PhII 5-Azacytidine Plus Valproic Acid and Eventually Atra in Intermediate II and High Risk MDS
The primary objective of the trial is to assess the activity of the combined use of Valproic Acid (VPA)in combination with 5-Azacytidine (5-Aza C) in the treatment of MDS.
Activity will be evaluated as percentage of patients achieving complete or partial remission.
Drug: Valproic Acid
|Study Design:||Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Open Label, Phase II, Non Randomized, Clinical Trial of Chemotherapy Treatment With 5-Azacytidine Plus Valproic Acid and Eventually Atra for Patients Diagnosed With Intermediate II and High Risk Myelodysplastic Syndrome (MDS). EudraCT Number 2005-004811-31. GIMEMA Protocol MDS0205|
- The primary objective of the trial is to assess the efficacy of the combined use of Valproic Acid (VPA) in combination with 5-Azacytidine (5-Aza C) in the treatment of MDS.
- Efficacy will be evaluated as percentage of patients achieving complete or partial remission.
- Rate of patients achieving sustained haematological improvement
- Number of blood and platelet transfusions
- Number of infections requiring intravenous antibiotics/antimycotics
- Number of days of hospitalization
- Time to relapse after CR, PR or disease progression
- Time to transformation to AML
- Time to death from any cause
- To assess the prognostic and clinical significance of the molecular and cytogenetic features associated with response to therapy , time to progression, disease free survival and overall survival
- To perform biological studies including reactivation of tumor suppressor gene transcription through effect on DNA methylation, histone de-acetylase inhibitor (HDAC inhibitor) and to study the "in vitro" mechanisms of drug resistance and drug sensit
- In non responding patients this protocol would like to explore the safety, feasibility and efficacy of adding ATRA to the mentioned association
|Study Start Date:||May 2007|
|Study Completion Date:||July 2010|
|Primary Completion Date:||July 2010 (Final data collection date for primary outcome measure)|
|USL 8 di Arezzo|
|Azienda Ospedaliera S. G. Moscati|
|Università degli studi di Bari|
|Istituto ematologia e oncologia medica L.A. Seragnoli|
|Ospedale Reg. A di Summa|
|Ospedale A. Businco|
|Università degli studi di Roma La Cattolica|
|Principal Investigator:||Giuseppe LEONE, MD, PHD||Università degli studi di Roma La Cattolica|