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
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Tracking Information | |||
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First Submitted Date ICMJE | February 14, 2005 | ||
First Posted Date ICMJE | February 15, 2005 | ||
Last Update Posted Date | June 24, 2005 | ||
Study Start Date ICMJE | May 2004 | ||
Primary Completion Date | Not Provided | ||
Current Primary Outcome Measures ICMJE |
Myocardial T2* | ||
Original Primary Outcome Measures ICMJE | Same as current | ||
Change History | No Changes Posted | ||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE | Same as current | ||
Current Other Pre-specified Outcome Measures | Not Provided | ||
Original Other Pre-specified Outcome Measures | Not Provided | ||
Descriptive Information | |||
Brief Title ICMJE | Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major | ||
Official Title ICMJE | 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 | ||
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. | ||
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. |
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Study Type ICMJE | Interventional | ||
Study Phase ICMJE | Phase 4 | ||
Study Design ICMJE | Allocation: Randomized Intervention Model: Single Group Assignment Masking: Double Primary Purpose: Treatment |
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Condition ICMJE | Beta-Thalassemia | ||
Intervention ICMJE | Drug: deferiprone | ||
Study Arms ICMJE | Not Provided | ||
Publications * | 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. |
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Recruitment Information | |||
Recruitment Status ICMJE | Unknown status | ||
Enrollment ICMJE |
65 | ||
Original Enrollment ICMJE | Same as current | ||
Study Completion Date ICMJE | June 2005 | ||
Primary Completion Date | Not Provided | ||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||
Accepts Healthy Volunteers ICMJE | No | ||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||
Listed Location Countries ICMJE | Italy | ||
Removed Location Countries | |||
Administrative Information | |||
NCT Number ICMJE | NCT00103753 | ||
Other Study ID Numbers ICMJE | 02 065 | ||
Has Data Monitoring Committee | Not Provided | ||
U.S. FDA-regulated Product | Not Provided | ||
IPD Sharing Statement ICMJE | Not Provided | ||
Responsible Party | Not Provided | ||
Study Sponsor ICMJE | Royal Brompton & Harefield NHS Foundation Trust | ||
Collaborators ICMJE |
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Investigators ICMJE | Not Provided | ||
PRS Account | Royal Brompton & Harefield NHS Foundation Trust | ||
Verification Date | February 2005 | ||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |