Ignite Creation Date:
2024-05-06 @ 11:55 AM
Last Modification Date:
2024-10-26 @ 12:52 PM
Study NCT ID:
NCT03637556
Status:
COMPLETED
Last Update Posted:
2022-02-01
First Post:
2018-07-30
Brief Title:
Pilot Study to Assess the Safety PK and Iron Chelating Activity of DST-0509 Deferasirox in Thalassemia Patients Refractory to Chelation
Sponsor:
DisperSol Technologies LLC
Organization:
DisperSol Technologies LLC
Study Overview
Official Title:
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
Status Verified Date:
2022-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 patients 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 0509ExjadeJadenu or ExjadeJadenuDST-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 11 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 patients 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
Up to 36 patients will be randomized 11 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 patients 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None