In Vitro Correction of Thrombin Generation by Concizumab (Anti-TFPI) for Severe Hemophilia Patients

NCT05617209 | Completed

• 1 other identifier: 21CH036
• Study Type: observational
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Studies have shown, in haemophilia patients, the effectiveness of Anti TFPI antibody (aTFPIAb) to prevent joint and muscular bleeding (tissues poor in Tissue Factor (TF)). However, cases of cerebral thrombosis (tissues rich in TF) have been observed in some patients treated with this antibody. Because an inter-individual variation in thrombin generation correction by aTFPIAb, an aTFPIAb concentration effective at low TF concentration could be at the same time thrombogenic at high TF concentration.

Conditions

Hemophilia

Keywords

anti-TFPI antibody; thrombin generation; tissue factor concentration

Outcome Measures

Primary Outcomes (1)

• minimum concentration of anti-TFPI antibody

  Determine the minimum concentration of anti-TFPI antibody (µg/ml) that normalizes the peak to 1 pM FT

  day 1

Secondary Outcomes (1)

• Measurement of the clot structure

  day 1

Study Arms (1)

Severe and/or moderate haemophilia A and B

At the end of the consultation and follow-up of the disease, an additional blood sample of 35 ml (8 tubes) will be taken

Other: thrombin generation measurement

Interventions

thrombin generation measurement OTHER

Thrombin generation will be measured in Platelets rich plasma induced either by 1 pM TF or 10 pM TF concentration in presence of different aTFPIAb concentrations (0; 0.5 µg/ml; 0.75 µg/ml; 1.0 µg/ml; 2.0 µg/ml; 4.0 µg/ml)

Severe and/or moderate haemophilia A and B

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Haemophilia A and B patients

You may qualify if:

• Severe or moderate haemophilia A or B patient with FVIII or FIX \<5% with or without prophylaxis
• Affiliated or beneficiary of a social security regimen
• Signature of consent

You may not qualify if:

• Haemophilia patients aged 12 years or less with a weight less than 30 kg (taking into account the volume of blood collection).
• Patients who received factor VIII concentrates less than 48 hours or factor IX concentrates less than 96 hours prior to the routine blood draw at the Hemophilia Clinic. 96 hours prior to the routine blood draw at the Hemophilia Centre

Sponsors & Collaborators

• Centre Hospitalier Universitaire de Saint Etienne: lead

Study Sites (1)

CHU Saint-Etienne

Saint-Etienne, 42055, France

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

Blood sampling: thrombin generation measurement

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

Study Officials

• Brigitte Tardy, MD

  Centre Hospitalier Universitaire de Saint Etienne

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: November 8, 2022
• First Posted: November 15, 2022
• Study Start: September 7, 2021
• Primary Completion: January 5, 2023
• Study Completion: January 5, 2023
• Last Updated: November 27, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)