TFPI Levels in Haemophilia A and B Patients

NCT04570696 | Unknown

• 1 other identifier: PO19085
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

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.

Conditions

Haemophilia A and B

Keywords

Haemophilia; TFPI inhibitors; TGA; TFPI

Outcome Measures

Primary Outcomes (1)

• Concentration of TFPI plasma level

  Correlation coefficient between concentration of TFPI inhibitor and parameter of thrombin generation

  Day 1

Study Arms (1)

Adult haemophilia patients

Adult (≥ 18 years old) haemophilia patients, only men

Other: NON INTERVENTIONAL

Interventions

NON INTERVENTIONAL OTHER

NON INTERVENTIONAL

Adult haemophilia patients

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• Men, adult (≥ 18 years old), haemophilia A or B disease (whatever the severity)
• Follow-up consultation in our center

You may not qualify if:

• Children (\< 18 years old), women
• No blood samples collection planed during the follow-up consultation

Sponsors & Collaborators

• CHU de Reims: lead

Study Sites (1)

Damien JOLLY

Reims, France

RECRUITING

MeSH Terms

Conditions

Hemophilia A

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

Central Study Contacts

Philippe NGUYEN

CONTACT

03 26 78 80 74; pnguyen@chu-reims.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2020
• First Posted: September 30, 2020
• Study Start: July 1, 2019
• Primary Completion: June 1, 2021
• Study Completion: June 1, 2021
• Last Updated: September 30, 2020

Record last verified: 2020-09

Locations

FR (1)