Phase 2
Completed N=151
Alternative Dosing Regimens of Subcutaneous Azacitidine for Myelodysplastic Syndromes
Source: ClinicalTrials.gov NCT00102687 ↗Enrolled (actual)
151
Serious AEs
45.6%
Results posted
May 2010
Primary outcomePrimary: Number of Participants In Best Hematological Response Categories as Determined by the Investigator Using International Working Group 2000 (IWG 2000) Criteria For Myelodysplastic Syndromes (MDS) During the Initial Study Period. — 4; 3; 4; 2 participants
Summary
The purpose of this study is to determine if azacitidine, combined with Best Supportive Care (BSC), is effective in treating myelodysplastic syndromes (MDS) when given according to a different doses and dosing schedules.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants In Best Hematological Response Categories as Determined by the Investigator Using International Working Group 2000 (IWG 2000) Criteria For Myelodysplastic Syndromes (MDS) During the Initial Study Period. |
4; 3; 4; 2; 2; 2 | — |
| PRIMARY Number of Participants With Best Hematological Improvement Derived Using International Working Group 2000 (IWG 2000) Criteria for MDS During the Initial Study Period. |
29; 20; 26; 18; 15; 18 | — |
| PRIMARY Number of Participants With Overall Best Hematologic Response and Hematologic Improvement Based on IWG 2000 Criteria For MDS During the Initial Study Period |
30; 20; 27 | — |
| PRIMARY Number of Participants Who Improved or Maintained The Hematologic Response From the Initial Study Period (Based on IWG 2000 Criteria For MDS) During the Maintenance Period |
7; 10; 12; 6; 5; 10 | — |
| SECONDARY Baseline Hemoglobin Values |
94.0; 98.0; 95.5 | — |
| SECONDARY Change From Baseline in Hemoglobin at End of Initial Study Period (6 Months) |
2.3; 2.9; 5.3 | — |
| SECONDARY Change From Baseline in Hemoglobin at the End of the Maintenance Study Period |
5.9; 7.3; 14.5 | — |
| SECONDARY Baseline Platelet Values |
110.0; 90.0; 86.0 | — |
| SECONDARY Change From Baseline in Platelets at the End of Initial Study Period (6 Months) |
4.8; 5.7; 12.6 | — |
| SECONDARY Change From Baseline in Platelets at the End of the Maintenance Study Period (Month 24) |
19.8; 7.7; 17.3 | — |
| SECONDARY Baseline Absolute Neutrophil Count (ANC) Values |
1.6; 1.9; 2.3 | — |
| SECONDARY Change From Baseline in Absolute Neutrophil Count (ANC) at the End of Initial Study Period (6 Months) |
-0.3; -0.3; -0.4 | — |
| SECONDARY Change From Baseline in Absolute Neutrophil Count (ANC) at the End of the Maintenance Study Period (Month 24) |
-0.1; -0.2; -0.4 | — |
| SECONDARY Red Blood Cell (RBC) Transfusion Status at Baseline and End of Initial Study Period (6 Months) |
16; 14; 13; 9; 10; 9 | — |
| SECONDARY Platelet Transfusion Status at Baseline and End of Initial Study Period (6 Months) |
3; 2; 0; 1; 0; 1 | — |
| SECONDARY Red Blood Cell (RBC) Transfusion Status at Baseline and End of Maintenance Study Period (24 Months) |
5; 12; 10; 1; 0; 2 | — |
| SECONDARY Platelet Transfusion Status at Baseline and End of Maintenance Study Period (24 Months) |
1; 1; 2; 0; 0; 0 | — |
| SECONDARY Change From Baseline in the Number of Infections Requiring Treatment With IV Antibiotics Per Treatment Cycle (28 Days) for the Initial Study Period |
0.00; 0.00; 0.00 | — |
| SECONDARY Change From Baseline in the Number of Infections Requiring Treatment With IV Antibiotics Per Treatment Cycle (28 Days) for the Maintenance Study Period |
0.00; 0.00; 0.00 | — |
Eligibility Criteria
Inclusion Criteria
- Diagnosis of refractory anemia, refractory anemia with ringed sideroblasts and at least one of the following: a)Anemia with hemoglobin 7 months.
- Unlikely to proceed to bone marrow or stem cell transplantation therapy following remission.
- Have serum bilirubin levels less than or equal to 1.5 times the upper limit of the normal (ULN) range for the laboratory.
- Have serum glutamic-oxaloacetic transaminase (aspartate aminotransferase) or serum glutamic-pyruvic transaminase (alanine aminotransferase) levels less than or equal to 2 x ULN.
- Have serum creatinine levels less than or equal to 1.5 x ULN.
Exclusion Criteria
- Secondary MDS.
- Prior treatment with azacitidine.
- Any prior history of Acute Myeloid Leukemia (AML).
- Malignant or metastatic disease within the previous 12 months.
- Uncorrected red cell folate deficiency or vitamin B12 deficiency.
- Hepatic tumors.
- Radiation, chemotherapy, or cytotoxic therapy for non-MDS conditions in the previous 12 months.
- Known or suspected hypersensitivity to azacitidine or mannitol.
- Prior transplantation or cytotoxic therapy to treat MDS. Prior use of Revlimid and Thalomid allowed after 30 day washout.
- Serious medical illness likely to limit survival to less than or equal to 7 months.
- Treatment with androgenic hormones during the previous 14 days
- Active viral infection with known human immunodeficiency virus or vial hepatitis Type B or C.
- Treatment with other investigational drugs with the previous 30 days.
Data sourced from ClinicalTrials.gov (NCT00102687). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.