Safety, Tolerability, and Efficacy of Deferasirox in MDS
- Registration Number
- NCT00469560
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 158
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Deferasirox Deferasirox -
- Primary Outcome Measures
Name Time Method To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis On a monthly basis thereafter from baseline assessment.
- Secondary Outcome Measures
Name Time Method To evaluate Deferasirox efficacy as chelation therapy in terms of reduction of serum ferritin levels compared to basal levels At 3, 6, 9, and 12 months from baseline assessment. Quality of Life evaluation. At 3, 6, 9, and 12 months from baseline assessment. Compliance to chelating therapy evaluation. On a monthly basis thereafter 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. On a monthly basis thereafter from baseline assessment.
Trial Locations
- Locations (17)
SOC EMATOLOGIA ASO SS Antonio e Biagio
🇮🇹Alessandria, Italy
Clinica Ematologica - Università degli Studi
🇮🇹Genova, Italy
Clinica Ematol Università di Perugia, Policlinico Monteluce
🇮🇹Perugia, Italy
Università Cattolica del Sacro Cuore
🇮🇹Roma, Italy
Università degli Studi di Tor Vergata
🇮🇹Roma, Italy
Ospedale Civile SS. Giovanni e Paolo
🇮🇹Venezia, Italy
U.O. Ematologia, Azienda Ospedaliera Universitaria Senese
🇮🇹Siena, Italy
Ist.Ematologia e Oncologia Medica L.e A. Seragnoli
🇮🇹Bologna, Italy
Policlinico di Careggi, Università delgi studi di Firenze
🇮🇹Firenze, Italy
US Dipartimentale Centro per le Malattie del Sangue
🇮🇹Castelfranco Veneto, Italy
Divisione di Ematologia e TMO - Ospedale "A. Cardarelli "- Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"
🇮🇹Napoli, Italy
CTMO-Ematologia Ospedale Binaghi
🇮🇹Cagliari, Italy
Ematologia- Università degli Studi "La Sapienza"
🇮🇹Roma, Italy
Ospedale "A. Businco"
🇮🇹Cagliari, Italy
Oncoematologia "A.O.R.N. S'Anna e S.Sebastiano"
🇮🇹Caserta, Italy
Università di Catania - Cattedra di Ematologia - Ospedale "Ferrarotto"
🇮🇹Catania, Italy
Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
🇮🇹San Giovanni Rotondo, Italy