Date: 2011-12-13
Type of information:
phase: 3
Announcement: results
Company: Novartis (Switzerland)
Product: Exjade® (deferasirox)
Action mechanism: Exjade® is an iron chelator.
Disease: non-transfusion-dependent thalassemia (NTDT)
Therapeutic area: Hematological diseases - Genetic diseases
Country:
Trial
details: THALASSA was a one-year, randomized, double-blind, placebo-controlled pivotal study, including 166 patients with beta-thalassemia intermedia (n=95), alpha-thalassemia (n=22) or Hemoglobin E/beta-thalassemia (n=49). Patients older than 10 years of age with LIC greather than or equal to 5 mg Fe/g dw and serum ferritin >300 ng/mL were randomized to starting Exjade® doses of 5 mg/kg/day (n=55) or matching placebo (n=28) and 10 mg/kg/day (n=55) or matching placebo (n=28).
Latest
news: Results from THALASSA, the first pivotal placebo-controlled study examining the benefit of iron chelation with Exjade® (deferasirox) in patients with non-transfusion-dependent thalassemia (NTDT), show that Exjade® can significantly reduce iron overload. These data were presented at the 53rd Annual Meeting of the American Society of Hematology in San Diego. THALASSA investigated whether patients with NTDT and iron overload can benefit from iron chelation therapy as determined by liver iron concentration (LIC). The study met its primary endpoint, showing that Exjade® at a 10mg/kg/day starting dose significantly reduced 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 in patients on placebo (p<0.001). The study also determined that a 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 patients had a LIC decrease of at least 30% from baseline versus only 2% in the placebo arm. In addition, 56% of patients in the 10 mg/kg arm had a LIC decrease greather than or equal to 3 mg at one year compared to 11% in the placebo arm. The most common adverse events reported werenausea, rash, diarrhea, headache and upper abdominal pain. Adverse events were similar in all patient groups, including the placebo arm. These data will serve as basis for first regulatory filings in US and Europe by year end.
