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
  Purpose

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


Condition Intervention
Thalassemia Major (TM)
Thalassemia Intermedia (TI)
Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes
Other: Physiopathology

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Official Title: Beta-thalassemia and Microparticles

Resource links provided by NLM:


Further study details as provided by Assistance Publique Hopitaux De Marseille:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Enrollment: 33
Study Start Date: March 2010
Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: TM patients
thalassemia major (TM) Need transfusion for survive
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.

Active Comparator: TI patients
thalassemia intermedia (TI) Patients with TI have a milder clinical phenotype than those with TM
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.

Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   15 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01284738

Locations
France
APHM
Marseille, France, 13
Sponsors and Collaborators
Assistance Publique Hopitaux De Marseille
Investigators
Study Director: Isabelle Thuret, Doctor APHM
  More Information

No publications provided

Responsible Party: Assistance Publique Hopitaux De Marseille
ClinicalTrials.gov Identifier: NCT01284738     History of Changes
Other Study ID Numbers: 2010-A00198-31, 2009-18
Study First Received: January 26, 2011
Last Updated: August 28, 2014
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by Assistance Publique Hopitaux De Marseille:
TM
TI
MP

Additional relevant MeSH terms:
Beta-Thalassemia
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on September 14, 2014