Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
Condition: Thalassemia; Sickle Cell Disease; Diamond Blackfan Anemia; Myelofibrosis
MeSH terms: Anemia; Anemia, Sickle Cell; Primary Myelofibrosis; Thalassemia; Anemia, Diamond-Blackfan; Iron Overload
Intervention: Deferasirox
Intervention description: 125 mg, 250 mg and 500 mg tablets. Dosage was calculated based on participant's body weight. Tablets were dispersed in water, orange or apple juice and taken orally once a day.
Intervention MeSH terms: Deferasirox
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 1683.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No
Keywords: Deferasirox; Congenital Anemias; Anemias; Red Blood Cell Disorders; Chronic Iron Overload; Transfusional Iron Overload; Iron Chelators; Oral Iron Chelators; Thalassemia; Sickle Cell Disease; Diamond Blackfan Anemia; Myelofibrosis; ICL670A

Summary:

      This is an open-label, non-randomized, multi-center trial designed to provide expanded access
      of deferasirox to patients with congenital disorders of red blood cells and chronic iron
      overload from blood transfusions who cannot adequately be treated with locally approved iron
      chelators.
    

Eligibility criteria:

        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.