Factor IX as Adjunctive Therapy to Emicizumab (EMIX)

NCT05281718 | Completed Early Phase 1 FDA Drug

• 1 other identifier: 2022-200
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Hemophilia A

Keywords

Hemophilia A with inhibitors; In vitro; Emicizumab

Outcome Measures

Primary Outcomes (4)

• 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

Study Arms (1)

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).

Drug: Factor IX

Interventions

Factor IX DRUG

Factor IX is on of the substrates of emicizumab.

Patient Group

Eligibility Criteria

• Age: 12 Years - 120 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Tulane University: lead
• Genentech, Inc.: collaborator

Study Sites (2)

Tulane University Lakeside Hospital and Clinics

Metairie, Louisiana, 70001, United States

Location

University Medical Center New Orleans

New Orleans, Louisiana, 70112, United States

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

Factor IX

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Maissa Janbain, MD, MS

  Tulane University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Open Label

Study Record Dates

• First Submitted: February 11, 2022
• First Posted: March 16, 2022
• Study Start: August 1, 2022
• Primary Completion: March 7, 2024
• Study Completion: July 14, 2024
• Last Updated: January 20, 2025

Record last verified: 2025-01

Locations

US (2)