Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

Inclusion Criteria:

• Male or female patients greater than or equal to 2 years of age

• Documented congenital disorder of red blood cells (e.g., β-thalassemia major, sicklecell anemia, diamond-blackfan anemia) requiring ongoing blood transfusions

• Cannot be adequately treated with a locally approved iron chelator due to one of thefollowing reasons:

• Documented non-compliance, defined as having taken less than 50% of theprescribed chelation therapy doses in the 12 months prior to study entry

• Contraindications, unacceptable toxicities and/or documented poor response tolocally approved iron chelators despite proper compliance

• History of at least 20 blood transfusions (equivalent to 100 mL/kg of packed red bloodcells (PRBC])

• Serum ferritin value greater than or equal to 1000 µg/L

• Ability to comply with all study-related procedures, medications, and evaluations

Exclusion Criteria:

• Ongoing treatment with another iron chelator (Any other iron chelation therapy must bediscontinued at least 24 hours prior to study entry.)

• Patients who meet the eligibility criteria for any other ongoing Novartis sponsoredclinical study protocol with deferasirox and who have geographic access to these sites

• Patients unable to tolerate (or who have unacceptable toxicities to) prior treatmentwith deferasirox

• Serum creatinine above the upper limit of normal at screening.

• Patients with ALT ≥ 500 U/L at screening.

• Evidence of chelation-related cataracts or hearing loss within 4 weeks prior tobaseline

• Pregnancy (as indicated by serum β-HCG pregnancy test at screening for all femalepatients with the potential to become pregnant) and patients who are breastfeeding

• Patients treated with systemic investigational drug within 4 weeks prior to or withtopical investigational drug within 7 days prior to the baseline visit Other protocol-defined inclusion/exclusion criteria may apply.