Myelodysplastic SyndromesPhase 3NCT00469560
Safety, Tolerability, and Efficacy of Deferasirox in MDS
A Phase 3 study testing Deferasirox in people with Myelodysplastic Syndromes and Hemosiderosis, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 17 sites in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes, Hemosiderosis
- What is being tested?
- Deferasirox
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 158 participants
- Where
- 17 sitesItaly
- Study dates
- Jun 2007to Nov 2013
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
17 sites in 1 country.
17 sites in 1 country
Italy17 sites
SOC EMATOLOGIA ASO SS Antonio e Biagio
Alessandria
Ist.Ematologia e Oncologia Medica L.e A. Seragnoli
Bologna
CTMO-Ematologia Ospedale Binaghi
Cagliari
Ospedale "A. Businco"
Cagliari
Oncoematologia "A.O.R.N. S'Anna e S.Sebastiano"
Caserta
US Dipartimentale Centro per le Malattie del Sangue
Castelfranco Veneto
Università di Catania - Cattedra di Ematologia - Ospedale "Ferrarotto"
Catania
Policlinico di Careggi, Università delgi studi di Firenze
Florence
Clinica Ematologica - Università degli Studi
Genova
Divisione di Ematologia e TMO - Ospedale "A. Cardarelli "- Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"
Naples
Clinica Ematol Università di Perugia, Policlinico Monteluce
Perugia
Ematologia- Università degli Studi "La Sapienza"
Roma
Università Cattolica del Sacro Cuore
Roma
Università degli Studi di Tor Vergata
Roma
Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
San Giovanni Rotondo
U.O. Ematologia, Azienda Ospedaliera Universitaria Senese
Siena
Ospedale Civile SS. Giovanni e Paolo
Venezia
About this study
Official title: Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.
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.
Read the 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.
- Conditions
- Myelodysplastic Syndromes, Hemosiderosis
- Being tested
- Deferasirox
- Keywords
- MDS, Chronic transfusional hemosiderosis, Deferasirox
- Medical subject headings
- Myelodysplastic Syndromes, Hemosiderosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferasiroxDrug
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | Not described Receives: Drug: Deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the tolerability and safety profile of Deferasirox in pts with MDS with post-transfusional hemosiderosis
Measured: On a monthly basis thereafter from baseline assessment.
Secondary outcomes (4)
To evaluate Deferasirox efficacy as chelation therapy in terms of reduction of serum ferritin levels compared to basal levels
Measured: 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.
Measured: On a monthly basis thereafter from baseline assessment.
Quality of Life evaluation.
Measured: At 3, 6, 9, and 12 months from baseline assessment.
Compliance to chelating therapy evaluation.
Measured: On a monthly basis thereafter from baseline assessment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2007 (estimated)
- Study startJun 2007
- Primary completionMar 2010
- Study completionNov 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 158 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gruppo Italiano Malattie EMatologiche dell'Adulto
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Emanuele ANGELUCCI, Pr., Principal investigator, Ospedale "A. Businco", Cagliari
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00469560
- Sponsor's protocol ID
- MDS0306
- Status last verified
- Nov 2016
- First submitted
- 3 May 2007
- Last updated
- 22 Nov 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
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. doi: 10.1093/jnci/85.5.365.
PubMed 8433390Alessandrino 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.
PubMed 12495903Anderson 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.
PubMed 11913479Borgna-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.
PubMed 9825575Efficace 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.
PubMed 12972527Caocci 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.
PubMed 16814382Galanello 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.
PubMed 16339664Efficace 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.
PubMed 25204541
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis.
- ClinicalTrials.gov ID
- NCT00469560
- Acronym / sponsor ID
- MDS0306
- Conditions
- Myelodysplastic Syndromes, Hemosiderosis
- Keywords
- MDS, Chronic transfusional hemosiderosis, Deferasirox
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 158 participants (actual)
- Lead sponsor
- Gruppo Italiano Malattie EMatologiche dell'AdultoUniversity, hospital or other
- Study start
- Jun 2007
- Primary completion
- Mar 2010 (actual)
- Study completion
- Nov 2013 (actual)
- First posted
- 4 May 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deferasirox | Experimental | Drug: Deferasirox |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Deferasirox | Drug | Deferasirox |
Primary outcome
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.
4 secondary outcomes
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.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2016.
- Background: PubMed 8433390Aaronson 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.
- Background: PubMed 12495903Alessandrino 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.
- Background: PubMed 11913479Anderson 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.
- Background: PubMed 9825575Borgna-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.
- Background: PubMed 12972527Efficace 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.
- Background: PubMed 16814382Caocci 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.
- Background: PubMed 16339664Galanello 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.
- Derived from this study: PubMed 25204541Efficace 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Emanuele ANGELUCCI, Pr. principal investigator
Ospedale "A. Businco", Cagliari
17 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Deferasirox in people with Myelodysplastic Syndromes and Hemosiderosis, run by Gruppo Italiano Malattie EMatologiche dell'Adulto at 17 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00469560, which also lists 8 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Nov 2016.