Safety and Efficacy of Azacitidine, and Thalidomide in Higher Risk MDS (Myelodysplastic Syndrome)
Status:
Unknown status
Trial end date:
2011-04-01
Target enrollment:
Participant gender:
Summary
Study Objectives The aim of the study is to evaluate the safety and efficacy of the
combination of 5-Aza-Cytidine + Thalidomide on the course of hrMDS patients.
Primary end point:
• To evaluate the overall response rate (CR+PR) of the combination of 5-Aza-Cytidine +
Thalidomide in hrMDS patients (INT-2 and High risk as defined by IPSS).
Secondary end points:
- To evaluate the safety of the combination of Thalidomide+5-Aza-Cytidine in high risk MDS
patients.
- Hematological improvement rate.
- Cytogenetic response.
- Progression free survival (PFS).
- Quality of life assessment (FACT: MDS and peripheral neuropathy QOL Questionnaires).
Study design:
This is a multicenter, phase II, single arm study designed to evaluate the safety and
efficacy of the combination of Thalidomide+5-Aza-Cytidine in high risk MDS patients (INT-2
and High risk defined by IPSS) who are older than 18 years of age. Potential study subjects
will sign an informed consent prior to undergoing any study related procedure. Number of
patients to be enrolled 50.
Treatment plan: 5-aza-cytidine (75 mg m2/d) will be injected subcutaneously in 5-day cycle
every 28 days, for a total of 12 cycles.
Thalidomide will be given at the dose of 50 mg/d, from day 1 until for 6 months together with
5-aza-cytidine .
Treatment period includes 5-aza-cytidine (75 mg m2/d) will be injected subcutaneously in
5-day cycle every 28 days. Total number of 12 cycles or until progression or toxicity. Cycle
delay of maximum 2 weeks in case of hematological toxicity grade 3-4 at investigator
discretion.
Duration of the follow up period is 6 months. Duration of study The duration of the treatment
period is approximately 12 months. This time is required to complete the treatment, and to
determine the safety profile and the response rate. The duration of the Follow period will be
approximately a half year. The occurrence of PD will determine the duration of
progression-free survival of each patient.