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Clinical trial

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.

ID
Name
2022-200
Description
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.
Trial arms
Trial start
2022-08-01
Estimated PCD
2024-07-01
Trial end
2024-07-01
Status
Recruiting
Phase
Early phase I
Treatment
Factor IX
Factor IX is on of the substrates of emicizumab.
Arms:
Patient Group
Size
40
Primary endpoint
Determine the homeostatic effect of adding Factor IX concentrate to the plasma of patients with Hemophilia A with inhibitors on emicizumab using peak thrombin
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
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)
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)
1 Year
Eligibility criteria
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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['EARLY_PHASE1'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open Label', 'primaryPurpose': 'BASIC_SCIENCE', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 40, 'type': 'ESTIMATED'}}
Updated at
2023-12-22

1 organization

1 product

1 indication

Organization
Tulane University
Product
Factor IX
Indication
Hemophilia A