TGA (Thrombin Generation Assay) and Prophylaxis in Haemophilia (HOPE)

July 9, 2019 updated by: Hospices Civils de Lyon

Thrombin Generation Capacity of Patients With Severe Haemophilia Receiving Prophylaxis

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

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

50

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Bordeaux, France, 33076
        • Not yet recruiting
        • Hopital Pellegrin
        • Contact:
        • Principal Investigator:
          • Sabine CASTET
      • Bron, France, 69500
        • Recruiting
        • Unité d'Hémostase Clinique Centre Régional de Traitement des Hémophiles Centre de Référence de Maladies Hémorragiques Hôpital Cardiologique Louis Pradel
        • Contact:
      • Chambéry, France, 73011
      • Dijon, France, 21079
        • Not yet recruiting
        • CHU Dijon Bourgogne Hôpital François Mitterrand
        • Contact:
        • Principal Investigator:
          • Fabienne GENRE VOLOT
      • Marseille, France, 13385
        • Not yet recruiting
        • CHU Timone
        • Contact:
      • Nantes, France, 44093
      • Saint-Étienne, France, 42055

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 45 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: OTHER
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
OTHER: One arm: patients with severe haemophilia A on prophylaxis
Patients with severe haemophilia A (FVIII < 1 IU/dl), currently on prophylactic therapy , having the same prophylaxis regimen in the last six months, aged between 2 (with a body weight ≥12.5 kg ) and 45 years , with adequate venous access, having patient's diary or equivalent regularly completed and able to give informed consent
Other: Thrombin generation measurement.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Time Frame: 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.
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 Outcome Measures

Outcome Measure
Time Frame
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
Time Frame: 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.
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.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hospices Civils de Lyon

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

August 8, 2016

Primary Completion (ANTICIPATED)

September 8, 2019

Study Completion (ANTICIPATED)

September 8, 2019

Study Registration Dates

First Submitted

May 20, 2016

First Submitted That Met QC Criteria

May 23, 2016

First Posted (ESTIMATE)

May 24, 2016

Study Record Updates

Last Update Posted (ACTUAL)

July 10, 2019

Last Update Submitted That Met QC Criteria

July 9, 2019

Last Verified

July 1, 2019

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2012-765

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Haemophilia A

Clinical Trials on Thrombin generation measurement.

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in South Korea

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe