E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated | |
E.1.1.1 | Medical condition in easily understood language |
• Haemoglobinopathies
•Myelodysplastic Syndromes
•Other Inherited or Acquired Anaemia (e.g. MPD, Diamond-blackfan Anaemia and Other Rare Anaemias),
•Transfusional Iron Overload
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028534 |
E.1.2 | Term | Myelodysplastic syndrome NOS |
E.1.2 | System Organ Class | 100000004864 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks. [ Time Frame: 12 months ] |
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E.2.2 | Secondary objectives of the trial |
•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 ]
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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
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E.4 | Principal 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.
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E.5 End points |
E.5.1 | Primary end point(s) |
Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Change in cardiac iron load and cardiac ejection fraction by MRI recorded at baseline and after 53 weeks:Time Frame: 12 months |
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E.5.2 | Secondary end point(s) |
•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
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
•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
•Changes in markers of iron load levels between baseline and 53 weeks: 12 months
•The safety and tolerability of deferasirox therapy from baseline to 53 weeks : 12 months
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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September 2011 (final data collection date for primary outcome measure) |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 10 |
E.8.9.2 | In all countries concerned by the trial days | 0 |