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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

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

NCT ID

NCT00673608

First Posted

May 5, 2008

Last Updated

February 20, 2017

Sponsor

Novartis

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

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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