A Randomized, Phase II, Comparative Study With a Parallel Control for Evaluating the Efficacy and Safety of 5-day Azacitidine for Patients With Lower-risk Myelodysplastic Syndrome
- Using block randomization, subjects of Low or intermediate (INT)-1 patients will be
equally allocated to the following two types of regimens.
1. Group A: azacitidine 75mg/m^2 subcutaneously for 7 days every 28 days + best
2. Group B: azacitidine 75mg/m^2 subcutaneously for 5 days every 28 days + best
- The study drug, azacitidine, is provided free of charge by Celgene until disease
progression or relapse after response, or intolerable toxicity occurs in clinical study
subject, or informed consent is withdrawn.
- No crossover between arms is allowed.
- Dose escalation in this study is not allowed; on the contrary, dose reduction or dose
delay is possible based on adverse events and hematologic recovery.
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Overall response rate by modified IWG 2006 response criteria
Overall response rate is evaluated by assessing the percentage of patients with response (complete remission (CR), partial remission (PR), bone marrow CR, and hematologic improvement), response period, and transfusion requirement. Best response during at least 6 cycles of treatment will be assessed if there is no treatment failure or disease progression within 6 cycles of treatment. For patients who can keep the 4 week interval the Time Frame will be 25 weeks. The cycle interval can be extended up to 8 weeks which makes 49 weeks the maximum Time Frame.
After 6 cycles of treatment up to 25-49 weeks
Yoo-Jin Kim, MD, PhD
Division of hematology, Department of Internal Medicine, Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital
Korea: Food and Drug Administration