E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
patients with myelodisplastic syndrome |
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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 | LLT |
E.1.2 | Classification code | 10054350 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10054350 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10054350 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10054350 |
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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 |
The primary objective of the trial is to assess the activity of decitabine in the treatment of CMML. Activity will be evaluated as percentage of patients achieving complete or partial remission according to the International Working Group (IWG) response criteria in myelodysplasia13 |
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E.2.2 | Secondary objectives of the trial |
Secondary objectives of the trial are to assess the activity of decitabine in the treatment of CMML in terms of: Overall survival Time to leukemic transformation Response duration in terms of time to relapse after CR, PR Time to disease progression Response less than CR and PR evaluated according to IWG criteria for defining a haematology improvement (HI) Transfusion needs Safety of the treatment Quality of life Haematological and extra-haematological toxicity Relationship between biological marker (molecular and cytogenetic data) and efficacy of decitabine (response to therapy, time to progression, disease free survival and overall survival). |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
QUALITA DELLA VITA: Versione: Data: Titolo:QUESTIONARIO QOL in accordo con oliva et al. Obiettivi:
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E.3 | Principal inclusion criteria |
1. Patients aged 18 and older 2. CMML diagnosis according to WHO criteria 3. If WBC  12000/mm3: IPSS High or INT-2 If WBC < 12000/mm3: at least two of the following criteria Blast cells > 5% in the bone marrow Cytogenetic abnormality other than t(5;12) (q33; p13) Anemia (Hb < 10 g/dl) Thrombocytopenia (Plt < 100000/mm3) Splenomegaly (> 5 cm below costal margin) Extramedullary localization 4. Patients untreated or previously treated with Hydroxyurea or Imatinib or Etoposide given orally or non intensive chemotherapy or intensive chemotherapy given more than 3 months before inclusion 5. Performance Status ECOG 0, 1 or 2 6. Estimated life expectancy ≥6 months 7. Adequate hepatic function: Total bilirubin < 1.5 times upper limit of normal (except moderate unconjugate hyperbilirubinemia due to intra medullary hemolysis) AST and ALT < 3 times limit of normal 8. Adequate renal function: Serum creatinine < 1.5 times limit of normal Creatinine clearance > 30 ml/min 9. Informed consent 10. Negative pregnancy test or adequate contraception methods |
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E.4 | Principal exclusion criteria |
1. Myeloproliferative/myelodysplastic syndrome other than CMML 2. Acute blastic transformation of CMML with bone marrow blast cells > 20% 3. Patients eligible for allogenic bone marrow transplantation with identified donor 4. CMML with t(5;12) o PDGFBR rearrangement 5. Intensive chemotherapy given less than 3 months before 6. Previous treatment with hypomethylating agent 7. Age < 18 years old 8. Pregnancy or breastfeeding 9. Performance Status ECOG > 2 10. Estimate life expectancy < 6 months 11. HIV infection 12. Chronic active hepatitis secondary to HCV or HBV (HBSAg positive) 13. Serious concomitant systemic disorders, including active bacterial fungal or viral infection, that, in the opinion of the investigator, would compromise the safety of the patient and/or his/her ability to complete the study. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Response rate (CR+PR) evaluated according to 2006 IWG response criteria in myelodysplasia |
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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 | 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 | 9 |
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 | 6 |
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 |