TGA (Thrombin Generation Assay) and Prophylaxis in Haemophilia

Haemophilia A Clinical Trial

— HOPE  

Official title:

Thrombin Generation Capacity of Patients With Severe Haemophilia Receiving Prophylaxis

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02781766
• Other study ID #: 2012-765
• Status: Recruiting
• Phase: N/A
• Start date: August 8, 2016
• Est. completion date: September 8, 2019

Study information

• Verified date: July 2019
• Source: Hospices Civils de Lyon
• Contact: Yesim DARGAUD, Pr
• Phone: (0)4.72.11.88.10
• Email: ydargaud[@]univ-lyon1.fr
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Haemophilic arthropathy is one of the major complications of severe haemophilia. In order to maintain plasma clotting factor activity levels above 1% and avoid spontaneous joint bleeds and other serious bleeding events, prophylactic factor replacement therapy is used. Because of the high cost and limited availability of clotting factor concentrates, dosing is a crucial issue for prophylaxis therapy. Several studies reported a better correlation between clinical bleeding tendency of patients with haemophilia and thrombin generation assay results compared to FVIII/FIX levels. However there is no specific data showing that thrombin generation may be a better indicator of the clinical efficacy of prophylaxis compared to the conventional FVIII measurement.

The main objective of this open, multicentre, prospective study is to compare trough thrombin generation capacity and FVIII levels in severe haemophilia patients and compare these two laboratory results with:

• the number of spontaneous haemarthroses and other spontaneous serious bleeds occurred in the last 6 months

• the number of additional FVIII units used in the last 6 months

This project requires no change in term of type of treatment : During the study, each patient will be treated by his usual clotting factor at the usual regimen (frequency and dosage).

The clinical outcome with the usual prophylaxis regimen will be correlated to TGA results

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: September 8, 2019
• Est. primary completion date: September 8, 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 2 Years to 45 Years

Eligibility

Inclusion Criteria:

• Severe haemophilia A (FVIII < 1 IU/dl)

• Currently on prophylactic therapy

• The same prophylaxis regimen in the last six months

• Age: 2 (with a body weight =12.5 kg ) - 45 years

• Adequate venous access

• Haemophilia patient's diary or equivalent regularly completed

• Ability of patient or family (for minors) to give informed consent

• Subject with health insurance

Exclusion Criteria:

• Age <2 and >45 years

• Haemophilia A with documented inhibitor in the last 12 months

• Clinically symptomatic liver disease (historical evidence documented in patient's medical file)

• Platelet count < 100x109/l

• Poor venous access

• Presence of a documented target joint

• Subject under legal protection measure.

Related Conditions & MeSH terms

• Haemophilia A
• Hemophilia A

Intervention

Other:
• Thrombin generation measurement.

Locations

Country	Name	City	State
France	Hôpital Pellegrin	Bordeaux
France	Unité d'Hémostase Clinique Centre Régional de Traitement des Hémophiles Centre de Référence de Maladies Hémorragiques Hôpital Cardiologique Louis Pradel	Bron
France	CH Metropole Savoie site de Chambéry	Chambéry
France	CHU Dijon Bourgogne Hôpital François Mitterrand	Dijon
France	Chu Timone	Marseille
France	CHU de Nantes	Nantes
France	CHU Saint Etienne - Hôpital Nord	Saint-Étienne

Sponsors (1)

Lead Sponsor	Collaborator
Hospices Civils de Lyon

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The correlation between the trough TGA (ETP) results of severe haemophilia patients who are on prophylaxis and the clinical efficacy of the ongoing prophylaxis regimen will be studied		The clinical efficacy of prophylaxis will be evaluated on the basis of the number of spontaneous joint bleeds the number of other relevant spontaneous bleeds and the consumption of clotting factor concentrate in the last 6 months prior to TGA.
Secondary	The correlation between the trough FVIII:C results of severe haemophilia patients who are on prophylaxis and the clinical efficacy of the ongoing prophylaxis regimen will be studied		The clinical efficacy of prophylaxis will be evaluated on the basis of the number of spontaneous joint bleeds the number of other relevant spontaneous bleeds and the consumption of clotting factor concentrate in the last 6 months prior to FVIII.