Beta-thalassemia and Microparticles

August 28, 2014 updated by: Assistance Publique Hopitaux De Marseille
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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

33

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
      • Marseille, France, 13
        • APHM

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

15 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

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

  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.

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: 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.

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Relationship between TM and TI
Time Frame: 36 months
  • 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.
36 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Investigate the mechanisms of the elevated production of MP in thalassemias
Time Frame: 36 months
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.
36 months

Collaborators and Investigators

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

Sponsor

Assistance Publique Hopitaux De Marseille

Investigators

  • Study Director: Isabelle Thuret, Doctor, APHM

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

March 1, 2010

Primary Completion (Actual)

February 1, 2014

Study Registration Dates

First Submitted

January 26, 2011

First Submitted That Met QC Criteria

January 26, 2011

First Posted (Estimate)

January 27, 2011

Study Record Updates

Last Update Posted (Estimate)

August 29, 2014

Last Update Submitted That Met QC Criteria

August 28, 2014

Last Verified

August 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2010-A00198-31
  • 2009-18

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.

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