TGA (Thrombin Generation Assay) and Prophylaxis in Haemophilia (HOPE)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02781766|
Recruitment Status : Recruiting
First Posted : May 24, 2016
Last Update Posted : February 20, 2017
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)|
|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|
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.
- 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 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. ]
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
|Contact: Yesim DARGAUD, Pr||(0)188.8.131.52.10 ext +email@example.com|
|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)184.108.40.206.10 ext +33 firstname.lastname@example.org|