NCT00673608

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 Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
118

participants targeted

Target at P50-P75 for phase_4

Timeline
Completed

Started Nov 2007

Longer than P75 for phase_4

Geographic Reach
1 country

4 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2007

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

May 5, 2008

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 7, 2008

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2011

Completed
Last Updated

February 23, 2017

Status Verified

February 1, 2017

Enrollment Period

3.8 years

First QC Date

May 5, 2008

Last Update Submit

February 20, 2017

Conditions

HemoglobinopathiesMyelodysplastic SyndromesOther Inherited or Acquired AnaemiaMPD SyndromeDiamond-Blackfan AnemiaOther Rare AnaemiasTransfusional Iron Overload

Keywords

Haemoglobinopathies,Myelodysplastic Syndromes,Transfusional iron overload,cardiac iron load

Outcome Measures

Primary Outcomes (1)

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

    12 months

Secondary Outcomes (7)

  • Change in ventricular ejection fraction values, ventricular volumes and masses from baseline values after 53 weeks.

    12 months

  • Change in cardiac T2* from baseline to 53 weeks in the MDS and other anaemias subgroup, compared to the thalassaemia subgroup.

    12 months

  • Changes in serum ferritin from baseline values to 53 weeks.

    12 months

  • Changes in Liver Iron Content (LIC) by MRI from baseline values to 53 weeks

    12 months

  • The relationship between the dosing regimen of Exjade® and changes in cardiac T2* and LIC R2 MRI

    12 months

  • +2 more secondary outcomes

Study Arms (1)

Deferasirox

EXPERIMENTAL
Drug: deferasirox

Interventions

deferasiroxDRUG

Deferasirox up to 40mg/kg/day, PO, Dispersible tablets, taken once daily

Also known as: ICL670
Deferasirox

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Novarts Investigative Site

Adelaide, Australia

Location

Novarts Investigative Site

Brisbane, Australia

Location

Novarts Investigative Site

Melbourne, Australia

Location

Novarts Investigative Site

Sydney, Australia

Location

Related Publications (1)

  • 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.

    PMID: 27537786DERIVED

MeSH Terms

Conditions

HemoglobinopathiesMyelodysplastic SyndromesFibromyalgiaAnemia, Diamond-Blackfan

Interventions

Deferasirox

Condition Hierarchy (Ancestors)

Hematologic DiseasesHemic and Lymphatic DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesBone Marrow DiseasesMuscular DiseasesMusculoskeletal DiseasesRheumatic DiseasesNeuromuscular DiseasesNervous System DiseasesAnemia, Hypoplastic, CongenitalAnemia, AplasticAnemiaRed-Cell Aplasia, PureCongenital Bone Marrow Failure SyndromesBone Marrow Failure Disorders

Intervention Hierarchy (Ancestors)

BenzoatesAcids, CarbocyclicCarboxylic AcidsOrganic ChemicalsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsTriazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic Compounds

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmeceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 5, 2008

First Posted

May 7, 2008

Study Start

November 1, 2007

Primary Completion

September 1, 2011

Study Completion

September 1, 2011

Last Updated

February 23, 2017

Record last verified: 2017-02

Locations

AU(4)