MedDRA version: 19.0 Level: LLT Classification code 10028534 Term: Myelodysplastic syndrome NOS System Organ Class: 100000004864
Conditions
Sponsors
Novartis
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: •Male or female WITH haemoglobinopathy, Myelodysplastic Syndromes or other inherited or acquired anaemia (e.g. MPD, Diamond-blackfan anaemia and other rare anaemias) patients = 18 years and weighing >40kg. •Lifetime minimum of > 20 units of packed red blood cell transfusions •Normal or minimally abnormal cardiac function Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 41 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 7
Exclusion criteria
Exclusion criteria: •Contraindication to MRI scans •High risk myelodysplastic syndromes patients and patients with other haematological and non-haematological malignancies who are not expected to benefit from chelation therapy due to the rapid progression of their disease •Patients with uncontrolled high blood pressure •An organ transplant less than 3 months previously Other protocol-defined inclusion/exclusion criteria may apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks. [ Time Frame: 12 months ];Secondary Objective: •Change in ventricular ejection fraction values, ventricular volumes and masses from baseline values after 53 weeks. [ Time Frame: 12 months ] •Change in cardiac T2* from baseline to 53 weeks in the MDS and other anaemias subgroup, compared to the thalassaemia subgroup. [ Time Frame: 12 months ] •Changes in serum ferritin from baseline values to 53 weeks. [ Time Frame: 12 months ] •Changes in Liver Iron Content (LIC) by MRI from baseline values to 53 weeks [ Time Frame: 12 months ] •The relationship between the dosing regimen of Exjade® and changes in cardiac T2* and LIC R2 MRI [ Time Frame: 12 months ] •Changes in markers of iron load levels between baseline and 53 weeks. [ Time Frame: 12 months ] •The safety and tolerability of deferasirox therapy from baseline to 53 weeks [ Time Frame: 12 months ] ;Primary end point(s): Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks. ;Timepoint(s) of evaluation of this end point: Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks:Time Frame: 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: •Change in ventricular ejection fraction values, ventricular volumes and masses from baseline values after 53 weeks: 12 months •Change in cardiac T2* from baseline to 53 weeks in the MDS and other anaemias subgroup, compared to the thalassaemia subgroup: 12 months •Changes in serum ferritin from baseline values to 53 weeks: 12 months •Changes in Liver Iron Content (LIC) by MRI from baseline values to 53 weeks : 12 months •The relationship between the dosing regimen of Exjade® and changes in cardiac T2* and LIC R2 MRI : 12 months •Changes in markers of iron load levels between baseline and 53 weeks: 12 months •The safety and tolerability of deferasirox therapy from baseline to 53 weeks : 12 months ;Secondary end point(s): •Change in ventricular ejection fraction values, ventricular volumes and masses from baseline values after 53 weeks. •Change in cardiac T2* from baseline to 53 weeks in the MDS and other anaemias subgroup, compared to the thalassaemia subgroup. [ •Changes in serum ferritin from baseline values to 53 weeks. •Changes in Liver Iron Content (LIC) by MRI from baseline values to 53 weeks •The relationship between the dosing regimen of Exjade® and changes in cardiac T2* and LIC R2 MRI •Changes in markers of iron load levels between baseline and 53 weeks. •The safety and tolerability of deferasirox therapy from baseline to 53 weeks | — |
Countries
Australia
Contacts
Public ContactClinical Trial Information Desk
Novartis Pharma AG
Outcome results
None listed