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| Name | Class |
|---|---|
| Genentech, Inc. | INDUSTRY |
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This trial will enroll patients with severe hemophilia A. Experiments will be run in vitro by spiking patients' blood with different molecules (currently used and theoretically proposed as adjunctive therapy to emicizumab), therefore no more than minimal direct risk to patients is expected. This is a pilot preclinical study.
The study aims to determine the hemostatic effect of different Factor IX (FIX) concentrates when added to the plasma of patients on emicizumab prophylaxis and compare it to the hemostatic effect obtained with the addition of bypassing agents (activated prothrombin complex aPCC, and recombinant VIIa rVIIA, recombinant porcine FVIII).
The investigators will study the hemostatic effect using Thrombin Generation assay (CAT) in the presence of different concentrations of FIX concentrates. The investigators will compare the hemostatic effect of added FIX to the effect of added bypassing agents (activated prothrombin complex aPCC, and recombinant VIIa rVIIA, recombinant porcine FVIII)
Plasma will be collected from patients with hemophilia A and inhibitors treated with Emicizumab, a group of plasma samples will be spiked with different concentrations of Factor IX. Thrombin generation profiles will be obtained using CAT.
Another group of plasma samples will be spiked with different concentrations of by passing agents (activated prothrombin complex aPCC, and recombinant VIIa rVIIA, recombinant porcine FVIII). Thrombin generation profiles will be obtained using CAT.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patient Group | Experimental | In vitro spiking experiments will be realized in plasmas from patients with severe haemophilia A on emicizumab using increasing concentrations of factor IX (rFIX), Activated prothrombin complex (aPCC) and recombinant VIIa (rFVIIa). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Factor IX | Drug | Factor IX is on of the substrates of emicizumab. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Determine the homeostatic effect of adding Factor IX concentrate to the plasma of patients with Hemophilia A with inhibitors on emicizumab using peak thrombin | Peak thrombin is a thrombin generation profile. The investigators will compare the peak thrombin obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with aPCC and rFVIIa on emicizumab prophylaxis. | 1 Year |
| Determine the hemostatic effect of Factor IX concentrates and recombinant porcine FVIII when added to the plasma of patients with Hemophilia A with inhibitors on emicizumab using peak thrombin | The investigators will compare the peak thrombin obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with recombinant porcine FVIII on emicizumab prophylaxis. | 1 Year |
| Determine the homeostatic effect of adding Factor IX concentrate to the plasma of patients with Hemophilia A with inhibitors on emicizumab using Endogenous Thrombin Potential (ETP) | Endogenous Thrombin Potential is a thrombin generation profile. The investigators will compare the Endogenous Thrombin Potential obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with aPCC and rFVIIa on emicizumab prophylaxis. | 1 Year |
| Determine the hemostatic effect of Factor IX concentrates and recombinant porcine FVIII when added to the plasma of patients with Hemophilia A with inhibitors on emicizumab using Endogenous Thrombin Potential (ETP) | The investigators will compare the Endogenous Thrombin Potential obtained when spiking patient's plasma with Factor IX concentrates to that obtained when spiking patient's plasma with recombinant porcine FVIII on emicizumab prophylaxis. | 1 Year |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Maissa Janbain, MD, MS | Tulane University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Tulane University Lakeside Hospital and Clinics | Metairie | Louisiana | 70001 | United States | ||
| University Medical Center New Orleans |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| D005164 | Factor IX |
| ID | Term |
|---|---|
| D004792 | Enzyme Precursors |
| D045762 | Enzymes and Coenzymes |
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
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Open Label
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| New Orleans |
| Louisiana |
| 70112 |
| United States |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D011506 |
| Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |