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NCT Number: NCT05281718

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

12 year–120 year

Sex eligibility

Male

Study type

Interventional

Phase

Early Phase 1

Primary location

Tulane University Lakeside Hospital and Clinics, Metairie, Louisiana, United States

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About this 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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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.

Treatment and study plan

factor ix

Drug

Factor IX is on of the substrates of emicizumab.

Primary outcomes

  1. Determine the homeostatic effect of adding Factor IX concentrate to the plasma of patients with Hemophilia A with inhibitors on emicizumab using peak thrombin

    Time frame: 1 Year

    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.

  2. 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

    Time frame: 1 Year

    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.

  3. 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)

    Time frame: 1 Year

    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.

  4. 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)

    Time frame: 1 Year

    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.

Sponsors and collaborators

Lead sponsor

Tulane University

Other

Collaborators

  • Genentech, Inc.

Registry information

Official study title

An in Vitro Pilot Study of the Role of Factor IX as an Alternative to Bypassing Agents in the Treatment of Breakthrough Bleeds in Patients With Hemophilia A and Inhibitors Treated With Emicizumab.

Important dates

Study start
2022
Primary completion
2024
Study completion
2024
First posted
Mar 16, 2022
Registry last updated
Jan 20, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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