E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Adlut patients with low risk MDS (IPPS 0-1) |
|
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 |
|
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 |
To evaluate the efficacy (hematologic response), and toxicity of five days monthly 5-Aza treatment schedule in patients with low-risk MDS (IPSS 0-1). |
|
E.2.2 | Secondary objectives of the trial |
To evaluate the QoL by the FACT-An questionnaire To evaluate time to progression of MDS To evaluate the gene expression profile of MDS patients sensitive or resistant to 5-Aza To evaluate oncostatin M, interleukin-6 and interleukin-11 levels in the responders and non responders |
|
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Patients with low-risk (IPSS 0-INT1) MDS according to WHO classification, presenting one or more of the followings: - Symptomatic anemia requiring RBC transfusion supportive therapy previously unresponsive to EPO or not expected to respond to EPO, - Thrombocytopenia requiring platelet transfusion with or without muco-cutaneous haemorragic syndrome. Persistent (>3 months) absolute neutrophil count less then 1,5 x 109/L , with or without infections, requiring or not myeloid growth factor therapy. ≥18 years old. Life expectancy≥3months. ECOG performance Status Grade 0-2. Serum bilirubin levels ≤1.5 upper limit of the normal (ULN) Serum GOT and GPT levels ≤ 2x UNL. Creatinine levels ≤1.5x UNL. Negative serum β-human chorionic gonadotropin (β-HCG) pregnancy test 24 hours prior to beginning of therapy with 5-AZA, for fertile women. Written informed consent. |
|
E.4 | Principal exclusion criteria |
Patients with MDS according to WHO classification with INT-2 or high IPSS risk. Life expectancy < 3months; ECOG performance Status Grade > 2; Serum bilirubin levels >1.5 upper limit of the normal (ULN); Serum GOT and GPT levels > 2 x UNL; Creatinine levels >1.5 x UNL; Pregnancy or breast feeding Insulin-dependent diabetes and uncontrolled non insulin-dependent diabetes; Severe cardiac or pulmonary disease incompatible with the conduction of the protocol; Patient with a clear indication to receive long-term anticoagulant therapy; Other active hematologic or solid tumors; Severe CNS disease; Malignant hepatic tumors Hypersensitivity to mannitol or azacitidine No written informed consent. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
therapy respnse according to Cheson (Blood, 2006); SAE number and grade |
|
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 | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Information not present in EudraCT |
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.8.5 | 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 | Information not present in EudraCT |
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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
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 | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |