TFPI Levels in Haemophilia A and B Patients

NCT ID: NCT04570696

Last Updated: 2020-09-30

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

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Total Enrollment

100 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-07-01

Study Completion Date

2021-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Haemophilia A and B

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

Haemophilia TFPI inhibitors TGA TFPI

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Adult haemophilia patients

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

NON INTERVENTIONAL

Intervention Type OTHER

NON INTERVENTIONAL

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

NON INTERVENTIONAL

NON INTERVENTIONAL

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

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

Exclusion Criteria

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

18 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

CHU de Reims

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Damien JOLLY

Reims, , France

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

France

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Philippe NGUYEN

Role: CONTACT

Phone: 03 26 78 80 74

Email: [email protected]

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Philippe NGUYEN

Role: primary

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

PO19085

Identifier Type: -

Identifier Source: org_study_id