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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00673608




Registration number
NCT00673608
Ethics application status
Date submitted
5/05/2008
Date registered
7/05/2008
Date last updated
23/02/2017

Titles & IDs
Public title
Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
Scientific 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)
Secondary ID [1] 0 0
CICL670AAU01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemoglobinopathies 0 0
Myelodysplastic Syndromes 0 0
Other Inherited or Acquired Anaemia 0 0
MPD Syndrome 0 0
Diamond-Blackfan Anemia 0 0
Other Rare Anaemias 0 0
Transfusional Iron Overload 0 0
Condition category
Condition code
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Diet and Nutrition 0 0 0 0
Other diet and nutrition disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - deferasirox

Experimental: Deferasirox -


Treatment: Drugs: deferasirox
Deferasirox up to 40mg/kg/day, PO, Dispersible tablets, taken once daily

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks.
Timepoint [1] 0 0
12 months
Secondary outcome [1] 0 0
Change in ventricular ejection fraction values, ventricular volumes and masses from baseline values after 53 weeks.
Timepoint [1] 0 0
12 months
Secondary outcome [2] 0 0
Change in cardiac T2* from baseline to 53 weeks in the MDS and other anaemias subgroup, compared to the thalassaemia subgroup.
Timepoint [2] 0 0
12 months
Secondary outcome [3] 0 0
Changes in serum ferritin from baseline values to 53 weeks.
Timepoint [3] 0 0
12 months
Secondary outcome [4] 0 0
Changes in Liver Iron Content (LIC) by MRI from baseline values to 53 weeks
Timepoint [4] 0 0
12 months
Secondary outcome [5] 0 0
The relationship between the dosing regimen of Exjade® and changes in cardiac T2* and LIC R2 MRI
Timepoint [5] 0 0
12 months
Secondary outcome [6] 0 0
Changes in markers of iron load levels between baseline and 53 weeks.
Timepoint [6] 0 0
12 months
Secondary outcome [7] 0 0
The safety and tolerability of deferasirox therapy from baseline to 53 weeks
Timepoint [7] 0 0
12 months

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 4
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Novarts Investigative Site - Adelaide
Recruitment hospital [2] 0 0
Novarts Investigative Site - Brisbane
Recruitment hospital [3] 0 0
Novarts Investigative Site - Melbourne
Recruitment hospital [4] 0 0
Novarts Investigative Site - Sydney
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Melbourne
Recruitment postcode(s) [4] 0 0
- Sydney

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis
Address
Country

Ethics approval
Ethics application status

Summary
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
Trial website
https://clinicaltrials.gov/show/NCT00673608
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmeceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications