Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
Condition: Chronic Iron Overload Due to Transfusion-dependant Anemias
MeSH terms: Iron Overload
Intervention: Deferasirox dispersible tablet; Defearisox film-coated tablet
Intervention description: Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.; Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
Intervention MeSH terms: Deferasirox
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 173.0
Sex: All
Minimum age: 10 Years
Healthy volunteers: No
Keywords: Iron overload; Chelation; Thalassemia; Myelodysplastic syndrome (MDS); Transfusional hemisiderosis; Deferasirox; ICL670; Dispersible tablet; Film-coated tablet

Summary:

      Assessed the new film-coated tablet formulation to the currently approved dispersible tablet
      formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability,
      satisfaction and compliance
    

Eligibility criteria:

        Key Inclusion Criteria:

          -  Male and female patients aged ≥ 10 years

          -  Patients with transfusion-dependent thalassemia and iron overload, requiring
             deferasirox DT at doses of ≥ 30 mg/kg/day as per the investigator's decision OR
             Patients with very low, low or intermediate (int) risk myelodysplastic syndrome (MDS)
             and iron overload, requiring deferasirox DT at doses of ≥ 20 mg/kg/day as per the
             investigator's decision.

          -  History of transfusion of at least 20 PRBC units and anticipated to be transfused with
             at least 8 units of PRBCs annually during the study

          -  Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the
             mean value will be used for eligibility criteria).

        Key Exclusion Criteria:

          -  Creatinine clearance below the contraindication limit in the locally approved
             prescribing information. Creatinine clearance will be estimated from serum creatinine
             at screening Visit 1 and screening Visit 2 and the mean value will be used for
             eligibility criteria.

          -  Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and screening
             Visit 2 (the mean value will be used for eligibility criteria).

          -  ALT (SGPT) > 5xULN, unless LIC confirmed as >10 mg Fe/dw within 6 months prior to
             screening visit 1.

          -  Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg
             in a non-first void urine sample at screening Visit 1 or screening Visit 2.

          -  Patients with significant impaired gastrointestinal (GI) function or GI disease that
             may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases,
             uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel
             resection).

          -  Liver disease with severity of Child-Pugh Class B or C