HemoglobinopathiesPhase 4NCT00673608
Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload
A Phase 4 study testing deferasirox in people with Hemoglobinopathies and Myelodysplastic Syndromes, run by Novartis at 4 sites in Australia.
Trial summary
This study will evaluate the change in cardiac iron load over a 53 week period measured by MRI in 2 cohorts of patients Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemoglobinopathies, Myelodysplastic Syndromes, Other Inherited or Acquired Anaemia
- What is being tested?
- deferasirox
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 118 participants
- Where
- 4 sitesAustralia
- Study dates
- Nov 2007to Sep 2011
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
4 sites in 1 country.
Australia4 sites
Novarts Investigative Site
Adelaide
Novarts Investigative Site
Brisbane
Novarts Investigative Site
Melbourne
Novarts Investigative Site
Sydney
About this study
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)
This study will evaluate the change in cardiac iron load over a 53 week period measured by MRI in 2 cohorts of patients
- Conditions
- Hemoglobinopathies, Myelodysplastic Syndromes, Other Inherited or Acquired Anaemia, MPD Syndrome, Diamond-Blackfan Anemia, Other Rare Anaemias
- Being tested
- deferasirox
- Keywords
- Haemoglobinopathies,, Myelodysplastic Syndromes,, Transfusional iron overload,, cardiac iron load
- Medical subject headings
- Hemoglobinopathies, Myelodysplastic Syndromes, Fibromyalgia, Anemia, Diamond-Blackfan
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
deferasiroxDrug
Also called ICL670
Deferasirox up to 40mg/kg/day, PO, Dispersible tablets, taken once daily
| Study group | Role | What they receive |
|---|---|---|
| Deferasirox | Experimental | Not described Receives: Drug: deferasirox |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks.
Measured: 12 months
Secondary outcomes (7)
Change in ventricular ejection fraction values, ventricular volumes and masses from baseline values after 53 weeks.
Measured: 12 months
Change in cardiac T2* from baseline to 53 weeks in the MDS and other anaemias subgroup, compared to the thalassaemia subgroup.
Measured: 12 months
Changes in serum ferritin from baseline values to 53 weeks.
Measured: 12 months
Changes in Liver Iron Content (LIC) by MRI from baseline values to 53 weeks
Measured: 12 months
The relationship between the dosing regimen of Exjade® and changes in cardiac T2* and LIC R2 MRI
Measured: 12 months
Changes in markers of iron load levels between baseline and 53 weeks.
Measured: 12 months
The safety and tolerability of deferasirox therapy from baseline to 53 weeks
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 May 2008 (estimated)
- Study startNov 2007
- Primary completionSep 2011
- Study completionSep 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HemoglobinopathiesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 118 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmeceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00673608
- Sponsor's protocol ID
- CICL670AAU01
- Status last verified
- Feb 2017
- First submitted
- 5 May 2008
- Last updated
- 23 Feb 2017
- Results posted
- No
Publications
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.
PubMed 27537786
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT00673608
- Acronym / sponsor ID
- CICL670AAU01
- Conditions
- Hemoglobinopathies, Myelodysplastic Syndromes, Other Inherited or Acquired Anaemia, MPD Syndrome, Diamond-Blackfan Anemia, Other Rare Anaemias
- Keywords
- Haemoglobinopathies,, Myelodysplastic Syndromes,, Transfusional iron overload,, cardiac iron load
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 118 participants (actual)
- Lead sponsor
- NovartisIndustry (pharma, biotech, devices)
- Study start
- Nov 2007
- Primary completion
- Sep 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 7 May 2008 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study will evaluate the change in cardiac iron load over a 53 week period measured by MRI in 2 cohorts of patients
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Deferasirox | Experimental | Drug: deferasirox |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| deferasiroxAlso: ICL670Deferasirox up to 40mg/kg/day, PO, Dispersible tablets, taken once daily | Drug | Deferasirox |
Primary outcome
Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks.
Time frame: 12 months
7 secondary outcomes
Change in ventricular ejection fraction values, ventricular volumes and masses from baseline values after 53 weeks.
Time frame: 12 months
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
Changes in serum ferritin from baseline values to 53 weeks.
Time frame: 12 months
Changes in Liver Iron Content (LIC) by MRI from baseline values to 53 weeks
Time frame: 12 months
The relationship between the dosing regimen of Exjade® and changes in cardiac T2* and LIC R2 MRI
Time frame: 12 months
Changes in markers of iron load levels between baseline and 53 weeks.
Time frame: 12 months
The safety and tolerability of deferasirox therapy from baseline to 53 weeks
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2017.
- Derived from this study: PubMed 27537786Ho 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmeceuticals
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing deferasirox in people with Hemoglobinopathies and Myelodysplastic Syndromes, run by Novartis at 4 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Feb 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks. (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00673608, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Feb 2017.