Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.
18 Years
N/A
Both
Myelodysplastic Syndromes, Hemosiderosis
Trial Information
Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.
It has been widely shown that an appropriate chelating therapy in chronic anemias
transfusion dependent can prevent the overstock of iron and can reduce the already existing
overstock reducing, then, the co-morbidity and improving survival.
In particular, some authors have shown in MDS affected patients undergoing intensive
chelating therapy with deferoxamine haematological recovery with a reduction of the need of
transfusions.
With the present study, we plan to evaluate the safety and efficacy of a therapy with the
new oral chelating Deferasirox in MDS patients with transfusional hemosiderosis.
This is an open label, single arm study on Deferasirox treatment in MDS patients with
chronic transfusional hemosiderosis.
Patients will receive daily oral dosis of Deferasirox in order to eliminate the quantity of
iron administered during transfusions and, if needed, to reduce the overload of already
present iron.
After an screening phase in which patients are evaluated according to eligibility criteria,
a one year treatment phase foresees monthly visits to evaluate safety and efficacy signs.
Inclusion Criteria:
- Patients, both males and females, with low and intermediate I risk (IPSS score)
Myelodysplastic syndrome and transfusion-induced hemosiderosis.
- Age >=18 years
- Patients who never received chelation therapy or who received a therapy with Desferal
after a day of wash out
- Medical history of at least 20 blood transfusions (equivalent to 100 ml/kg of red
cells concentrate).
- Availability of data concerning blood transfusions during the 12 weeks before
screening
- Serum ferritin >= 1000 µg/L at least twice (at least 2 week interval between the 2
analysis) during the year before the screening
- Life expectancy > 12 months
- Availability of at least 3 complete blood counts (before transfusions) during the 12
weeks before the screening
Exclusion Criteria:
- Diagnosis different from MDS (i.e. myelofibrosis)
- Severe renal impairment (creatinine clearance < 60 ml/min)
- ALT/AST > 500 U/L
- Active B and/or C hepatitis
- Patients treated during the past 4 weeks with experimental drugs for MDS (including
thalidomide, azacitidine, arsenic trioxide). These patients become eligible after a
"wash out" of at least 4 weeks
- Concomitant treatment with another iron-chelating agent
Type of Study:
Interventional
Study Design:
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Outcome Measure:
To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis
Outcome Time Frame:
On a monthly basis thereafter from baseline assessment.
Safety Issue:
No
Principal Investigator
Emanuele ANGELUCCI, Pr.
Investigator Role:
Principal Investigator
Investigator Affiliation:
Ospedale "A. Businco", Cagliari
Authority:
Italy: The Italian Medicines Agency
Study ID:
MDS0306
NCT ID:
NCT00469560
Start Date:
June 2007
Completion Date:
Related Keywords:
- Myelodysplastic Syndromes
- Hemosiderosis
- MDS
- Chronic transfusional hemosiderosis
- Deferasirox
- Low and intermediate I risk (IPSS score) Myelodysplastic syndrome and transfusion-induced hemosiderosis.
- Hemosiderosis
- Myelodysplastic Syndromes
- Preleukemia
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