Objective¹

A randomised, multi-centre, open-label, Phase II study evaluating the safety of EXJADE DT and EXJADE FCT formulations in patients with transfusion-dependent thalassaemia or an IPSS score of very-low-, low- or intermediate-risk MDS.

Trial cohort¹

Overall, 173 patients were randomised in a 1:1 ratio where 86 patients received EXJADE DT and 87 patients received EXJADE FCT.

Key inclusion criteria¹

• ≥10 years of age
• Patients with transfusion-dependent thalassaemia or an IPSS score of very-low-, low-, or intermediate-risk MDS
• Chelation-naïve or pre-treated patients
• Transfusion history of ≥20 pRBC units
• Anticipated transfusion requirements of ≥8 units/year during study
• Serum ferritin >1000 ng/mL

EXJADE treatment regimen¹

• For chelation-naïve patients, the starting dose was 20 mg/kg/day with EXJADE DT or 14 mg/kg/day with EXJADE FCT
• For previously treated patients, a washout period of 5 days was required
  • All pre-treated patients previously treated with EXJADE DT, deferoxamine or deferiprone were requested to start on an EXJADE DT or EXJADE FCT dose equivalent to their pre-washout dose
• Dose adjustments were used to improve treatment response based on serum ferritin levels and investigator’s judgment. Dose adjustments were recommended to be:
  • Every 4 weeks for chelation-naïve patients
  • Every 3 months for pre-treated patients
  • In increments of 5–10 mg/kg/day for patients receiving DT
  • In increments of 3.5–7 mg/kg/day for patients receiving FCT
  • Up to a maximum dose of 40 mg/kg/day for DT and 28 mg/kg/day for FCT
• Dose adjustments based on safety and dose reductions for patients unable to tolerate the protocol-specified dosing schedule were allowed at any time during the study

Primary endpoint¹

• The overall safety of EXJADE DT and EXJADE FCT, measured by frequency and severity of adverse events, and changes in laboratory values from BL over 24 weeks

Secondary endpoints¹

• Pharmacokinetics of EXJADE DT and EXJADE FCT
• Patient-reported outcomes of EXJADE DT and EXJADE FCT

Abbreviations: BL, baseline; DT, dispersible tablets; FCT, film-coated tablets; ICT, iron chelation therapy; IPSS, International Prognostic Scoring System; MDS, myelodysplastic syndromes; pRBC, packed red blood cells.

Reference

1. Taher A, et al. New film-coated tablet formulation of deferasirox is well tolerated in patients with thalassemia or lower-risk MDS: Results of the randomized, Phase II ECLIPSE study. Am J Hematol 2017;92(5):420–428.