Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload

Study Overview
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Brief Summary
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This study will evaluate the change in cardiac iron load over a 53 week period measured by MRI in 2 cohorts of patients

Official Title
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A Study of Magnetic Resonance Imaging Assessment of Cardiac and Liver Iron Load in Patients With Haemoglobinopathies, Myelodysplastic Syndromes (MDS) or Other Anaemias Treated With Exjade® (Deferasirox) (The MILE Study)

Conditions
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Hemoglobinopathies, Myelodysplastic Syndromes, Other Inherited or Acquired Anaemia, MPD Syndrome, Diamond-Blackfan Anemia, Other Rare Anaemias, Transfusional Iron Overload

Intervention / Treatment
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* Drug: deferasirox


Participation Criteria
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Eligibility Criteria
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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 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Deferasirox<br> | Drug: deferasirox<br>* Deferasirox up to 40mg/kg/day, PO, Dispersible tablets, taken once daily<br>* Other names: ICL670;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks. |  | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Haemoglobinopathies,, Myelodysplastic Syndromes,, Transfusional iron overload,, cardiac iron load