Safety, Tolerability, and Efficacy of Deferasirox in MDS

Brief Title

Safety, Tolerability, and Efficacy of Deferasirox in MDS

Official Title

Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.

Brief 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.
    

Detailed 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.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis

Secondary Outcome

 To evaluate Deferasirox efficacy as chelation therapy in terms of reduction of serum ferritin levels compared to basal levels

Condition

Myelodysplastic Syndromes

Intervention

Deferasirox

Study Arms / Comparison Groups

 Deferasirox
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

158

Start Date

June 2007

Completion Date

November 2013

Primary Completion Date

March 2010

Eligibility 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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Emanuele ANGELUCCI, Pr., , 

Location Countries

Italy

Location Countries

Italy

Administrative Informations


NCT ID

NCT00469560

Organization ID

MDS0306


Responsible Party

Sponsor

Study Sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto


Study Sponsor

Emanuele ANGELUCCI, Pr., Principal Investigator, Ospedale "A. Businco", Cagliari


Verification Date

November 2016