None listed
Conditions
Sponsors
Novartis Pharmaceuticals
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion Criteria •Patients who had HSCT for beta-thalassemia major •HSCT was performed minimum 6 months and maximum 2 years ago •The washout period after the immunosuppressive therapy should be at least 3 months. •Signifacant IOL should be present including: A. Serum ferritin >1000 µg/L or B. cardiac MRI =65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: Exclusion Criteria •Patients who have any contraindication for treatment with deferasirox according to the prescribing information •Patients who depend on transfusion •Patients with clinical symptoms of cardiac dysfunction (shortness of breath at rest or exertion, orthopnea, exercise intolerance, lower extremity edema, arrhythmias) •Patients who are experiencing severe complication of HSCT (e.g. acute GVHD) •Significant proteinuria / Increase in serum creatinine
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: •Safety of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major [ Time Frame: 12 months ] • To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major in 12 months period;Secondary Objective: • Efficacy of deferasirox in the treatment of iron overload after HSCT in patients with beta-thalassemia major by 12th month. [ Time Frame: 12 Months ] • To evaluate change the serum ferritin level and change from baseline to 12th month • To evaluate change in the further parameters of iron overload (cardiac iron and liver iron concentration by MR examination) from baseline to 12th month • To determine the percentage of patients reaching serum ferritin levels lower than 500 µg/L at week 28 and at week 52.;Primary end point(s): Incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters;Timepoint(s) of evaluation of this end point: Incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters : 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - To evaluate change the serum ferritin level and change from baseline to 12th month - To evaluate change in the further parameters of iron overload (cardiac iron and liver iron concentration by MR examination) from baseline to 12th month - To determine the percentage of patients reaching serum ferritin levels lower than 500 µg/L at week 28 and at week 52.;Timepoint(s) of evaluation of this end point: - To evaluate change the serum ferritin level and change from baseline to 12th month - To evaluate change in the further parameters of iron overload (cardiac iron and liver iron concentration by MR examination) from baseline to 12th month - To determine the percentage of patients reaching serum ferritin levels lower than 500 µg/L at week 28 and at week 52. | — |
Countries
Turkey
Contacts
Public ContactClinical Trial Information Desk
Novartis Pharma AG
Outcome results
None listed