Safety, Tolerability, and Efficacy of Deferasirox in MDS
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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. |
- 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 ]
- 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) |
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 |
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| 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 | |
| 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:
|
Hemosiderosis Myelodysplastic Syndromes Preleukemia Iron Overload Iron Metabolism Disorders Metabolic Diseases Bone Marrow Diseases Hematologic Diseases |
Precancerous Conditions Neoplasms Deferasirox Iron Chelating Agents Chelating Agents Molecular Mechanisms of Pharmacological Action Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 21, 2013