Clinical trial NCT00673608: Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
Official title: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hemoglobinopathies
Myelodysplastic Syndromes
Other Inherited or Acquired Anaemia
MPD Syndrome
Diamond-Blackfan Anemia
Other Rare Anaemias
Transfusional Iron Overload
Keywords: Haemoglobinopathies,
Myelodysplastic Syndromes,
Transfusional iron overload,
cardiac iron load
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2008-05-07
Enrollment: 118
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility 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

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.

Primary outcomes:
measure: Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks.
timeFrame: 12 months
Secondary outcomes:
measure: Change in ventricular ejection fraction values, ventricular volumes and masses from baseline values after 53 weeks.
timeFrame: 12 months
measure: Change in cardiac T2* from baseline to 53 weeks in the MDS and other anaemias subgroup, compared to the thalassaemia subgroup.
timeFrame: 12 months
measure: Changes in serum ferritin from baseline values to 53 weeks.
timeFrame: 12 months
measure: Changes in Liver Iron Content (LIC) by MRI from baseline values to 53 weeks
timeFrame: 12 months
measure: The relationship between the dosing regimen of Exjade® and changes in cardiac T2* and LIC R2 MRI
timeFrame: 12 months
measure: Changes in markers of iron load levels between baseline and 53 weeks.
timeFrame: 12 months
measure: The safety and tolerability of deferasirox therapy from baseline to 53 weeks
timeFrame: 12 months
Other outcomes:
Locations:
city: Adelaide
country: Australia
facility: Novarts Investigative Site
city: Brisbane
country: Australia
facility: Novarts Investigative Site
city: Melbourne
country: Australia
facility: Novarts Investigative Site
city: Sydney
country: Australia
facility: Novarts Investigative Site
Study officials:
affiliation: Novartis Pharmeceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Ho PJ, Tay L, Teo J, Marlton P, Grigg A, St Pierre T, Brown G, Badcock CA, Traficante R, Gervasio OL, Bowden DK. Cardiac iron load and function in transfused patients treated with deferasirox (the MILE study). Eur J Haematol. 2017 Feb;98(2):97-105. doi: 10.1111/ejh.12793. Epub 2016 Sep 20.