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

Primary Purpose

Myelodysplastic Syndromes

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Azacitidine
Physician Choice
Sponsored by
Celgene
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Myelodysplastic Syndromes

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Alabama School of Medicine
  • Indiana University Cancer Center
  • Washington University School of Medicine
  • Mount Sinai Medical Center
  • Case Western Reserve University
  • Oregon Cancer Center
  • Western Pennsylvania Cancer Institute
  • Froedtert Memorial Lutheran Hospital
  • Liverpool Hospital
  • Royal North Shore Hospital
  • The Newcastle Mater Miseriecordiae Hospital
  • Royal Brisbane Hospital
  • Princess Alexandra Hospital
  • Royal Adelaide Hospital
  • Peter MacCallum Cancer Institute
  • Royal Melbourne Hospital
  • The Alfred Hospital
  • The Royal Perth Hospital
  • First Clinical Base - Clinic of Hematology, MHAT - Pleven
  • MHAT "St George" Clinic of Hematology, Plovdiv
  • III-rd Internal Department, District Dispensary for Oncology diseases with stationary(DDOncDIU)
  • National Centre of Hematology and Transfusiology, Sofia
  • Multiprofile Hospital for Active Treatment (MHAT), "St. Marina" Clinic of Hematology
  • University Multiprofile Hospital for Active Treatment "Sveta Marina"
  • Fakultni nemocnice Brno
  • Fakultni nemocnice Hradec Kralove
  • Fakultni Nemocnice Olomouc
  • Vseobecna Fakultni Nemocnice
  • Uslav Hematologie a Krevni Transfuze
  • Chu D'Angers
  • Hopital Beaujon
  • Che De Lille
  • Hospital Edouard Herriot
  • Institute Paoli Calmettes
  • Chu De Nantes
  • Hospital Saint Louis
  • Hopital Cochin
  • Centre Henri Becquerel
  • Chu Purpan
  • Universitatsklinikum Benjamin Franklin
  • Universitatsklinikum Bonn
  • Klinikum Chemnitz gGmbH
  • Universitatsklinikum Carl Gustav Carus
  • St Johannes Hospital
  • Heinrich-Heine University Dusseldorf
  • University Essen
  • Gerorg-August-Universitat Gottingen
  • Allgemeines Krankenhaus St. Georg
  • Universitatsklinikum Hambur-Eppendorf
  • Universitatsklinikum Kiel II
  • Universitatsklinikum Ulm
  • University Hospital-Attikon
  • University General Hospital of Heraklio Voutes
  • District General Hospital of Athens
  • General Hospital of Chest Disease
  • University General Hospital of Ioannina
  • University General Hospital of Patra Rio
  • Orszagos Gyogyintezeti Kozpont
  • University of Pecs, 1st Dept of Internal Medicine
  • University of Szeged, 2nd Department of Internal Medicine
  • Policlinico S. Orsola-Malpighi
  • Universita di Firenze
  • Ospedale San Martino
  • Instituto Nazionale Dei Tumori
  • Centro Oncologico Modenese
  • Ospedale San Eugenio
  • Policlinico Gemelli
  • Instituto Nazionale Tumori "Regina Elena"
  • Ospedale Casa Sollievo Della Sofferenza - Irrc
  • Universita Degli Studi Di Sassari
  • VU University Medical Center Amsterdam
  • Univ Hospital St. Radboud
  • Samodzielny Publiczny Szpital Kliniczny Nr 1
  • Wojewodzki Szpital Specjalistyczny
  • Samodzielny Publiczny Szpital Kliniczny
  • Wojskowy Instytut Medyczny
  • Samodzelny Publiczny Centralny Szpital Kliniczny
  • Samodzielny Publiczny Szpital Kliniczny Nr 1
  • Burdenko Central Military Clinical Hospital
  • Blokhin Cancer Research Center
  • Scientific Haematology Center, Moscow
  • Institute of Haematology & Blood Transfusion
  • Pavlov State Medical University
  • Pavlov State Medical University
  • City Hospital #31
  • Hospital Santa Creu I Sant Pau
  • Hospital Clinic
  • Hospital Universitario Germans Trias I Pujol
  • Hospital de Leon
  • Hospital Universitario De La Princesa
  • Hospital Ramon Y Cajal
  • Hospital La Paz, Madrid
  • Hospital Clinico San Carlos
  • Hospital Son Llatzer
  • Hospital Universitario Del Salamanca
  • Hospital Universitario La Fe
  • Sahlgrenska University Hospital
  • Lund Universtiy Hospital
  • University Hospital MAS
  • Huddinge University Hospital
  • Uppsala University Hospital
  • Royal Bournemouth General Hospital
  • St. Bartholomew's Hospital
  • Kings College Hospital NHS Trust
  • Christie Hospital
  • Norfolk and Norwich University Hospital
  • John Radcliffe Hospital
  • Royal Cornwall Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Azacitidine

