Safety, Tolerability, and Efficacy of Deferasirox in MDS

Sponsor

Gruppo Italiano Malattie EMatologiche dell'Adulto (Other)

Overall Status

Completed

CT.gov ID

NCT00469560

Collaborator

(none)

158

Enrollment

Locations

Arm

Duration (Months)

9.3

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Myelodysplastic Syndromes Hemosiderosis	Drug: Deferasirox	Phase 3

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 Design

Study Type:

Interventional

Actual Enrollment :

158 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Jun 1, 2007

Actual Primary Completion Date :

Mar 1, 2010

Actual Study Completion Date :

Nov 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Deferasirox	Drug: Deferasirox

Arm

Intervention/Treatment

Experimental: Deferasirox

Drug: Deferasirox

Outcome Measures

Primary Outcome Measures

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

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.]
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.]
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.]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	Country
1	SOC EMATOLOGIA ASO SS Antonio e Biagio	Alessandria	Italy
2	Ist.Ematologia e Oncologia Medica L.e A. Seragnoli	Bologna	Italy
3	CTMO-Ematologia Ospedale Binaghi	Cagliari	Italy
4	Ospedale "A. Businco"	Cagliari	Italy
5	Oncoematologia "A.O.R.N. S'Anna e S.Sebastiano"	Caserta	Italy
6	US Dipartimentale Centro per le Malattie del Sangue	Castelfranco Veneto	Italy
7	Università di Catania - Cattedra di Ematologia - Ospedale "Ferrarotto"	Catania	Italy
8	Policlinico di Careggi, Università delgi studi di Firenze	Firenze	Italy
9	Clinica Ematologica - Università degli Studi	Genova	Italy
10	Divisione di Ematologia e TMO - Ospedale "A. Cardarelli "- Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"	Napoli	Italy
11	Clinica Ematol Università di Perugia, Policlinico Monteluce	Perugia	Italy
12	Ematologia- Università degli Studi "La Sapienza"	Roma	Italy
13	Università Cattolica del Sacro Cuore	Roma	Italy
14	Università degli Studi di Tor Vergata	Roma	Italy
15	Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza	San Giovanni Rotondo	Italy
16	U.O. Ematologia, Azienda Ospedaliera Universitaria Senese	Siena	Italy
17	Ospedale Civile SS. Giovanni e Paolo	Venezia	Italy