Myelodysplastic Syndrome
Conditions
Keywords
Myelodysplastic Syndrome, Decitabine, Dacogen, MGI Pharma
Brief summary
The purpose of this study is to determine the overall response rate in patients with myelodysplastic syndromes (MDS) given a daily dosing schedule of decitabine.
Interventions
DRUGDecitabine
20mg/m\^2, IV on days 1-5 of each 28 day cycle; until progression, death or unacceptable toxicity develops.
Sponsors
Eisai Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Must sign an Institutional Review Board (IRB) -approved informed consent form. 2. Must be 18 years of age or older. 3. Must have a diagnosis for MDS fitting any of the recognized French-American-British (FAB) classifications and International Prognostic Scoring System (IPSS) greater than or equal to 0.5 as determined by Complete Blood Count (CBC), bone marrow assessment, and cytogenetics within 28 days of receiving study drug. If FAB classification is Refractory anemia (RA) or Refractory anemia with ringed sideroblasts (RARS), then must be red cell transfusion dependent, defined as needing red cells more frequently than once every 4 weeks. 4. If receiving erythropoietin(Procrit), must have been on a stable dose for at least 8 weeks before first dose of study drug. 5. If receiving darbepoetin(Aranesp), must have been on a stable dose for at least 12 weeks before first dose of study drug.
Exclusion criteria
1. Must not have a diagnosis of Acute Myeloid Leukemia (AML) or other progressive malignant disease. 2. Must not have received any investigational agent within the 30 days preceding the first dose of study drug. 3. Must not have uncontrolled cardiac disease or uncontrolled congestive heart failure. 4. Must not have an active viral or bacterial infection.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Who Achieved Overall Response | 1 year | Overall Response = complete remission (disappearance of all target lesions) + partial remission (at least 30% decrease in the sum of the longest diameters of target lesions) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Best Response and Overall Improvement | 1 year | Overall Improvement = complete remission + marrow complete remission + partial remission + hematologic improvement (CR+mCR+PR+HI) |
Countries
Canada, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Decitabine 20 mg/m2 Intravenous Decitabine 20 mg/m2 intravenous IV on Days 1-5 of each 28 day cycle. | 99 |
| Total | 99 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 17 |
| Overall Study | Death | 13 |
| Overall Study | Medication Noncompliance | 1 |
| Overall Study | Not otherwise specified | 8 |
| Overall Study | Physician Decision | 19 |
| Overall Study | Progressive Disease | 22 |
| Overall Study | Withdrawal by Subject | 13 |
Baseline characteristics
| Characteristic | Decitabine 20 mg/m2 Intravenous |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 84 Participants |
| Age, Categorical Between 18 and 65 years | 15 Participants |
| Age Continuous | 70.9 years STANDARD_DEVIATION 8.78 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 4 Participants |
| Race (NIH/OMB) Black or African American | 6 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 3 Participants |
| Race (NIH/OMB) White | 86 Participants |
| Region of Enrollment United States | 99 participants |
| Sex: Female, Male Female | 28 Participants |
| Sex: Female, Male Male | 71 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 96 / 99 |
| serious Total, serious adverse events | 65 / 99 |
Outcome results
Primary
Number of Participants Who Achieved Overall Response
Overall Response = complete remission (disappearance of all target lesions) + partial remission (at least 30% decrease in the sum of the longest diameters of target lesions)
Time frame: 1 year
Population: Intent-to-treat (ITT)
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Decitabine 20 mg/m2 Intravenous | Number of Participants Who Achieved Overall Response | 33 participants |
95% CI: [24.2, 43.5]
Secondary
Best Response and Overall Improvement
Overall Improvement = complete remission + marrow complete remission + partial remission + hematologic improvement (CR+mCR+PR+HI)
Time frame: 1 year
Population: Intent-to-treat (ITT)
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Decitabine 20 mg/m2 Intravenous | Best Response and Overall Improvement | Overall Improvement | 51 Participants |
| Decitabine 20 mg/m2 Intravenous | Best Response and Overall Improvement | Complete Remission (CR) | 17 Participants |
| Decitabine 20 mg/m2 Intravenous | Best Response and Overall Improvement | Marrow Complete Remission (mCR) | 16 Participants |
| Decitabine 20 mg/m2 Intravenous | Best Response and Overall Improvement | Partial Remission (PR) | 0 Participants |
| Decitabine 20 mg/m2 Intravenous | Best Response and Overall Improvement | Hematologic Improvement (HI) | 18 Participants |
95% CI: [41.3, 61.7]