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

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ClinicalTrials.gov Identifier: NCT02781766
Recruitment Status : Recruiting
First Posted : May 24, 2016
Last Update Posted : February 20, 2017
Sponsor:
Information provided by (Responsible Party):
Hospices Civils de Lyon

Brief 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


Condition or disease Intervention/treatment Phase
Haemophilia A Other: Thrombin generation measurement. Not Applicable

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: Thrombin Generation Capacity of Patients With Severe Haemophilia Receiving Prophylaxis
Actual Study Start Date : August 2016
Estimated Primary Completion Date : February 2018
Estimated Study Completion Date : February 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arm Intervention/treatment
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.



Primary Outcome Measures :
  1. 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. ]

Secondary Outcome Measures :
  1. 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. ]


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Ages Eligible for Study:   2 Years to 45 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02781766


Contacts
Contact: Yesim DARGAUD, Pr (0)4.72.11.88.10 ext +33 ydargaud@univ-lyon1.fr

Locations
France
Unité d'Hémostase Clinique Centre Régional de Traitement des Hémophiles Centre de Référence de Maladies Hémorragiques Hôpital Cardiologique Louis Pradel Recruiting
Bron, France, 69500
Contact: Yesim DARGAUD, Pr    (0)4.72.11.88.10 ext +33    ydargaud@univ-lyon1.fr   
Sponsors and Collaborators
Hospices Civils de Lyon

Responsible Party: Hospices Civils de Lyon
ClinicalTrials.gov Identifier: NCT02781766     History of Changes
Other Study ID Numbers: 2012-765
First Posted: May 24, 2016    Key Record Dates
Last Update Posted: February 20, 2017
Last Verified: February 2017

Keywords provided by Hospices Civils de Lyon:
Haemophilia A
prophylaxis
thrombin generation assay
factor VIII
prophylactic therapy

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Thrombin
Coagulants
Hemostatics