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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

February 14, 2005
February 15, 2005
June 24, 2005
May 2004
Not Provided
Myocardial T2*
Same as current
No Changes Posted
  • Liver T2*
  • LV and RV volumes and function in systole and diastole
  • Brachial artery reactivity
  • B-type natriuretic peptide
  • Patient compliance
  • Adverse events
  • Success of blinding
Same as current
Not Provided
Not Provided
 
Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major
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
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.

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.

Interventional
Phase 4
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double
Primary Purpose: Treatment
Beta-Thalassemia
Drug: deferiprone
Not Provided
Tanner MA, Galanello R, Dessi C, Smith GC, Westwood MA, Agus A, Pibiri M, Nair SV, Walker JM, Pennell DJ. Combined chelation therapy in thalassemia major for the treatment of severe myocardial siderosis with left ventricular dysfunction. J Cardiovasc Magn Reson. 2008 Feb 25;10:12. doi: 10.1186/1532-429X-10-12.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Unknown status
65
Same as current
June 2005
Not Provided

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
Sexes Eligible for Study: All
18 Years and older   (Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Italy
 
 
NCT00103753
02 065
Not Provided
Not Provided
Not Provided
Not Provided
Royal Brompton & Harefield NHS Foundation Trust
  • CORDA, The Heart Charity
  • The Cooley’s Anemia Foundation,
  • Apotex Inc.
  • The UK Thalassemia Society
Not Provided
Royal Brompton & Harefield NHS Foundation Trust
February 2005

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP