Beta-thalassemia and Microparticles

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier:
NCT01284738
First received: January 26, 2011
Last updated: August 28, 2014
Last verified: August 2014

January 26, 2011
August 28, 2014
March 2010
February 2014   (final data collection date for primary outcome measure)
Relationship between TM and TI [ Time Frame: 36 months ] [ Designated as safety issue: No ]
  • In TM, to quantify the elevation of MP as well as their procoagulant activity, to describe their production kinetic, to determine the transfusional or endogenous origin of erythrocytic MP and finally to compare their characteristics with those found in TI patients.
  • To study, in TM and TI patients, the relationship between the number, the procoagulant activity of MP and the clinical (thromboembolic episodes,splenectomy, presence of pulmonary hypertension) biological and plasmatic data reflecting the patient's prothrombotic state.
Same as current
Complete list of historical versions of study NCT01284738 on ClinicalTrials.gov Archive Site
Investigate the mechanisms of the elevated production of MP in thalassemias [ Time Frame: 36 months ] [ Designated as safety issue: No ]
Studying the correlation between the number, the activity of erythrocytes and platelets derived-MP and the hemolysis, the dyserythropoiesis, the oxidative stress and iron overload markers.
Same as current
Not Provided
Not Provided
 
Beta-thalassemia and Microparticles
Beta-thalassemia and Microparticles

The results will allow us to evaluate the role of MP in the thrombo-embolic risk observed in thalassemic patients and to underline a possible difference between TM and TI. The in vitro and in vivo study of MP in erythrocytes concentrates is a new approach to explore the consequence of transfusion in polytransfused patients. Finally, the identification of a possible relationship between the oxidative stress and the production of MP may lead to the development of specific therapeutical approaches

Microparticles (MP) are intact vesicles derived from cell membranes which arise mainly through cell membrane activation processes and from apoptosis. MP originating from platelets, endothelial cells and monocytes have been most extensively studied, though similar particles can arise from red cells and granulocytes. The ability to form microparticles is an essential part of physiological coagulation.However, MP may play an important procoagulant role in several diseases including sickle cell disease, and paroxysmal nocturnal haemoglobinuria (PNH).

Several studies reported the presence of MP in TI and their potential role in the hypercoagulable state. The investigators propose in this study to investigate the presence and origin of MP in TM patients.

Interventional
Not Provided
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
  • Thalassemia Major (TM)
  • Thalassemia Intermedia (TI)
  • Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes
  • Other: Physiopathology

    Three sequential biological evaluations will be performed for each patient and will consist in :

    • the dosages of MP carried out by the UMR 608 in Marseille,
    • the evaluation of the oxidative stress markers and of iron performed in the UMR 773 in Paris-Bichat.

    In vitro production of MP of transfused red blood cells origin will also be evaluated in erythrocytes concentrates during the storage of the units.

  • Other: Physiopathology

    Three sequential biological evaluations will be performed for each patient and will consist in :

    • the dosages of MP carried out by the UMR 608 in Marseille,
    • the evaluation of the oxidative stress markers and of iron performed in the UMR 773 in Paris-Bichat.
  • Active Comparator: TM patients
    thalassemia major (TM) Need transfusion for survive
    Intervention: Other: Physiopathology
  • Active Comparator: TI patients
    thalassemia intermedia (TI) Patients with TI have a milder clinical phenotype than those with TM
    Intervention: Other: Physiopathology
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
33
Not Provided
February 2014   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Patient recorded in the national register of the patients attained by beta-thalassemia (TI) or (TM)
  • Patient monitoring in one of 5 recruiters centers
  • Patient more than 15 years
  • Patient consented and informed

Exclusion Criteria:

  • Blood transfusion dating from less than 3 months for TI
  • Composite Heterozygotes HbE /beta-thalassemia
  • pregnant women
  • other disease
Both
15 Years and older
No
Contact information is only displayed when the study is recruiting subjects
France
 
NCT01284738
2010-A00198-31, 2009-18
No
Assistance Publique Hopitaux De Marseille
Assistance Publique Hopitaux De Marseille
Not Provided
Study Director: Isabelle Thuret, Doctor APHM
Assistance Publique Hopitaux De Marseille
August 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP