MedDRA version: 19.0 Level: LLT Classification code 10054658 Term: Thalassemia System Organ Class: 100000004850 MedDRA version: 19.0 Level: LLT Classification code 10028534 Term: Myelodysplastic syndrome NOS System Organ Class: 100000004864
Conditions
Interventions
Trade Name: Exjade
Product Name: Deferasirox
Product Code: ICL670
Pharmaceutical Form: Dispersible tablet
Sponsors
Novartis Pharmaceuticals
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion Criteria: 1.Age = 2 years 2.Primary Diagnosis: Myelodysplastic Syndrome (presenting with low or intermediate-1 IPSS risk), thalassemia or rare anemias patients (anemia Diamond-Blackfan, Fanconi's anemia, Sideroblastic anemia, Red cell aplasia) 3.ECOG Performance Status = 2 4.Transfusion overload confirmed with ferritin level >1000 µg/l. 5.No severe concomitant uncontrolled disease (uncontrolled diabetes mellitus, heart failure, renal failure). 6.Serum creatine level > ULN 7.No proteinuria 8.Liver enzymes level =65 years) yes F.1.3.1 Number of subjects for this age range 24
Exclusion criteria
Exclusion criteria: Exclusion Criteria: 1.Age ULN or/and proteinuria 6.Liver enzymes level >5 ULN. 7.Pregnancy or lactation. Other protocol-defined inclusion/exclusion criteria may apply
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade (Time Frame: Baseline assessment is followed by monthly assessments for up to 1 year) ;Secondary Objective: •changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination [ Time Frame: Baseline assessment is followed by monthly assessments for up to 1 year. ] •changes in iron overload evidence on cardiac and liver magnetic resonance imaging (MRI) T2*, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade [ Time Frame: at baseline and 1 at year (at the end of study). ] •Number of participants with adverse events. Safety is evaluated through the continuous monitoring and recording of adverse events, as well as though routine laboratory assessments and physical examination. [ Time Frame: From the start of study up to 1 year ] ;Primary end point(s): changes in ferritin level, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade ;Timepoint(s) of evaluation of this end point: Baseline assessment is followed by monthly assessments for up to 1 year | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1.changes in clinical manifestations of iron overload by means of echocardiogram (ECHO), electrocardiogram (ECG), routine laboratory assessments and physical examination 2.changes in iron overload evidence on cardiac and liver magnetic resonance imaging (MRI) T2*, compared to baseline, in patients with transfusion-induced iron overload treated with Exjade 3.Number of participants with adverse events. Safety is evaluated through the continuous monitoring and recording of adverse events, as well as though routine laboratory assessments and physical examination.;Timepoint(s) of evaluation of this end point: 1. Baseline assessment is followed by monthly assessments for up to 1 year. 2. at baseline and 1 at year (at the end of study). 3. From the start of study up to 1 year | — |
Countries
Russian Federation
Contacts
Public ContactClinical Trial Informtaion Desk
Novartis Pharma AG
Outcome results
None listed