Factor IX as Adjunctive Therapy to Emicizumab (EMIX)

NCT ID: NCT05281718

Last Updated: 2025-01-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

EARLY_PHASE1

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-08-01

Study Completion Date

2024-07-14

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Hemophilia A

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Open Label
Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

NONE

Study Groups

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Patient Group

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).

Group Type EXPERIMENTAL

Factor IX

Intervention Type DRUG

Factor IX is on of the substrates of emicizumab.

Interventions

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Factor IX

Factor IX is on of the substrates of emicizumab.

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

12 Years

Maximum Eligible Age

120 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Genentech, Inc.

INDUSTRY

Sponsor Role collaborator

Tulane University

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Maissa Janbain, MD, MS

Role: PRINCIPAL_INVESTIGATOR

Tulane University

Locations

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Tulane University Lakeside Hospital and Clinics

Metairie, Louisiana, United States

Site Status

University Medical Center New Orleans

New Orleans, Louisiana, United States

Site Status

Countries

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United States

Other Identifiers

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2022-200

Identifier Type: -

Identifier Source: org_study_id

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