Safety, Tolerability, and Efficacy of Deferasirox in MDS
NCT ID: NCT00469560
Last Updated: 2016-11-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
158 participants
INTERVENTIONAL
2007-06-30
2013-11-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
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.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Deferasirox
Deferasirox
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Deferasirox
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* 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
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Gruppo Italiano Malattie EMatologiche dell'Adulto
OTHER
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Emanuele ANGELUCCI, Pr.
Role: PRINCIPAL_INVESTIGATOR
Ospedale "A. Businco", Cagliari
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
SOC EMATOLOGIA ASO SS Antonio e Biagio
Alessandria, , Italy
Ist.Ematologia e Oncologia Medica L.e A. Seragnoli
Bologna, , Italy
CTMO-Ematologia Ospedale Binaghi
Cagliari, , Italy
Ospedale "A. Businco"
Cagliari, , Italy
Oncoematologia "A.O.R.N. S'Anna e S.Sebastiano"
Caserta, , Italy
US Dipartimentale Centro per le Malattie del Sangue
Castelfranco Veneto, , Italy
Università di Catania - Cattedra di Ematologia - Ospedale "Ferrarotto"
Catania, , Italy
Policlinico di Careggi, Università delgi studi di Firenze
Florence, , Italy
Clinica Ematologica - Università degli Studi
Genova, , Italy
Divisione di Ematologia e TMO - Ospedale "A. Cardarelli "- Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"
Napoli, , Italy
Clinica Ematol Università di Perugia, Policlinico Monteluce
Perugia, , Italy
Ematologia- Università degli Studi "La Sapienza"
Roma, , Italy
Università Cattolica del Sacro Cuore
Roma, , Italy
Università degli Studi di Tor Vergata
Roma, , Italy
Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
San Giovanni Rotondo, , Italy
U.O. Ematologia, Azienda Ospedaliera Universitaria Senese
Siena, , Italy
Ospedale Civile SS. Giovanni e Paolo
Venezia, , Italy
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
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. doi: 10.1093/jnci/85.5.365.
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. doi: 10.1053/euhj.2001.2822.
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. doi: 10.1200/JCO.2003.12.121.
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. doi: 10.1016/j.leukres.2006.05.015. 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. doi: 10.1196/annals.1345.021.
Efficace F, Santini V, La Nasa G, Cottone F, Finelli C, Borin L, Quaresmini G, Di Tucci AA, Volpe A, Cilloni D, Quarta G, Sanpaolo G, Rivellini F, Salvi F, Molteni A, Voso MT, Alimena G, Fenu S, Mandelli F, Angelucci E. Health-related quality of life in transfusion-dependent patients with myelodysplastic syndromes: a prospective study to assess the impact of iron chelation therapy. BMJ Support Palliat Care. 2016 Mar;6(1):80-8. doi: 10.1136/bmjspcare-2014-000726. Epub 2014 Sep 9.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
MDS0306
Identifier Type: -
Identifier Source: org_study_id