Combined Chelation Treatment With Deferiprone and Deferoxamine in Thalassemia Major

• Status: Unknown status
• Trial end date: 2005-06-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Royal Brompton & Harefield NHS Foundation Trust
• Collaborators: Apotex Inc.; CORDA, The Heart Charity; The Cooley's Anemia Foundation,; The Cooley’s Anemia Foundation,; The UK Thalassemia Society
• Treatments: Deferiprone; Deferoxamine

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