NCT03637556

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

A Multi-Center, Open-Label, Two-Period Cross-Over, Patient-Pilot Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Iron Chelating Activity of DST-0509 (Deferasirox) Tablets in Thalassemia Patients With Inadequate Response to Standard Chelation Therapy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (actual)
• Sponsor: DisperSol Technologies, LLC · Industry
• Sex: All
• Age: 8 Years
• Healthy volunteers: Not accepted

Summary

This study is a multicenter, open-label, two-period crossover design that evaluates the safety, tolerability, pharmacokinetics and preliminary evidence of iron chelating activity of DST-0509 as compared to Jadenu and Exjade in transfusion-dependent thalassemia patients with transfusional iron overload, requiring iron chelation therapy and demonstrating an inadequate response to Jadenu or Exjade for greater than 3 months duration. Up to 36 patients will be evaluated (18 in each treatment arm), however, the balanced randomization may enroll fewer patients based on recruitment status.

Detailed 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 6-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 0509→Exjade/Jadenu or Exjade/Jadenu→DST-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 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 6-day washout period. A sufficient number of patients will be enrolled so there will be no need to replace study drop-outs. Study duration is approximately 14 weeks for each patient.

Conditions

• Thalassemia Major

Interventions

Type	Name	Description
DRUG	DST-0509	Novel iron chelator with improved absorption characteristics
DRUG	Jadenu	Commercial iron chelator with standard absorption characteristics
DRUG	Exjade	Commercial iron chelator with standard absorption characteristics

Timeline

• Start date: 2019-08-20
• Primary completion: 2021-06-08
• Completion: 2021-08-16
• First posted: 2018-08-20
• Last updated: 2022-02-01

Locations

4 sites across 2 countries: United States, Thailand

Regulatory

• FDA-regulated drug study