Factor IX as Adjunctive Therapy to Emicizumab (EMIX)
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.
- Hemophilia A
- Eligible Ages
- Between 12 Years and 120 Years
- Eligible Genders
- Accepts Healthy Volunteers
- Signed Informed Consent/Assent Form - Male patients, Age 12 years and older at time of signing Informed Consent Form - Ability to comply with the study protocol, in the investigator's judgment - Diagnosis of severe hemophilia A and are receiving emicizumab prophylaxis, on a weekly maintenance regimen
- Not able to sign consent - Not willing to provide extra blood for the experiments. - Patients carrying the diagnoses of other coagulopathies in addition to hemophilia A - Patients that have received any hemostatic agent within 5 half-lives of the blood draw. This applies to any agent that might have been used for any reason prior to the blood draw and might affect the global hemostasis.
- Early Phase 1
- Study Type
- Intervention Model
- Single Group Assignment
- Intervention Model Description
- Open Label
- Primary Purpose
- Basic Science
- None (Open Label)
|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).||
- Tulane University
Study ContactMaissa Janbain, MD, MS
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.