Back to resultsSafety, Tolerability, and Efficacy of Deferasirox in MDS
Primary Purpose
Myelodysplastic Syndromes, Hemosiderosis
Status
Completed
Phase
Phase 3
Locations
Italy
Study Type
Interventional
Intervention
Deferasirox
Sponsored by
About this trial
This is an interventional treatment trial for Myelodysplastic Syndromes focused on measuring MDS, Chronic transfusional hemosiderosis, Deferasirox
Eligibility 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
Sites / Locations
- SOC EMATOLOGIA ASO SS Antonio e Biagio
- Ist.Ematologia e Oncologia Medica L.e A. Seragnoli
- CTMO-Ematologia Ospedale Binaghi
- Ospedale "A. Businco"
- Oncoematologia "A.O.R.N. S'Anna e S.Sebastiano"
- US Dipartimentale Centro per le Malattie del Sangue
- Università di Catania - Cattedra di Ematologia - Ospedale "Ferrarotto"
- Policlinico di Careggi, Università delgi studi di Firenze
- Clinica Ematologica - Università degli Studi
- Divisione di Ematologia e TMO - Ospedale "A. Cardarelli "- Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"
- Clinica Ematol Università di Perugia, Policlinico Monteluce
- Ematologia- Università degli Studi "La Sapienza"
- Università Cattolica del Sacro Cuore
- Università degli Studi di Tor Vergata
- Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
- U.O. Ematologia, Azienda Ospedaliera Universitaria Senese
- Ospedale Civile SS. Giovanni e Paolo
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Deferasirox
Arm Description
Outcomes
Primary Outcome Measures
To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis
Secondary Outcome Measures
To evaluate Deferasirox efficacy as chelation therapy in terms of reduction of serum ferritin levels compared to basal levels
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.
Quality of Life evaluation.
Compliance to chelating therapy evaluation.
Full Information
NCT ID
NCT00469560
First Posted
May 3, 2007
Last Updated
November 21, 2016
Sponsor
Gruppo Italiano Malattie EMatologiche dell'Adulto
1. Study Identification
Unique Protocol Identification Number
NCT00469560
Brief Title
Safety, Tolerability, and Efficacy of Deferasirox in MDS
Official Title
Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.
Study Type
Interventional
2. Study Status
Record Verification Date
November 2016
Overall Recruitment Status
Completed
Study Start Date
June 2007 (undefined)
Primary Completion Date
March 2010 (Actual)
Study Completion Date
November 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Gruppo Italiano Malattie EMatologiche dell'Adulto
4. Oversight
Data Monitoring Committee
No
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndromes, Hemosiderosis
Keywords
MDS, Chronic transfusional hemosiderosis, Deferasirox
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
158 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Deferasirox
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Deferasirox
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
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.
Title
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.
Title
Quality of Life evaluation.
Time Frame
At 3, 6, 9, and 12 months from baseline assessment.
Title
Compliance to chelating therapy evaluation.
Time Frame
On a monthly basis thereafter from baseline assessment.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Emanuele ANGELUCCI, Pr.
Organizational Affiliation
Ospedale "A. Businco", Cagliari
Official's Role
Principal Investigator
Facility Information:
Facility Name
SOC EMATOLOGIA ASO SS Antonio e Biagio
City
Alessandria
Country
Italy
Facility Name
Ist.Ematologia e Oncologia Medica L.e A. Seragnoli
City
Bologna
Country
Italy
Facility Name
CTMO-Ematologia Ospedale Binaghi
City
Cagliari
Country
Italy
Facility Name
Ospedale "A. Businco"
City
Cagliari
Country
Italy
Facility Name
Oncoematologia "A.O.R.N. S'Anna e S.Sebastiano"
City
Caserta
Country
Italy
Facility Name
US Dipartimentale Centro per le Malattie del Sangue
City
Castelfranco Veneto
Country
Italy
Facility Name
Università di Catania - Cattedra di Ematologia - Ospedale "Ferrarotto"
City
Catania
Country
Italy
Facility Name
Policlinico di Careggi, Università delgi studi di Firenze
City
Firenze
Country
Italy
Facility Name
Clinica Ematologica - Università degli Studi
City
Genova
Country
Italy
Facility Name
Divisione di Ematologia e TMO - Ospedale "A. Cardarelli "- Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"
City
Napoli
Country
Italy
Facility Name
Clinica Ematol Università di Perugia, Policlinico Monteluce
City
Perugia
Country
Italy
Facility Name
Ematologia- Università degli Studi "La Sapienza"
City
Roma
Country
Italy
Facility Name
Università Cattolica del Sacro Cuore
City
Roma
Country
Italy
Facility Name
Università degli Studi di Tor Vergata
City
Roma
Country
Italy
Facility Name
Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
City
San Giovanni Rotondo
Country
Italy
Facility Name
U.O. Ematologia, Azienda Ospedaliera Universitaria Senese
City
Siena
Country
Italy
Facility Name
Ospedale Civile SS. Giovanni e Paolo
City
Venezia
Country
Italy
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
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8433390
Citation
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Results Reference
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Citation
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Safety, Tolerability, and Efficacy of Deferasirox in MDS
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