Thalassemia Major (TM)NCT01284738
Beta-thalassemia and Microparticles
A study testing Physiopathology in people with Thalassemia Major (TM) and Thalassemia Intermedia (TI), run by Assistance Publique Hopitaux De Marseille at 1 site in France.
Trial 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… Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Thalassemia Major (TM), Thalassemia Intermedia (TI), Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes
- What is being tested?
- Physiopathology
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 33 participants
- Where
- 1 siteFrance
- Study dates
- Mar 2010to Feb 2014
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Blood transfusion dating from less than 3 months for TI
- Composite Heterozygotes HbE /beta-thalassemia
- pregnant women
- other disease
Trial locations and contacts
1 site in 1 country.
About this study
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
Read the 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.
- Conditions
- Thalassemia Major (TM), Thalassemia Intermedia (TI), Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes
- Being tested
- Physiopathology
- Keywords
- TM, TI, MP
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PhysiopathologyOther
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.
PhysiopathologyOther
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 group | Role | What they receive |
|---|---|---|
| TM patients | Active comparator | thalassemia major (TM) Need transfusion for survive Receives: Other: Physiopathology |
| TI patients | Active comparator | thalassemia intermedia (TI) Patients with TI have a milder clinical phenotype than those with TM Receives: Other: Physiopathology |
What is being measured
The primary outcome is the main question the study is designed to answer.
Relationship between TM and TI
- 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.
Measured: 36 months
Secondary outcomes (1)
Investigate the mechanisms of the elevated production of MP in thalassemias
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.
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2011 (estimated)
- Study startMar 2010
- Primary completionFeb 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia Major (TM)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique Hopitaux De Marseille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Isabelle Thuret, Doctor, Study director, APHM
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01284738
- Sponsor's protocol ID
- 2010-A00198-31
- Other IDs
- 2009-18
- Status last verified
- Aug 2014
- First submitted
- 26 Jan 2011
- Last updated
- 29 Aug 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Beta-thalassemia and Microparticles
- ClinicalTrials.gov ID
- NCT01284738
- Acronym / sponsor ID
- 2010-A00198-31
- Conditions
- Thalassemia Major (TM), Thalassemia Intermedia (TI), Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes
- Keywords
- TM, TI, MP
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- Assistance Publique Hopitaux De MarseilleUniversity, hospital or other
- Study start
- Mar 2010
- Primary completion
- Feb 2014 (actual)
- First posted
- 27 Jan 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| TM patients | Active comparator | Other: Physiopathologythalassemia major (TM) Need transfusion for survive |
| TI patients | Active comparator | Other: Physiopathologythalassemia intermedia (TI) Patients with TI have a milder clinical phenotype than those with TM |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (4 listed)
- Blood transfusion dating from less than 3 months for TI
- Composite Heterozygotes HbE /beta-thalassemia
- pregnant women
- other disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PhysiopathologyThree 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 | TM patients |
| PhysiopathologyThree 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. | Other | TI patients |
Primary outcome
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.
1 secondary outcome
Investigate the mechanisms of the elevated production of MP in thalassemias
Time frame: 36 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2014.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Isabelle Thuret, Doctor study director
APHM
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Physiopathology in people with Thalassemia Major (TM) and Thalassemia Intermedia (TI), run by Assistance Publique Hopitaux De Marseille at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Aug 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Relationship between TM and TI (time frame: 36 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Aug 2014.