Safety and Efficacy of Azacitidine, and Thalidomide in Higher Risk MDS (Myelodysplastic Syndrome) (IMDS001)
Recruitment status was Not yet recruiting
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).
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.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase 2, Single Arm Study to Determine the Safety and Efficacy of Azacitidine, and Thalidomide in Higher Risk Myelodysplastic Syndrome|
- overall response rate measured by complete blood counts and bone marrow examinations [ Time Frame: at study entry. after 6 months and after one year ] [ Designated as safety issue: No ]
- overall response rate [ Time Frame: 6 and 12 months ] [ Designated as safety issue: No ]
- *safety *hematological improvement *cytogenetic response *progression free survival * Quality of life assessment (FACT: MDS and peripheral neuropathy QOL Questionnaires). [ Time Frame: at entry , 6 and 12 months ] [ Designated as safety issue: Yes ]
|Study Start Date:||September 2008|
|Estimated Study Completion Date:||April 2011|
|Estimated Primary Completion Date:||September 2010 (Final data collection date for primary outcome measure)|
Drug: 5-aza-cytidine and Thalidomide
|Contact: Moshe Mittelman, MD||972-3-6973366||moshemt@TASMC.HEALTH.GOV.IL|
|Contact: Elizabeth Naparstek, MDfirstname.lastname@example.org|
|Hematolpgical department Tel Aviv Medical Center||Not yet recruiting|
|Contact: Moshe Mittelman, MD 972-3-6973366 email@example.com|
|Contact 972-52-4266435 firstname.lastname@example.org|
|Principal Investigator: Moshe Mittelman, MD|