Conventional Care

Arm Description

Study Drug plus best supportive care. Treatment with erythropoietin was not permitted

Physician choice of low dose cytarabine (plus best supportive care), standard chemotherapy (plus best supportive care) or best supportive care (only). Treatment with erythropoietin was not permitted

Outcomes

Primary Outcome Measures

Kaplan-Meier Estimates for Median Time to Death From Any Cause
Kaplan-Meier estimates for the median months until death from any cause within the intent-to-treat population. Patients surviving at the end of the follow-up period were censored at the date of last contact. If a patient withdrew consent to follow-up or was lost to follow-up, the patient was censored as of the last date of contact.
Summary of Subgroup Analyses for Kaplan-Meier Estimates for Time to Death From Any Cause
Kaplan-Meier estimates for the median months until death from any cause within the intent-to-treat population. Patients surviving at the end of the follow-up period were censored at the date of last contact. If a patient withdrew consent to follow-up or was lost to follow-up, the patient was censored as of the last date of contact. Subgroups that were analyzed are age, gender, French-American-British (FAB) classification, World Health Organization (WHO) classification and International Prognostic Scoring System (IPSS) classification.
Number of Participants Who Died
Count of participants who died during the study

Secondary Outcome Measures

Kaplan-Meier Estimate for Median Time to Transformation to Acute Myeloid Leukemia (AML) or Death From Any Cause, Whichever Occurred First
The time to transformation to AML or death from any cause (whichever occurred first) was defined as the number of days from the date of randomization until the date of documented AML transformation or death from any cause. Patients who did not transform to AML or die were censored at the date of last follow-up.
Kaplan-Meier Estimates for Median Time to Transformation to Acute Myeloid Leukemia (AML)
The time to transformation to AML was defined as the number of days from the date of randomization until the date of documented AML transformation, defined as a bone marrow blast count ≥ 30% independent of baseline bone marrow count. Patients who did not transform to AML were censored at the date of last follow-up or date of death.
Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Dependent at Baseline
Summary of dependence and independence from red blood cell (RBC) transfusion at baseline and during treatment, for patients who were dependent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Independent at Baseline
Summary of dependence and independence from red blood cell (RBC) transfusion at baseline and during treatment, for patients who were independent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Dependent at Baseline
Summary of dependence and independence from platelet transfusion at baseline and during treatment for patients who were dependent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Independent at Baseline
Summary of dependence and independence from platelet transfusion at baseline and during treatment for patients who were independent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Number of Participants Considered Hematologic Responders by Investigator Determinations Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS)
Investigator determined responses followed IWG criteria for complete remission(CR): repeat bone marrow show <5% myeloblasts, and peripheral blood evaluations lasting >=2 months of hemoglobin(>110 g/L), neutrophils(>=1.5x10^9/L), platelets(>=100x10^9/L), blasts (0%) and no dysplasia partial remission(PR) is the same as CR for peripheral blood: bone marrow shows blasts decrease by >=50% or a less advanced FAB classification from pretreatment stable disease(SD) is a failure to achieve at least a partial remission, but with no evidence of progression for at least 2 months.
Number of Participants Showing Hematologic Improvement Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS) Assessed by Independent Review Committee
IWG 2000 Criteria: Pretreatment=hemoglobin <100g/L or RBC transfusion-dependent, platelet count <100x10^9/L or platelet transfusion dependent, absolute neutrophil count <1.5x10^9/L. Erythroid response: Major->20g/L increase or transfusion independent. Minor- 10-20g/L increase or >=50% decrease in transfusion requirements. Platelet response: Major-absolute increase of >=30x10^9/L or platelet transfusion independence. Minor->=50% increase. Neutrophil response: Major->=100% increase or an absolute increase of >0.5x10^9/L. Minor->=100% increase and absolute increase of <0.5x10^9/L.
Time to Disease Progression, Relapse After Complete or Partial Remission, or Death From Any Cause
The time to disease progression, relapse after complete or partial remission (CR, PR), or death from any cause was defined as the time from the date of randomization until the first date of documented disease progression, relapse after CR or PR, or death from any cause.
Duration of Any Hematologic Improvement
The duration of improvement was defined as the time from the date of hematologic improvement until the date of first documented progression or relapse after hematologic improvement or death from any cause.
Number of Infections Per Treatment Year Requiring Intravenous Antibiotics, Antifungals or Antivirals
The on-treatment adverse event rate of infection requiring IV antibiotics, antifungals, or antivirals per patient-years. The on-treatment period was considered the period from the date of randomization to the last treatment study visit.
Number of Participants in Different Categories of Adverse Experiences During Core Study Period
Patient counts for a variety of subsets of adverse experiences for the core study period (day 1 to 42 months). The individual options for Conventional Care Regimens (Best Supportive Care Only, Low-Dose Cytarabine, and Standard Chemotherapy) are presented as separate treatments.

