Beta-thalassemia and Microparticles

• ClinicalTrials.gov Identifier: NCT01284738
• Recruitment Status: Completed
• First Posted: January 27, 2011
• Last Update Posted: August 29, 2014
• Sponsor: Assistance Publique Hopitaux De Marseille
• Information provided by (Responsible Party): Assistance Publique Hopitaux De Marseille

Tracking Information

• First Submitted Date: January 26, 2011
• First Posted Date: January 27, 2011
• Last Update Posted Date: August 29, 2014
• Study Start Date: March 2010
• Actual Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: January 26, 2011)

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.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: January 26, 2011)

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.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Beta-thalassemia and Microparticles
• Official Title: Beta-thalassemia and Microparticles
• Brief Summary: 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

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
• Study Phase: Not Applicable
• Study Design: Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: None (Open Label)

Condition

• Thalassemia Major (TM)
• Thalassemia Intermedia (TI)
• Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes

Intervention

• 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.

Study Arms

• 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

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 28, 2014): 33
• Original Estimated Enrollment (submitted: January 26, 2011): 55
• Study Completion Date: Not Provided
• Actual Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 15 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: France

Administrative Information

• NCT Number: NCT01284738
• Other Study ID Numbers: 2010-A00198-31; 2009-18
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Assistance Publique Hopitaux De Marseille
• Original Responsible Party: APHM, Direction de la recherche
• Current Study Sponsor: Assistance Publique Hopitaux De Marseille
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Study Director: Isabelle Thuret, Doctor; APHM

• PRS Account: Assistance Publique Hopitaux De Marseille
• Verification Date: August 2014