Patients with beta-thalassemia (Cooley's Anemia) continue to suffer from the
transfusion-induced iron overload due to the inadequacies of current iron-chelation therapy.
Compliance with the use of the only FDA-approved drug for removing excess iron from patients
(Desferal) continues to be a major problem despite convincing evidence that it markedly
reduces morbidity and prolongs life. The full potential of iron-chelation therapy will not be
realized until an orally-effective drug is available. This small trial is testing the premise
that a combination of drugs as a new approach to iron chelation therapy may reduce side
effects and increase efficacy. If both drugs can be given orally, there may be a better
chance of finding a suitable alternative to Desferal. Several combinations of experimental
iron chelating drugs are being used in this trial.
Phase:
Phase 1
Details
Lead Sponsor:
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)