Adlut patients with low risk MDS (IPPS 0-1) MedDRA version: 9.1 Level: SOC Classification code 10005329 Term: Blood and lymphatic system disorders
Conditions
Interventions
Product Name: azacitidine
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: azacitidine
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10
Sponsors
AZIENDA OSPEDALIERA SPEDALI CIVILI DI BRESCIA
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients with MDS according to WHO classification with INT-2 or high IPSS risk. Life expectancy 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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).;Secondary Objective: 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;Primary end point(s): therapy respnse according to Cheson (Blood, 2006); SAE number and grade | — |
Countries
Italy
Outcome results
None listed