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, sickle cell anemia, diamond-blackfan anemia) requiring ongoing blood transfusions

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

  • Documented non-compliance, defined as having taken less than 50% of the prescribed chelation therapy doses in the 12 months prior to study entry
  • Contraindications, unacceptable toxicities and/or documented poor response to locally approved iron chelators despite proper compliance
• History of at least 20 blood transfusions (equivalent to 100 mL/kg of packed red blood cells (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 be discontinued at least 24 hours prior to study entry.)
• Patients who meet the eligibility criteria for any other ongoing Novartis sponsored clinical study protocol with deferasirox and who have geographic access to these sites
• Patients unable to tolerate (or who have unacceptable toxicities to) prior treatment with 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 to baseline
• Pregnancy (as indicated by serum β-HCG pregnancy test at screening for all female patients with the potential to become pregnant) and patients who are breastfeeding
• Patients treated with systemic investigational drug within 4 weeks prior to or with topical investigational drug within 7 days prior to the baseline visit

Other protocol-defined inclusion/exclusion criteria may apply.