E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
patients with IPSS low- and intermediate-1 risk myelodysplastic syndromes |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10028533 |
E.1.2 | Term | Myelodysplastic syndrome |
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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 |
Assess the level of haematological response according to IWG 2000 and IWG 2006 criteria with a pattern of administration of high doses epoetin beta (NeoRecormon ®) for 12 and 24 weeks of treatment;
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E.2.2 | Secondary objectives of the trial |
•To evaluate the predictive value of the rate of reticulocytes at Day 8 on the erythroid response; •Assess time to response •Assess tolerance •Assess the impact on quality of life using the FACT-An and EQ-5D measurement scales; •Assess functional capacity: Cardiovascular performance on effort by the 6-minute walk test Performing the « Short Physical Performance Battery » tests
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•Written informed consent; •Must be 18 years of age or older at the time of screening ; •Myelodysplastic Syndrome of the following subtypes : RA, RAS, RCMD, RCMD-RS, RAEB 1, with IPSS score low or intermediate 1 (IPSS score < 1 ) and with anemia defined by Hb < 10 g/dl (with RBC transfusion requirement or not); •For women of childbearing potential, need for effective contraception throughout the study period.
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E.4 | Principal exclusion criteria |
•Intensive Chemotherapy within 3 months before inclusion; •Myelodysplastic Syndrome with IPSS score >1 ; •Treatment with rHu-Epo or rHu-GCSF within 2 months before inclusion •EGOG > 3 •Other causes of anemia (eg. , Iron deficiency, vitamin B12 or folic acid, hypothyroidism before correction) •Uncontrolled arterial hypertension •Life expectancy less than 6 months •CMML •Pregnant or breast feeding female subjects •Patients with creatinine clearance less than 30ml/min
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E.5 End points |
E.5.1 | Primary end point(s) |
The evaluation of response according to the IWG 2000 and IWG 2006 criteria will be held at 12 and 24 weeks of 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 | No |
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) | 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 | 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 |
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 | 26 |
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 | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |