Clinical Trials Resource Center

New Medical Therapies™

Thalassemia

December 19, 2011

Novartis reported results from a phase III trial of Exjade for non-transfusion-dependent thalassemia. This one-year, randomized, double-blind, placebo-controlled pivotal study, THALASSA, enrolled 166 subjects 10 years of age and older with beta-thalassemia intermedia, alpha-thalassemia or Hemoglobin E/beta-thalassemia. The subjects were randomized to Exjade doses of 5 mg/kg/day or 10 mg/kg/day or matching placebo. Exjade at a 10 mg/kg/day starting dose significantly reduced liver iron concentration (LIC) from baseline by 3.8 mg of iron per gram of liver dry weight (Fe/g dw) compared to an increase of 0.38 mg Fe/g dw for placebo (p<0.001). The 10 mg/kg/day dose was superior to a 5 mg/kg/day dose (p≡0.009). In the 10 mg/kg arm, 49% of subjects had a LIC decrease of at least 30% from baseline versus only 2% in the placebo arm. In addition, 56% of subjects in the 10 mg/kg arm had a LIC decrease of greater than or equal to 3 mg at one year compared to 11% in the placebo arm.

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