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Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major

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ClinicalTrials.gov Identifier: NCT00103753
Recruitment Status : Unknown
Verified February 2005 by Royal Brompton & Harefield NHS Foundation Trust.
Recruitment status was:  Active, not recruiting
First Posted : February 15, 2005
Last Update Posted : June 24, 2005
Sponsor:
Collaborators:
CORDA, The Heart Charity
The Cooley's Anemia Foundation,
Apotex Inc.
The UK Thalassemia Society
Information provided by:
Royal Brompton & Harefield NHS Foundation Trust

Study Description
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Brief Summary:
Thalassemia major is a genetic disorder affecting hemoglobin synthesis, rendering individuals dependent upon lifelong blood transfusions. Consequently, iron overload occurs and patients have shortened life expectancy with the most common cause of death being heart failure. This trial tests whether the combination of traditional therapy (deferoxamine) with a newer drug (deferiprone) will prove more effective in removing cardiac iron than deferoxamine alone.

Condition or disease Intervention/treatment Phase
Beta-Thalassemia Drug: deferiprone Phase 4

Detailed Description:

Thalassemia Major (TM) is a hereditary anemia resulting from a single gene defect that results in abnormal red cell production. The survival of affected individuals is dependent upon lifelong blood transfusions. Unfortunately, this causes total body iron overload, and 50% of the patients in the UK are dead by the age of 35. Approximately 70% of these deaths result from heart failure which results as a consequence of cardiac iron toxicity.

A Cardiovascular Magnetic Resonance (CMR) technique (which exploits the fact that T2* signal decay relates to tissue iron) developed at the Royal Brompton Hospital provides a non-invasive and reproducible assessment of cardiac iron. CMR therefore provides a very useful method to assess response to new treatments in this condition. Using cardiac T2* as a primary endpoint, we will investigate whether the oral chelator, deferiprone in combination with traditional treatment (deferoxamine), is superior in removing cardiac iron as compared to deferoxamine alone. This trial will provide the first randomized controlled, double-blinded, evidence for the efficacy of combination treatment in TM.

Study Design
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Study Type : Interventional  (Clinical Trial)
Enrollment : 65 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double
Primary Purpose: Treatment
Official Title: A Randomized, Placebo Controlled, Double Blind Trial of the Effect of Combined Therapy With Deferoxamine and Deferiprone on Myocardial Iron in Thalassemia Major Using Cardiovascular Magnetic Resonance
Study Start Date : May 2004
Study Completion Date : June 2005

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Iron Thalassemia

Arms and Interventions
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Outcome Measures
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Primary Outcome Measures :
  1. Myocardial T2*

Secondary Outcome Measures :
  1. Liver T2*
  2. LV and RV volumes and function in systole and diastole
  3. Brachial artery reactivity
  4. B-type natriuretic peptide
  5. Patient compliance
  6. Adverse events
  7. Success of blinding

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Beta thalassemia major
  • Maintaining pre-transfusion hemoglobin of 9 g/dL
  • Myocardial T2* between 8 and 20 ms
  • Ability to give informed consent
  • Male or female
  • Age >18 years
  • Any ejection fraction
  • Confirmation of effective contraception throughout the trial (both men and women)

Exclusion Criteria:

  • Implant incompatible with MR (magnetic resonance), such as pacemaker, claustrophobia, or other condition making CMR impossible or inadvisable
  • Neutropenia within 12 months (ANC <1.5 x10^9/L), unless normal at screening
  • Thrombocytopenia within 12 months (<50 x10^9/L), unless normal at screening
  • Liver enzymes > 3 times upper limit of normal
  • Patients who have previously received deferiprone for a total of more than 6 months over the last 5 years.
  • Patients with a previous reaction to deferiprone
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00103753


Locations
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Italy
Ospedale Microcitemico, Via Jenner
Cagliari, Sardinia, Italy, 09121
Sponsors and Collaborators
Royal Brompton & Harefield NHS Foundation Trust
CORDA, The Heart Charity
The Cooley's Anemia Foundation,
Apotex Inc.
The UK Thalassemia Society
More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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ClinicalTrials.gov Identifier: NCT00103753    
Other Study ID Numbers: 02 065
First Posted: February 15, 2005    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: February 2005
Keywords provided by Royal Brompton & Harefield NHS Foundation Trust:
Randomized Controlled Trial
Deferiprone
Deferoxamine
Iron chelation
Beta Thalassemia Major
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
 Genetic Diseases, Inborn
Deferiprone
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action