ROTEM parameters obtained at baseline and with activated recombinant human factor VII |  |  |