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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

Phase 2
18 Years
80 Years
Open (Enrolling)
Myelodysplastic Syndrome

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Trial Information

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
supportive care

2. Group B: azacitidine 75mg/m^2 subcutaneously for 5 days every 28 days + best
supportive care

- 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subject, or informed consent is withdrawn.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.

Inclusion Criteria:

- Subjects must satisfy the following criteria in order to be enrolled in this clinical
trial: Patients who have been diagnosed with MDS by the FAB criteria and belong to
Low or INT-1 risk by the IPSS classification will be enrolled in this study. For the
purpose of analysis, chronic myelomonocytic leukemia (CMML) patients with less than
5% of myeloblasts are also classified by the IPSS risk classification. Secondary or
treatment-related MDS is allowed, but recurrent or persistent MDS after stem cell is
not applicable. The enrolled patients should have anemia (hemoglobin < 10.0g/dL),
transfusion dependence, thrombocytopenia (less than 100×10^9/L), or absolute
neutrophil count less than 1.80×10^9/L.

- 18 years of age or older

- Life expectancy of at least 12 months

- ECOG performance status 2 or less

- Serum creatinine less than 1.5 times the upper limit of normal (ULN) level of the
investigating institution

- Serum bilirubin less than 2.0 times the upper limit of normal (ULN) level of the
investigating institution

- AST, ALT, and alkaline phosphatase less than 3 times the upper limit of normal (ULN)
level of the investigation institution

- Patients who can have informed consent and signed the informed consent form

- Male patients who have a female partner of childbearing potential must agree to use
two types of effective contraceptive methods during the study and for 30 days
following the last dose.

- Females of childbearing potential (FCBP) must satisfy the following criteria: must
agree to use the contraceptive method (oral contraceptives, injectables, hormonal
implants; tubal ligation; intra uterine device; spermicidal contraceptives, the
sterilized partner) approved by the physician during azacitidine treatment and for 3
months following the last dose, and must have a negative result of serum pregnancy
test that was performed within 72 hours prior to starting study drug therapy.

Exclusion Criteria:

- Any coexisting major illness or organ failure

- HIV positive, or active hepatitis B or C infection

- Uncontrolled acute infection

- Uncontrolled hemorrhage

- Pregnant or lactating

- Known or suspected hypersensitivity to azacitidine

- Patients diagnosed with malignant hepatic carcinoma or malignant disease within the
past 12 months (except in situ carcinoma without complication, cervical or breast
intraepithelial neoplasia, or other local malignant carcinoma that is likely to be
treated by surgical removal or radiotherapy)

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Overall response rate by modified IWG 2006 response criteria

Outcome Description:

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.

Outcome Time Frame:

After 6 cycles of treatment up to 25-49 weeks

Safety Issue:


Principal Investigator

Yoo-Jin Kim, MD, PhD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Division of hematology, Department of Internal Medicine, Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital


Korea: Food and Drug Administration

Study ID:




Start Date:

March 2012

Completion Date:

August 2014

Related Keywords:

  • Myelodysplastic Syndrome
  • myelodysplastic syndrome
  • azacitidine
  • dosing schedule
  • Myelodysplastic Syndromes
  • Preleukemia