Full Information

First Posted
October 31, 2003
Last Updated
October 16, 2019
Sponsor
Celgene
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1. Study Identification

Unique Protocol Identification Number
NCT00071799
Brief Title
A Survival Study in Patients With High Risk Myelodysplastic Syndromes Comparing Azacitidine Versus Conventional Care
Official 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)
Study Type
Interventional

2. Study Status

Record Verification Date
October 2019
Overall Recruitment Status
Completed
Study Start Date
November 1, 2003 (Actual)
Primary Completion Date
July 1, 2007 (Actual)
Study Completion Date
July 1, 2007 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Celgene

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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.
Detailed Description
Comparison/Control Interventions offered the physician three options: Best supportive care (BSC) alone, Low-dose cytarabine subcutaneously for 14 days every 28 to 42 days, or Standard chemotherapy administered for induction as a continuous intravenous infusion of cytarabine over 7 days plus an anthracycline (daunorubicin, idarubicin, or mitoxantrone) on Days 1, 2, and 3; and, for those eligible, 1 or 2 consolidation cycles administered as continuous intravenous infusions of cytarabine for 3 to 7 days with the same anthracycline that was used at induction on Days 1 and 2 (each cycle between 28 to 70 days from the start of the previous cycle). All three options included best supportive care. Neither the experimental group (azacitidine) nor any of the comparison/control options allowed use of erythropoietin. Duration of Intervention: Patients will be treated until death, withdrawal, unacceptable toxicity or conclusion of the study.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myelodysplastic Syndromes

