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TFPI Levels in Haemophilia A and B Patients

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ClinicalTrials.gov Identifier: NCT04570696
Recruitment Status : Recruiting
First Posted : September 30, 2020
Last Update Posted : September 30, 2020
Sponsor:
Information provided by (Responsible Party):
CHU de Reims

Study Description
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Brief Summary:

The goal of this study is to measure the TFPI plasma level, a molecule involved in the regulation of the coagulation system, in haemophilia patients. The second objective is to assess the effects of TFPI inhibition on thrombin generation. Indeed, there is sparse data on the physiological and pathological changes of TFPI levels in human and particularly in hemophilia patients. Yet, TFPI inhibitors may become one of the new by-passing treatments of haemophilia. Until now, published data mainly reports in vitro pharmacodynamics and pharmacokinetics of TFPI inhibitors. Hence, in vivo effects of TFPI inhibition remain unclear, especially in haemophilia patients.

The clinical development of such molecules requires a dedicated biological monitoring. Thrombin Generation Assay (TGA) in Poor Platelets Plasma (PPP) is a good candidate since it is sensitive to Factor VIII and Factor IX deficiencies as well as to TFPI. However, TGA results are very dependent on experimental conditions (i. e. Tissue Factor and phospholipids concentrations) and the relationship between TFPI plasma level and TGA parameters has not been studied yet. This study should provide with original data on TFPI plasma levels and the effect of TFPI on thrombin generation in haemophilia patients. This should help to define the monitoring of TFPI inhibitors in Haemophilia.


Condition or disease Intervention/treatment
Haemophilia A and B Other: NON INTERVENTIONAL

Detailed Description:

This study is a single-institution cohort study. Patients will be informed during their follow-up consultation. If the patient agrees to participate to this study, a non-opposition form will be signed.

If blood tests are prescribed during the consultation, the unused plasma samples that are not required to perform the prescribed analyses will be collected and anonymized in order to measure the TGA and TFPI levels.

TFPI plasmatic level and TFPI activity measurements will be performed by a quantitative ELISA assay (Total TFPI ELISA kit®, R&d Systems) and a chromogenic assay (Actichrom TFPI®, Cryopep) respectively.

TGA will be performed on a Fluoroskan® machine using 3 different levels of Tissue Factor (TF): 1, 5 and 20 pM. In order to study the relation between different levels of TFPI inhibitors and TGA parameters, TGA will also be performed by spiking blood samples with several concentrations of TFPI inhibitor. For each TGA performed, ETP (Endogenous Thrombin Potential, nM/min), peak (nM) and lagtime (min) will be analyzed.

Clinical data will be collected from the computerized patient record: date of birth, age, gender, haemophilia type (A or B) and its severity, ongoing treatments and deficient factor level.

The goal of this study is to measure the TFPI plasma level in haemophilia patients. The second objective is to assess the effects of TFPI inhibition on thrombin generation. This study should provide with original data on TFPI plasma levels and the effect of TFPI on thrombin generation in haemophilia patients. This should help to define the monitoring of TFPI inhibitors in Haemophilia.

There is no additional risk for the patient since only unused blood samples, not needed for the prescribed blood tests, will be collected.

Study Design
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Study Type : Observational
Estimated Enrollment : 100 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Measurement of the Plasmatic TFPI Level in Haemophilia Patients and Correlation With Thrombin Generation Assay (TGA) Parameters
Actual Study Start Date : July 1, 2019
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Groups and Cohorts
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Group/Cohort Intervention/treatment
Adult haemophilia patients
Adult (≥ 18 years old) haemophilia patients, only men
 Other: NON INTERVENTIONAL
NON INTERVENTIONAL


Outcome Measures
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Primary Outcome Measures :
  1. Concentration of TFPI plasma level [ Time Frame: Day 1 ]
    Correlation coefficient between concentration of TFPI inhibitor and parameter of thrombin generation


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Adult (≥ 18 years old) men haemophilia patients with follow-up consultation in our center and for whom blood tests are prescribed during the consultation.
Criteria

inclusion criteria :

  • Men, adult (≥ 18 years old), haemophilia A or B disease (whatever the severity)
  • Follow-up consultation in our center
  • Blood samples collection planed during the consultation exclusion criteria :
  • Children (< 18 years old), women
  • No blood samples collection planed during the follow-up consultation
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04570696


Contacts
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Contact: Philippe NGUYEN 03 26 78 80 74 pnguyen@chu-reims.fr

Locations
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France
Damien JOLLY Recruiting
Reims, France
Contact: Philippe NGUYEN    03 26 78 80 74 ext 0033    pnguyen@chu-reims.fr   
Sponsors and Collaborators
CHU de Reims
More Information
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Responsible Party: CHU de Reims
ClinicalTrials.gov Identifier: NCT04570696    
Other Study ID Numbers: PO19085
First Posted: September 30, 2020    Key Record Dates
Last Update Posted: September 30, 2020
Last Verified: September 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by CHU de Reims:
Haemophilia
TFPI inhibitors
TGA
TFPI
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn