Phase 3
N=358
A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care
Myelodysplastic Syndromes
Bottom Line
View on ClinicalTrials.gov: NCT00071799 ↗Enrolled (actual)
358
Serious AEs
66.5%
Results posted
Jul 2010
Primary outcome: Primary: Kaplan-Meier Estimates for Median Time to Death From Any Cause — 24.46; 15.02 months — p=0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Azacitidine (Drug); Physician Choice (Other)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Celgene
- Primary completion
- Jul 2007
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Kaplan-Meier Estimates for Median Time to Death From Any Cause |
24.46; 15.02 | 0.0001 sig |
| PRIMARY Summary of Subgroup Analyses for Kaplan-Meier Estimates for Time to Death From Any Cause |
11.31; 7.87; 24.46; 13.87; 8.92; 6.20 | 0.3973 |
| PRIMARY Number of Participants Who Died |
82; 113 | — |
| SECONDARY Kaplan-Meier Estimate for Median Time to Transformation to Acute Myeloid Leukemia (AML) or Death From Any Cause, Whichever Occurred First |
13.02; 7.61 | 0.0025 sig |
| SECONDARY Kaplan-Meier Estimates for Median Time to Transformation to Acute Myeloid Leukemia (AML) |
20.66; 15.44 | 0.2555 |
| SECONDARY Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Dependent at Baseline |
50; 13; 61; 101 | <0.0001 sig |
| SECONDARY Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Independent at Baseline |
58; 37; 10; 28 | 0.0005 sig |
| SECONDARY Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Dependent at Baseline |
16; 11; 22; 16 | 1.000 |
| SECONDARY Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Independent at Baseline |
126; 102; 15; 50 | <0.0001 sig |
| SECONDARY Number of Participants Considered Hematologic Responders by Investigator Determinations Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS) |
51; 21; 30; 14; 21; 7 | 0.0001 sig |
| SECONDARY Number of Participants Showing Hematologic Improvement Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS) Assessed by Independent Review Committee |
87; 51; 62; 17; 2; 1 | <0.0001 sig |
| SECONDARY Time to Disease Progression, Relapse After Complete or Partial Remission, or Death From Any Cause |
14.13; 8.82 | 0.0466 sig |
| SECONDARY Duration of Any Hematologic Improvement |
13.57; 5.18 | 0.0002 sig |
| SECONDARY Number of Infections Per Treatment Year Requiring Intravenous Antibiotics, Antifungals or Antivirals |
0.16; 0.24 | 0.1327 |
| SECONDARY Number of Participants in Different Categories of Adverse Experiences During Core Study Period |
175; 97; 44; 19; 169; 1 | — |
Summary
The purpose of this study is to determine whether patients with high-risk myelodysplastic syndromes (MDS) treated with azacitidine have improved survival compared to conventional care treatments. The study will also assess the effect of treatments on response, duration of response, and transformation to acute myeloid leukemia (AML). The study will continue for 12 months following last patient enrolled.
See study AZA PH GL 2003 CL 001 E for information about the extension to this study.
Eligibility Criteria
Inclusion Criteria
- Have a diagnosis of refractory anemia with excess blasts or refractory anemia with excess blasts in transformation according to the French-American-British classification system for myelodysplastic syndromes (MDS) and a relatively high risk of acute myeloid leukemia (AML) transformation, with an International Prognostic Scoring System score of INT-2 or High.
- Be 18 years of age or older
- Have a life expectancy of at least 3 months
- Be 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 normal 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 times the upper limit of normal (unless these are considered to be related to transfusion-induced secondary hemosiderosis)
- Have serum creatinine levels less than or equal to 1.5 times the upper limit of normal
Exclusion Criteria
- Secondary myelodysplastic syndromes (MDS)
- Prior treatment with azacitidine;
- Prior history of acute myeloid leukemia (AML);
- Malignant disease diagnosed within prior 12 months;
- Metastatic disease;
- Hepatic tumors;
- Radiation, chemotherapy, cytotoxic therapy for non-MDS conditions within prior 12 months;
- Prior transplantation or cytotoxic therapy to treat MDS;
- Serious medical illness likely to limit survival to 12 months or less;
- Treatment with erythropoietin or myeloid growth factors during prior 21 days or androgenic hormones during prior 13 days;
- Active HIV, viral hepatitis type B or C;
- Treatment with investigational drugs during prior 30 days;
- Within the 28-day screening period, documented red cell folate deficiency, as evidenced by red blood cell folate (not serum folate) or vitamin B12 deficiency
Data sourced from ClinicalTrials.gov (NCT00071799). 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.