Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Tha… (NCT03637556) | Clinical Trial Compass
CompletedPhase 2
Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation
United States, Thailand25 participantsStarted 2019-08-20
Plain-language 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.
Who can participate
Age range
8 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Written informed consent or assent as appropriate obtained prior to any study-related procedure being performed;
. Patients at least 8 years of age or older at the time of consent or assent;
. Patient with TDT syndrome and iron overload currently receiving iron chelation therapy with Jadenu or Exjade and demonstrating inadequate response assessed with serum ferritin and LIC;
. At least 8 or more blood transfusions in the past year;
. Survival expected of \>12 months;
. Patient previously on dual iron chelation therapy will be transfered to iron chelation monotherapy ,stable dosing with Jadenu or Exjade for ≥1month prior to screening and receiving doses in the maximal dose range per day (e.g., Jadenu: \>21 mg/kg or Exjade: \>30 mg/kg, with specific doses in these ranges prescribed at the physician's discretion);
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. Serum ferritin levels that are persistently \>800 mcg/L determined by 2 separate assessments during screening over the previous 2-4 weeks prior to study treatment and not showing a decreasing trend over these weeks OR, an LIC of \>5 mg Fe/g dw measured by MRI in the 52 weeks prior to study entry, OR clearly identified as a poor responder in medical records within 3 months prior to the study
. Compliant with chelation therapy in the 3 months prior to enrollment in the opinion of the Investigator; has taken at least 75% of medication prescribed on a regular basis was taken (Investigator enquiry into patient prescription refill records, preferably 3 months if available, SICT scores); and
Exclusion criteria
. Females of childbearing potential not on an adequate method of birth control, or who are pregnant or lactating;
. History of non-compliance with chelation therapy (determined by the investigator).
. Known history of human immunodeficiency virus (HIV)
. Active hepatitis B virus (HBV), hepatitis C virus (HCV), or other known active viral hepatitis;
. Screening blood counts as follows:
. Absolute neutrophil count \< 1,000/μL
. Platelets \< 50,000/μL
. Hemoglobin \< 7 g/dL (transfusion support is permitted);