Low and intermediate I risk (IPSS score) Myelodysplastic syndrome and transfusion-induced hemosiderosis. MedDRA version: 9.1 Level: LLT Classification code 10028533 Term: Myelodysplastic syndrome
Conditions
Interventions
Product Name: Deferasirox
Product Code: ICL 670
Pharmaceutical Form: Tablet
INN or Proposed INN: Deferasirox
CAS Number: 201530-41-8
Current Sponsor code: ICL 670
Concentration unit: mg milligram(s)
Sponsors
G.I.M.E.M.A. GRUPPO ITALIANO MALATTIE EMATOLOGICHE DELL'ADULTO
Eligibility
Sex/Gender
All
Inclusion 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 ?g/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 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: ?Diagnosis different from MDS (i.e. mielofibrosis) ?Severe renal impairment (creatinine clearance 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the trial is to assess the safety and tolerability of Deferasirox in patients with Myelodysplastic syndrome and transfusion-induced hemosiderosis.;Secondary Objective: ?To evaluate the efficacy of Deferasirox as chelation therapy in patients with Myelodysplastic syndrome, in terms of serum ferritin reduction compared to baseline values ?To evaluate the impact of chelation therapy with Deferasirox on the requirements of blood transfusions in a group of patients who do not receive growth factors or chemotherapy ?Quality of life assessment ?Chelation therapy compliance;Primary end point(s): Toxicity grade incidence >2 according to the NCI-CTCAE 3.0 | — |
Countries
Italy
Outcome results
None listed