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

Primary Purpose

Hemoglobinopathies, Myelodysplastic Syndromes, Other Inherited or Acquired Anaemia

Status
Completed
Phase
Phase 4
Locations
Australia
Study Type
Interventional
Intervention
deferasirox
Sponsored by
Novartis
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemoglobinopathies focused on measuring Haemoglobinopathies,, Myelodysplastic Syndromes,, Transfusional iron overload,, cardiac iron load

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Novarts Investigative Site
  • Novarts Investigative Site
  • Novarts Investigative Site
  • Novarts Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Deferasirox

Arm Description

Outcomes

Primary Outcome Measures

Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks.

Secondary Outcome Measures

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

Full Information

First Posted
May 5, 2008
Last Updated
February 20, 2017
Sponsor
Novartis
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1. Study Identification

Unique Protocol Identification Number
NCT00673608
Brief Title
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

2. Study Status

Record Verification Date
February 2017
Overall Recruitment Status
Completed
Study Start Date
November 2007 (undefined)
Primary Completion Date
September 2011 (Actual)
Study Completion Date
September 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis

4. Oversight

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
118 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Deferasirox
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
deferasirox
Other Intervention Name(s)
ICL670
Intervention Description
Deferasirox up to 40mg/kg/day, PO, Dispersible tablets, taken once daily
Primary Outcome Measure Information:
Title
Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks.
Time Frame
12 months
Secondary Outcome Measure Information:
Title
Change in ventricular ejection fraction values, ventricular volumes and masses from baseline values after 53 weeks.
Time Frame
12 months
Title
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
Title
Changes in serum ferritin from baseline values to 53 weeks.
Time Frame
12 months
Title
Changes in Liver Iron Content (LIC) by MRI from baseline values to 53 weeks
Time Frame
12 months
Title
The relationship between the dosing regimen of Exjade® and changes in cardiac T2* and LIC R2 MRI
Time Frame
12 months
Title
Changes in markers of iron load levels between baseline and 53 weeks.
Time Frame
12 months
Title
The safety and tolerability of deferasirox therapy from baseline to 53 weeks
Time Frame
12 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmeceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novarts Investigative Site
City
Adelaide
Country
Australia
Facility Name
Novarts Investigative Site
City
Brisbane
Country
Australia
Facility Name
Novarts Investigative Site
City
Melbourne
Country
Australia
Facility Name
Novarts Investigative Site
City
Sydney
Country
Australia

12. IPD Sharing Statement

Citations:
PubMed Identifier
27537786
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.
Results Reference
derived

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

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