Patients presenting with low or INT-1 risk MDS with only mild iron overload will be treated with deferasirox in this study. It will be analyzed if hematological improvement can be observed during this treatment.
Conditions
Interventions
Trade Name: Exjade®
Product Name: Deferasirox
Pharmaceutical Form: Tablet
INN or Proposed INN: Deferasirox
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 125-
INN
Sponsors
Friedrich-Alexander-Universitaet Erlangen-Nuernberg
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -MDS of subtype RA, RARS, RCMD, RCMD-RS (i.e. lower risk) -RAEB I allowed, if clinically stable for > 3 months -5q-minus syndrome allowed, if lenalidomide unsuccessful or unavailable at the time of inclusion -IPSS score 300 µg/l and 8 weeks (4 weeks for G-CSF). -no indication and/or no plans for stem cell transplantation -stable or worsening cytopenia during the past 8 weeks. If in doubt, extend screening period to > 8 weeks -Patients of either gender and age > 18 years -Life expectancy > 12 months -Females of childbearing potential must use double-barrier contraception. -Written informed consent by the patient Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: -Treatment with deferasirox or other chelation therapy for periods > 4 weeks before study start -Patients with a concomitant second malignant disease, possibly interfering with life expectancy -Patients with mean levels of alanine aminotransferase (ALT) > 5x ULN -Patients with uncontrolled systemic hypertension -Patients with serum creatinine > 1.5x the upper limit of normal (ULN) or a creatinine clearance 470 msec on screening ECG -Patients with a history of Torsades de Pointes
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Fraction of patients with hematologic improvement according to modified IWG criteria (reduction of transfusions and/or increase in Hb, improvement of neutropenia and thrombocytopenia);Secondary Objective: - Evaluate the safety and tolerability profile of deferasirox in MDS patients -Effectiveness of iron depletion -Correlation between hematological improvement and effectiveness of iron depletion -Development of bone marrow morphology -Correlation between hematological improvement and pretreatment parameters. Extension of this analysis to MDS patients on deferasirox within the licensed indication (more severe iron overload) -overall survival -AML-free survival ;Primary end point(s): Fraction of patients with hematologic improvement according to modified IWG criteria (reduction of transfusions and/or increase in Hb, improvement of neutropenia and thrombocytopenia) | — |
Countries
Germany
Outcome results
None listed