Status:

COMPLETED

PhII 5-Azacytidine Plus Valproic Acid and Eventually Atra in Intermediate II and High Risk MDS

Lead Sponsor:

Gruppo Italiano Malattie EMatologiche dell'Adulto

Conditions:

Myelodysplastic Syndromes

Eligibility:

All Genders

18+ years

Phase:

PHASE2

Brief Summary

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 ...

Detailed Description

Myelodisplastic Syndromes (MDS) are a heterogeneous group of diseases characterized by ineffective hematopoiesis (as a result of increased apoptosis of precursor cells), progressive peripheral cytopen...

Eligibility Criteria

Inclusion

  • Inclusion Criteria:
  • Have a diagnosis of refractory anemia with excess blasts (RAEB) or refractory anemia with excess blasts in transformation (RAEB-t) according to the French-American-British classification system for MDS with an International Prognostic Scoring System score of INT-2 or High or diagnosis of Myelodysplastic CMMoL per a modified FAB criteria and a relatively high risk of AML transformation;
  • Age ≥18 years;
  • life expectancy ≥3 months;
  • Be unlikely to proceed to bone marrow or stem cell transplantation therapy following remission;
  • Signed written informed consent according to IGH/EU/GCP and national local laws;
  • Eastern Cooperative Oncology Group Performance Status Grade of 0-2 (Appendix D);
  • Serum bilirubin levels ≤1.5 x the upper limit of the normal (ULN) range for the laboratory; higher levels are acceptable if these can be attributed to active hemolysis (as indicated by positive direct Coombs' testing, decreased haptoglobin level, elevated indirect bilirubin and/or lactate dehydrogenase), or ineffective erythropoiesis (as indicated by bone marrow findings);
  • Serum glutamic-oxaloacetic transaminase (aspartate aminotransferase) or serum glutamic-pyruvic transaminase (alanine aminotransferase) levels ≤2 x ULN;
  • Women of childbearing potential may participate, providing they meet the following conditions:
  • Must not start a pregnancy throughout the study and for 6 months following the date of the last dose of study medications;
  • Must have a negative serum pregnancy test obtained within 48 hours prior to Day 1.
  • Males with female partner of childbearing potential must avoid fathering throughout the study and for 6 months following the date of the last dose of study medication.
  • Exclusion criteria:
  • acute myeloid leukaemia (i.e. bone marrow blasts \>30%);
  • concurrent malignancy diagnosed in the past 12 months (with the exception of skin basalioma);
  • severe renal impairment (creatinine clearance \<30 ml/min);
  • pregnant or lactating, or are potentially fertile (both males and females) and have not agreed to avoid pregnancy during the trial period;
  • they have liver disease characterized by AST and ALT level \>2X ULN and total bilirubin \> 1.5X ULN (unless due to active hemolysis or ineffective erythropoiesis;
  • HIV infection;
  • active, uncontrolled HCV or HBV infections or liver cirrhosis;
  • clinically relevant neurological diseases;
  • psychiatric illness that would prevent granting of informed consent;
  • hypersensitivity (known or suspected) to Azacytidine or Mannitol
  • prior Treatments: Prior investigational drugs (within 30 days) Radiation therapy, chemotherapy, or cytotoxic therapy for non- MDS conditions within the previous 6 months Growth factors (EPO, G-CSF or GM-CSF) during the previous 21 days Androgenic hormones during the previous 14 days Prior transplantation or cytotoxic therapy, including azacitidine and chemotherapy, administered to treat MDS.

Exclusion

    Key Trial Info

    Start Date :

    May 1 2007

    Trial Type :

    INTERVENTIONAL

    Allocation :

    ACTUAL

    End Date :

    July 1 2010

    Estimated Enrollment :

    62 Patients enrolled

    Trial Details

    Trial ID

    NCT00439673

    Start Date

    May 1 2007

    End Date

    July 1 2010

    Last Update

    August 7 2018

    Active Locations (7)

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    Page 1 of 2 (7 locations)

    1

    USL 8 di Arezzo

    Arezzo, Italy

    2

    Azienda Ospedaliera S. G. Moscati

    Avellino, Italy

    3

    Università degli studi di Bari

    Bari, Italy

    4

    Istituto ematologia e oncologia medica L.A. Seragnoli

    Bologna, Italy