Prescribing information

 

    

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.
HCP20-C005n June 2020
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Adverse events should be reported. Reporting forms and information can be found at yellowcard.mhra.gov.uk. Adverse events should also be reported to Novartis via [email protected] or online through the pharmacovigilance intake (PVI) tool at http://www.report.novartis.com/
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