Clinical Trials Logo

Clinical Trial 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.


Clinical Trial 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

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Double-Blind, Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT00103753
Study type Interventional
Source Royal Brompton & Harefield NHS Foundation Trust
Contact
Status Active, not recruiting
Phase Phase 4
Start date May 2004
Completion date June 2005

See also
  Status Clinical Trial Phase
Completed NCT00069862 - Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia Phase 1/Phase 2
Completed NCT00733811 - Efficacy Study of the Use of Sequential DFP-DFO Versus DFP Phase 4
Completed NCT05506358 - Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada N/A
Withdrawn NCT04938635 - Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia Phase 2
Active, not recruiting NCT03655678 - A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia Phase 2/Phase 3
Completed NCT06239389 - Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study. Phase 2
Recruiting NCT05635266 - Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
Completed NCT03271541 - A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia Phase 2
Terminated NCT02274233 - Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia Phase 1
Completed NCT01206075 - Evaluating the Safety and Effectiveness of Mozobil Mobilization in Adults With Beta-Thalassemia Major N/A
Recruiting NCT05567458 - A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia. Phase 2
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Completed NCT03961828 - Hyalornic Acid Level in β-Thalassemic Children Treated for Hepatitis C Virus Phase 4
Recruiting NCT06065189 - Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major Early Phase 1
Recruiting NCT04143724 - Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia Phase 2
Terminated NCT03381833 - A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia Phase 2
Not yet recruiting NCT01996683 - Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml) N/A
Completed NCT02268409 - ACE-536 Extension Study - Beta Thalassemia Phase 2
Active, not recruiting NCT01016093 - Zoledronic Acid for the Prevention of Bone Loss Post-bone Marrow Transplantation for Thalassemia Major Patients Phase 2/Phase 3
Completed NCT01039636 - Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload Phase 1