Clinical Trial: Safety, Tolerability, and Efficacy of Deferasirox in MDS
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
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 Summary:
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.
Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Current Primary Outcome: To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis [ Time Frame: On a monthly basis thereafter from baseline assessment. ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- To evaluate Deferasirox efficacy as chelation therapy in terms of reduction of serum ferritin levels compared to basal levels [ Time Frame: At 3, 6, 9, and 12 months from baseline assessment. ]
- To evaluate the impact Deferasirox iron chelating therapy vs the normal demand of transfusions in a subgroup of pts that will not receive growth factors or chemotherapy according to their basal characteristics. [ Time Frame: On a monthly basis thereafter from baseline assessment. ]
- Quality of Life evaluation. [ Time Frame: At 3, 6, 9, and 12 months from baseline assessment. ]
- Compliance to chelating therapy evaluation. [ Time Frame: On a monthly basis thereafter from baseline assessment. ]
Original Secondary Outcome: Same as current
Information By: Gruppo Italiano Malattie EMatologiche dell'Adulto
Dates:
Date Received: May 3, 2007
Date Started: June 2007
Date Completion:
Last Updated: November 21, 2016
Last Verified: November 2016
