Safety, Tolerability, and Efficacy of Deferasirox in MDS

This study has been completed.
Sponsor:
Information provided by:
Gruppo Italiano Malattie EMatologiche dell'Adulto
ClinicalTrials.gov Identifier:
NCT00469560
First received: May 3, 2007
Last updated: November 24, 2010
Last verified: November 2010
  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 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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.

Resource links provided by NLM:


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. ] [ Designated as safety issue: No ]

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. ] [ Designated as safety issue: No ]
  • 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. ] [ Designated as safety issue: No ]
  • Quality of Life evaluation. [ Time Frame: At 3, 6, 9, and 12 months from baseline assessment. ] [ Designated as safety issue: No ]
  • Compliance to chelating therapy evaluation. [ Time Frame: On a monthly basis thereafter from baseline assessment. ] [ Designated as safety issue: No ]

Estimated Enrollment: 158
Study Start Date: June 2007
Primary Completion Date: March 2010 (Final data collection date for primary outcome measure)
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00469560

Locations
Italy
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
Firenze, 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
Università degli Studi di Tor Vergata
Roma, Italy
Università Cattolica del Sacro Cuore
Roma, Italy
Ematologia- Università degli Studi "La Sapienza"
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
Sponsors and Collaborators
Gruppo Italiano Malattie EMatologiche dell'Adulto
Investigators
Principal Investigator: Emanuele ANGELUCCI, Pr. Ospedale "A. Businco", Cagliari
  More Information

Additional Information:
Publications:

ClinicalTrials.gov Identifier: NCT00469560     History of Changes
Other Study ID Numbers: MDS0306
Study First Received: May 3, 2007
Last Updated: November 24, 2010
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.

Additional relevant MeSH terms:
Myelodysplastic Syndromes
Preleukemia
Syndrome
Hemosiderosis
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Neoplasms
Disease
Pathologic Processes
Iron Overload
Iron Metabolism Disorders
Metabolic Diseases
Deferasirox
Iron Chelating Agents
Chelating Agents
Sequestering Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 18, 2014