Safety, Tolerability, and Efficacy of Deferasirox in MDS

• Status: Completed
• Trial end date: 2013-11-01
• Target enrollment: 0
• Participant gender: All

Summary

Open label, single arm study on Deferasirox treatment in MDS patients with chronic transfusional hemosiderosis.

Patients 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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
• Treatments: Deferasirox

Criteria

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