Iron Balance Study of Deferasirox, Deferoxamine and the Combination of Both
Status:
Unknown status
Trial end date:
2009-12-01
Target enrollment:
Participant gender:
Summary
Subjects with thalassemia major require regular transfusion therapy to sustain life. The iron
present in the transfused blood remains in the body where it can cause a variety of organ
dysfunctions. Lifelong iron chelation therapy is needed to maintain iron balance but its
effectiveness varies greatly. Like that of deferoxamine (Desferal, DFO) the mainstay of
chelation therapy for 30 years, the effectiveness of deferasirox (Exjade, ICL670), the newly
approved, orally effective iron chelating drug, is not satisfactory in all subjects. Even
with good compliance, the iron excretion induced by a given drug exhibits wide
subject-to-subject variability. There is often persistent iron overload of extra hepatic
tissues such as the heart and pancreas leading to cardiac disease and diabetes. Combining the
drugs may be a better approach in those subjects at increased risk. The iron balance studies
proposed will permit an assessment of the potential of such a combination to place subjects
in net negative iron balance and the relative effectiveness of the combination in relation to
that of the individual drugs, an additive effect being expected. With such information,
physicians will be able to design individualized chelation regimens that maximize
effectiveness while minimizing side effects by adjusting the ratio and/or the dosing schedule
of the two drugs.