Safety, Tolerability, and Efficacy of Deferasirox in MDS

Last updated: November 21, 2016
Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Overall Status: Completed

Phase

3

Condition

Iron Overload (Hemachromatosis)

Myelodysplastic Syndromes (Mds)

White Cell Disorders

Treatment

N/A

Clinical Study ID

NCT00469560
MDS0306
  • Ages > 18
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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 Desferalafter a day of wash out

  • Medical history of at least 20 blood transfusions (equivalent to 100 ml/kg of redcells concentrate).

  • Availability of data concerning blood transfusions during the 12 weeks beforescreening

  • Serum ferritin >= 1000 µg/L at least twice (at least 2 week interval between the 2analysis) during the year before the screening

  • Life expectancy > 12 months

  • Availability of at least 3 complete blood counts (before transfusions) during the 12weeks before the screening

Exclusion

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 (includingthalidomide, azacitidine, arsenic trioxide). These patients become eligible after a "wash out" of at least 4 weeks

  • Concomitant treatment with another iron-chelating agent

Study Design

Total Participants: 158
Study Start date:
June 01, 2007
Estimated Completion Date:
November 30, 2013

Study Description

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.

Connect with a study center

  • SOC EMATOLOGIA ASO SS Antonio e Biagio

    Alessandria,
    Italy

    Site Not Available

  • Ist.Ematologia e Oncologia Medica L.e A. Seragnoli

    Bologna,
    Italy

    Site Not Available

  • CTMO-Ematologia Ospedale Binaghi

    Cagliari,
    Italy

    Site Not Available

  • Ospedale "A. Businco"

    Cagliari,
    Italy

    Site Not Available

  • Oncoematologia "A.O.R.N. S'Anna e S.Sebastiano"

    Caserta,
    Italy

    Site Not Available

  • US Dipartimentale Centro per le Malattie del Sangue

    Castelfranco Veneto,
    Italy

    Site Not Available

  • Università di Catania - Cattedra di Ematologia - Ospedale "Ferrarotto"

    Catania,
    Italy

    Site Not Available

  • Policlinico di Careggi, Università delgi studi di Firenze

    Firenze,
    Italy

    Site Not Available

  • Clinica Ematologica - Università degli Studi

    Genova,
    Italy

    Site Not Available

  • Divisione di Ematologia e TMO - Ospedale "A. Cardarelli "- Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"

    Napoli,
    Italy

    Site Not Available

  • Clinica Ematol Università di Perugia, Policlinico Monteluce

    Perugia,
    Italy

    Site Not Available

  • Ematologia- Università degli Studi "La Sapienza"

    Roma,
    Italy

    Site Not Available

  • Università Cattolica del Sacro Cuore

    Roma,
    Italy

    Site Not Available

  • Università degli Studi di Tor Vergata

    Roma,
    Italy

    Site Not Available

  • Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza

    San Giovanni Rotondo,
    Italy

    Site Not Available

  • U.O. Ematologia, Azienda Ospedaliera Universitaria Senese

    Siena,
    Italy

    Site Not Available

  • Ospedale Civile SS. Giovanni e Paolo

    Venezia,
    Italy

    Site Not Available

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