E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
In- or outpatients with myelodysplasia or beta thalassaemia major, who have chronic iron overload, as a consequence of frequent blood transfusion |
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MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Primary objective: to evaluate the clinical response to treatment with Exjade® from the baseline to weeks 36 by following serum-ferritin change in patients with transfusion dependent chronic anaemias ( Myelodysplastic Syndrome, Beta-thalassaemia major patients) with chronic iron overload. |
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E.2.2 | Secondary objectives of the trial |
Secondary objectives: to assess drug usage compliance, evaluate patient’s compliance by monthly counting returned study medication and retrospective interviews with patient/parent/caregiver, and assess safety data during 36 week long treatment with Exjade® in patients with transfusion dependent chronic anaemias ( Myelodysplastic Syndrome, Beta-thalassaemia major patients) with chronic iron overload.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•In- or outpatients with myelodysplasia / with risk of low or intermedier-1 according to the International Prognostic Scoring System (IPSS) confirmed by bone marrow evaluation within 3 month/ or beta thalassaemia major patients, who have chronic iron overload, as a consequence of frequent blood transfusion •Serum ferritin> 1800 µg/L • Age: 18-80 years •Chronic iron overload caused by at least 30 units and maximum 100 units of blood of packed red blood cells •Deferoxamin (Desferal®) therapy is contraindicated or inadequate or unable to use in the recommended dose due to intolerability or other reason •ECOG performance status score between 0-2 |
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E.4 | Principal exclusion criteria |
•beta thalassaemia minor, •haemosiderosis caused by other than chronic transfusional iron overload, •patients with impaired renal function (Creatinin clearance< 60 ml/ min), •pregnancy, •lactation, •patient of childbearing potencial unwilling to use contraceptive precautions •known hypersensitivity to Exjade or any ingredients, •impaired hepatic function (SGOT,SGPT 5x above UNL). •Patients severely ill due to underlying disease progression or other severe concomitant disease. •Patients participated in other clinical trial 30 days prior enrollment. •Patients with poor prognosis of karyotype according to the IPSS •ECOG performance status score above 2 •patients with malabsorption caused by inflammatory bowel disease, gastrectomy, pancreatitis or other medical condition •History of nephrotic syndrome •Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 (mg/mg) in second-voiding urine samples taken at either visits 1 or 2. •History of non-compliance to medical regimens •Patients with positive test to HIV • Life expectancy of < 1year |
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E.5 End points |
E.5.1 | Primary end point(s) |
Assessment of the efficacy of the Exjade treatment in patient with transfusional hemosiderosis in MDS patients and beta thalassaemia major patients based on changes in serum ferritin level during the study from the baseline up to the 36. weeks of the treatment |
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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 | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 5 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 8 |
E.8.9.1 | In the Member State concerned months | 12 |
E.8.9.1 | In the Member State concerned days | 31 |