E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Low or Intermediate-1 IPSS risk MDS with del(5q)as a single or combined cytogenetic abnormality and with anemia |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10005329 |
E.1.2 | Term | Blood and lymphatic system disorders |
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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 |
To evaluate 1. changes in quality of life in MDS patients undergoing treatment with lenalidomide 2. safety of Lenalidomide in patients with MDS. |
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E.2.2 | Secondary objectives of the trial |
To evaluate in MDS patients 1. the effect of lenalidomide on cardiac remodelling and its correlation with erythroid response in anemic patients with 5q-, low or intermediate-1 risk MDS 2. the efficacy of Lenalidomide in terms of response (response / no response) for the treatment of anemia in patients with MDS. 3. variations in hemoglobin levels, in the number of monthly transfusions, in platelet and absolute neutrophil counts (ANC) during treatment. 4. cytogenetic and molecular responses. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Primary MDS 2. Age ≥ 18 years 3. Hb < 10 g/dL 4. del(5q) as a single or associated (del(5q) +1 or more) bone marrow cytogenetic abnormality 5. Low or Int-1 IPSS risk 6. No evidence of other causes of anemia 7. Written informed consent. |
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E.4 | Principal exclusion criteria |
1. Secondary MDS or evolving from prior hematological malignancies 2. Intermediate-2 or High IPSS risk MDS 3. Absolute neutrophil counts (ANC) < 500 cells/mm3 4. Platelet counts < 50,000/mm3 5. Contemporary epoetin treatment (epoetin treatment must be interrupted 4 months prior to screening) 6. Prior chemotherapy 7. Creatinine clearance < 60 ml/min 8. Serum SGOT/AST or SGPT/ALT > 3.0 x upper limit of normal (ULN) and serum direct bilirubin > 2.0 mg/dL 9. Cardiac arrythmias. 10. Thrombophilia or prior thomboembolic events in the absence of proper prophylactic treatment. 11. Pregnancy or inadequate birth control in females of childbearing potential. |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Changes in quality of life (QoL-E) scores in the patients undergoing treatment. 2. The frequency of adverse events. |
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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 | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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) | 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 | 14 |
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 | Yes |
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
|
L'ultima visita registrata dell'ultimo soggetto arruolato se termina lo studio a 12 mesi; altrimenti, si conclude con il completamento dello studio a 12 mesi del paziente in ordine precedente. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |