A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00071799
• Date of registration: 31/10/2003
• Prospective Registration: Yes
• Primary sponsor: Celgene
• Public title: A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care
• Scientific title: A Multicenter, Randomized, Open-label, Parallel-group, Phase 3 Trial of Subcutaneous Azacitidine Plus Best Supportive Care Versus Conventional Care Regimens Plus Best Supportive Care for the Treatment of Myelodysplastic Syndromes (MDS)
• Date of first enrolment: November 1, 2003
• Target sample size: 358
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00071799
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Other. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Bulgaria, Czech Republic, Czechia, France, Germany, Greece, Hungary, Italy, Netherlands, Poland, Russian Federation, Spain, Sweden, United Kingdom, United States

Contacts

• Name: CL Beach
• Affiliation: Celgene Corporation

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Myelodysplastic Syndromes

Intervention(s)

Drug: Azacitidine

Other: Physician Choice

Primary Outcome(s)

Number of Participants Who Died [Time Frame: 42 months]

Kaplan-Meier Estimates for Median Time to Death From Any Cause [Time Frame: Day 1 (randomization) to 42 months]

Summary of Subgroup Analyses for Kaplan-Meier Estimates for Time to Death From Any Cause [Time Frame: Day 1 (randomization) to 42 months]

Secondary Outcome(s)

Kaplan-Meier Estimate for Median Time to Transformation to Acute Myeloid Leukemia (AML) or Death From Any Cause, Whichever Occurred First [Time Frame: Day 1 (randomization) to 42 months]

Time to Disease Progression, Relapse After Complete or Partial Remission, or Death From Any Cause [Time Frame: Day 1 (randomization) to 42 months]

Number of Participants Showing Hematologic Improvement Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS) Assessed by Independent Review Committee [Time Frame: Day 1 to 42 months]

Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Dependent at Baseline [Time Frame: Day 1 (randomization) to 42 months]

Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Dependent at Baseline [Time Frame: Day 1 (randomization) to 42 months]

Number of Infections Per Treatment Year Requiring Intravenous Antibiotics, Antifungals or Antivirals [Time Frame: Day 1 (randomization) to 42 months]

Number of Participants Considered Hematologic Responders by Investigator Determinations Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS) [Time Frame: Day 1 to 42 months]

Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Independent at Baseline [Time Frame: Day 1 (randomization) to 42 months]

Kaplan-Meier Estimates for Median Time to Transformation to Acute Myeloid Leukemia (AML) [Time Frame: Day 1 (randomization) to 42 months]

Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Independent at Baseline [Time Frame: Day 1 (randomization) to 42 months]

Number of Participants in Different Categories of Adverse Experiences During Core Study Period [Time Frame: Day 1 (randomization) to 42 months]

Duration of Any Hematologic Improvement [Time Frame: Day 1 (randomization) to 42 months]

Secondary ID(s)

AZA PH GL 2003 CL001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 05/07/2010
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00071799