Last updated on September 2018

Pilot Study to Assess the Safety PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation


Brief description of study

Open-label, two-period cross-over, patient-pilot study evaluating the safety, overall tolerability, and preliminary evidence of DST-0509 (investigational deferasirox tablets) iron chelating activity compared to commercial Jadenu or Exjade in patients with transfusion-dependent beta thalassemia (TDT), major or intermedia, or non-TDT (NTDT) patients with inadequate response to their current iron chelation therapy (ICT).

Detailed Study Description

This is a multi-center, open-label, two-period cross-over, patient-pilot study comparing DST-0509 to patient's prior ICT (Exjade or Jadenu) administered orally once daily (QD) for 28-days in each period, with a 7-day washout before the first treatment period, between treatment periods, and at the end of the study before patients recommence their prescription regimens. Patients will be randomized to one of two treatment sequences: DST 0509Exjade/Jadenu or Exjade/JadenuDST-0509 (with subjects who were taking Exjade prior to study start receiving Exjade and those taking Jadenu at study start receiving Jadenu). This study is designed to assess the safety, tolerability, evidence of iron chelating activity, and pharmacokinetics (PK) profile of DST-0509 compared to Jadenu or Exjade in inadequately responding patients.

Up to 36 patients will be randomized 1:1 into one of two treatment sequences (study arms), of which up to 100% may be on Jadenu or Exjade at study entry, or a mix of the two. The planned randomization will assign up to 18 patients in each of two sequences: Sequence A: DST-0509 crossed to Exjade or Jadenu; Sequence B: Jadenu or Exjade crossed to DST-0509. The comparator treatment will be the patient's current chelation treatment. At the end of the study, patients previously on Jadenu or Exjade will revert to receiving their pre-study medication and dose following a 7-day washout period.

Clinical Study Identifier: NCT03637556

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John L. Cosby, DO

Oceane 7 Medical & Research Center, Inc.
Miami, FL United States
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