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Safety, Tolerability, and Efficacy of Deferasirox in MDS
This study is currently recruiting participants.
Verified by Gruppo Italiano Malattie EMatologiche dell'Adulto, November 2008
Sponsored by: Gruppo Italiano Malattie EMatologiche dell'Adulto
Information provided by: Gruppo Italiano Malattie EMatologiche dell'Adulto
ClinicalTrials.gov Identifier: NCT00469560
  Purpose

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 Intervention Phase
Myelodysplastic Syndromes
Hemosiderosis
Drug: Deferasirox
Phase III

MedlinePlus related topics: Blood Transfusion and Donation
Drug Information available for: Deferasirox
U.S. FDA Resources
Study Type: Interventional
Study Design: Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title: Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.

Further study details as provided by Gruppo Italiano Malattie EMatologiche dell'Adulto:

Primary Outcome Measures:
  • 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. ]

Secondary Outcome Measures:
  • 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. ]

Estimated Enrollment: 158
Study Start Date: June 2007
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.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00469560

Contacts
Contact: Marco VIGNETTI, Dr. +39 06 441639831 m.vignetti@gimema.it
Contact: Paola FAZI, Dr. +39 06 441639830 p.fazi@gimema.it

Locations
Italy
Oncoematologia "A.O.R.N. S'Anna e S.Sebastiano" Not yet recruiting
Caserta, Italy
Contact: Antonio ABBADESSA            
Ematologia- Università degli Studi "La Sapienza" Recruiting
Roma, Italy
Contact: Giuliana ALIMENA, Pr.            
Università degli Studi di Tor Vergata Recruiting
Roma, Italy
Contact: Sergio AMADORI            
Ospedale "A. Businco" Recruiting
Cagliari, Italy
Contact: Emanuele ANGELUCCI            
Policlinico di Careggi, Università delgi studi di Firenze Recruiting
Firenze, Italy
Contact: Alberto BOSI            
Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza Not yet recruiting
San Giovanni Rotondo, Italy
Contact: Nicola CASCAVILLA            
Ospedale Civile SS. Giovanni e Paolo Not yet recruiting
Venezia, Italy
Contact: Teodoro CHISESI            
US Dipartimentale Centro per le Malattie del Sangue Not yet recruiting
Castelfranco Veneto, Italy
Contact: Ercole DE BIASI            
Ist.Ematologia e Oncologia Medica L.e A. Seragnoli Recruiting
Bologna, Italy
Contact: Giovanni MARTINELLI            
Università di Catania - Cattedra di Ematologia - Ospedale "Ferrarotto" Not yet recruiting
Catania, Italy
Contact: Francesco Di Raimondo            
Clinica Ematologica - Università degli Studi Not yet recruiting
Genova, Italy
Contact: Marco GOBBI            
CTMO-Ematologia Ospedale Binaghi Not yet recruiting
Cagliari, Italy
Contact: Giorgio LA NASA            
U.O. Ematologia, Azienda Ospedaliera Universitaria Senese Not yet recruiting
Siena, Italy
Contact: Francesco LAURIA            
Università Cattolica del Sacro Cuore Not yet recruiting
Roma, Italy
Contact: Giuseppe LEONE            
SOC EMATOLOGIA ASO SS Antonio e Biagio Not yet recruiting
Alessandria, Italy
Contact: Alessandro LEVIS            
Clinica Ematol Università di Perugia, Policlinico Monteluce Not yet recruiting
Perugia, Italy
Contact: Cristina Mecucci            
Divisione di Ematologia e TMO - Ospedale "A. Cardarelli "- Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli" Not yet recruiting
Napoli, Italy
Contact: Felicetto FERRARA            
Sponsors and Collaborators
Gruppo Italiano Malattie EMatologiche dell'Adulto
Investigators
Principal Investigator: Emanuele ANGELUCCI, Pr. Ospedale "A. Businco", Cagliari
  More Information

GIMEMA's Web page  This link exits the ClinicalTrials.gov site

Publications:
Aaronson NK, Ahmedzai S, Bergman B, Bullinger M, Cull A, Duez NJ, Filiberti A, Flechtner H, Fleishman SB, de Haes JC, et al. The European Organization for Research and Treatment of Cancer QLQ-C30: a quality-of-life instrument for use in international clinical trials in oncology. J Natl Cancer Inst. 1993 Mar 3;85(5):365-76.
Alessandrino EP, Amadori S, Barosi G, Cazzola M, Grossi A, Liberato LN, Locatelli F, Marchetti M, Morra E, Rebulla P, Visani G, Tura S; Italian Society of Hematology. Evidence- and consensus-based practice guidelines for the therapy of primary myelodysplastic syndromes. A statement from the Italian Society of Hematology. Haematologica. 2002 Dec;87(12):1286-306.
Anderson LJ, Holden S, Davis B, Prescott E, Charrier CC, Bunce NH, Firmin DN, Wonke B, Porter J, Walker JM, Pennell DJ. Cardiovascular T2-star (T2*) magnetic resonance for the early diagnosis of myocardial iron overload. Eur Heart J. 2001 Dec;22(23):2171-9.
Borgna-Pignatti C, Franchini M, Gandini G, Vassanelli A, De Gironcoli M, Aprili G. Subcutaneous bolus injection of deferoxamine in adult patients affected by onco-hematologic diseases and iron overload. Haematologica. 1998 Sep;83(9):788-90.
Efficace F, Bottomley A, Osoba D, Gotay C, Flechtner H, D'haese S, Zurlo A. Beyond the development of health-related quality-of-life (HRQOL) measures: a checklist for evaluating HRQOL outcomes in cancer clinical trials--does HRQOL evaluation in prostate cancer research inform clinical decision making? J Clin Oncol. 2003 Sep 15;21(18):3502-11.
Caocci G, Baccoli R, Ledda A, Littera R, La Nasa G. A mathematical model for the evaluation of amplitude of hemoglobin fluctuations in elderly anemic patients affected by myelodysplastic syndromes: correlation with quality of life and fatigue. Leuk Res. 2007 Feb;31(2):249-52. Epub 2006 Jun 30.
Galanello R. Evaluation of ICL670, a once-daily oral iron chelator in a phase III clinical trial of beta-thalassemia patients with transfusional iron overload. Ann N Y Acad Sci. 2005;1054:183-5.

Study ID Numbers: MDS0306
Study First Received: May 3, 2007
Last Updated: November 7, 2008
ClinicalTrials.gov Identifier: NCT00469560  
Health Authority: Italy: The Italian Medicines Agency

Keywords provided by Gruppo Italiano Malattie EMatologiche dell'Adulto:
MDS
Chronic transfusional hemosiderosis
Deferasirox
Low and intermediate I risk (IPSS score) Myelodysplastic syndrome and transfusion-induced hemosiderosis.

Study placed in the following topic categories:
Myelodysplastic syndromes
Metabolic Diseases
Precancerous Conditions
Hematologic Diseases
Deferasirox
Myelodysplastic Syndromes
Myelodysplasia
Iron Metabolism Disorders
Preleukemia
Hemosiderosis
Iron Overload
Bone Marrow Diseases
Metabolic disorder
Iron

Additional relevant MeSH terms:
Neoplasms
Pathologic Processes
Disease
Molecular Mechanisms of Pharmacological Action
Syndrome
Iron Chelating Agents
Chelating Agents
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 16, 2009