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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/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
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Anaemia
Blood 0 0 0 0
Other blood disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited 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
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

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
Sex
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
N/A
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
1/11/2007
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/09/2011
Sample size
Target
Accrual to date
Final
118
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/ct2/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

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00673608