7. Study Design

Primary Purpose
Other
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
358 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Azacitidine
Arm Type
Experimental
Arm Description
Study Drug plus best supportive care. Treatment with erythropoietin was not permitted
Arm Title
Conventional Care
Arm Type
Active Comparator
Arm Description
Physician choice of low dose cytarabine (plus best supportive care), standard chemotherapy (plus best supportive care) or best supportive care (only). Treatment with erythropoietin was not permitted
Intervention Type
Drug
Intervention Name(s)
Azacitidine
Other Intervention Name(s)
AZA
Intervention Description
Azacitidine was injected subcutaneously (SC) at an initial dose of 75mg/m^2/day for 7 days. The 7-day dosing was repeated every 28 days with dose adjustment based on predefined hematology and renal laboratory results. Number of cycles: Azacitidine treatment was to be continued until the end of the study unless treatment was discontinued due to unacceptable toxicity, relapse after complete or partial response, transformation to AML or disease progression.
Intervention Type
Other
Intervention Name(s)
Physician Choice
Other Intervention Name(s)
cytarabine, anthracycline
Intervention Description
Physician Choice was one of three options: Best supportive care (BSC) alone, Low-dose cytarabine subcutaneously for 14 days every 28 to 42 days, or Standard chemotherapy administered for induction as a continuous intravenous infusion of cytarabine over 7 days plus an anthracycline (daunorubicin, idarubicin, or mitoxantrone) on Days 1, 2, and 3; and, for those eligible, 1 or 2 consolidation cycles administered as continuous intravenous infusions of cytarabine for 3 to 7 days with the same anthracycline that was used at induction on Days 1 and 2 (each cycle between 28 to 70 days from the start of the previous cycle). All three options included best supportive care
Primary Outcome Measure Information:
Title
Kaplan-Meier Estimates for Median Time to Death From Any Cause
Description
Kaplan-Meier estimates for the median months until death from any cause within the intent-to-treat population. Patients surviving at the end of the follow-up period were censored at the date of last contact. If a patient withdrew consent to follow-up or was lost to follow-up, the patient was censored as of the last date of contact.
Time Frame
Day 1 (randomization) to 42 months
Title
Summary of Subgroup Analyses for Kaplan-Meier Estimates for Time to Death From Any Cause
Description
Kaplan-Meier estimates for the median months until death from any cause within the intent-to-treat population. Patients surviving at the end of the follow-up period were censored at the date of last contact. If a patient withdrew consent to follow-up or was lost to follow-up, the patient was censored as of the last date of contact. Subgroups that were analyzed are age, gender, French-American-British (FAB) classification, World Health Organization (WHO) classification and International Prognostic Scoring System (IPSS) classification.
Time Frame
Day 1 (randomization) to 42 months
Title
Number of Participants Who Died
Description
Count of participants who died during the study
Time Frame
42 months
Secondary Outcome Measure Information:
Title
Kaplan-Meier Estimate for Median Time to Transformation to Acute Myeloid Leukemia (AML) or Death From Any Cause, Whichever Occurred First
Description
The time to transformation to AML or death from any cause (whichever occurred first) was defined as the number of days from the date of randomization until the date of documented AML transformation or death from any cause. Patients who did not transform to AML or die were censored at the date of last follow-up.
Time Frame
Day 1 (randomization) to 42 months
Title
Kaplan-Meier Estimates for Median Time to Transformation to Acute Myeloid Leukemia (AML)
Description
The time to transformation to AML was defined as the number of days from the date of randomization until the date of documented AML transformation, defined as a bone marrow blast count ≥ 30% independent of baseline bone marrow count. Patients who did not transform to AML were censored at the date of last follow-up or date of death.
Time Frame
Day 1 (randomization) to 42 months
Title
Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Dependent at Baseline
Description
Summary of dependence and independence from red blood cell (RBC) transfusion at baseline and during treatment, for patients who were dependent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Time Frame
Day 1 (randomization) to 42 months
Title
Summary of Participants' Red Blood Cell (RBC) Transfusion Status for Participants Who Were Transfusion Independent at Baseline
Description
Summary of dependence and independence from red blood cell (RBC) transfusion at baseline and during treatment, for patients who were independent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Time Frame
Day 1 (randomization) to 42 months
Title
Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Dependent at Baseline
Description
Summary of dependence and independence from platelet transfusion at baseline and during treatment for patients who were dependent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Time Frame
Day 1 (randomization) to 42 months
Title
Summary of Participants' Platelet Transfusion Status for Participants Who Were Transfusion Independent at Baseline
Description
Summary of dependence and independence from platelet transfusion at baseline and during treatment for patients who were independent at baseline. A patient was considered transfusion independent at baseline if the patient had no transfusions during the 56 days prior to randomization. During study, a patient was considered transfusion independent during the on-treatment period if the patient had no transfusions during any 56 consecutive days or more. Otherwise, the patient was considered transfusion dependent.
Time Frame
Day 1 (randomization) to 42 months
Title
Number of Participants Considered Hematologic Responders by Investigator Determinations Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS)
Description
Investigator determined responses followed IWG criteria for complete remission(CR): repeat bone marrow show <5% myeloblasts, and peripheral blood evaluations lasting >=2 months of hemoglobin(>110 g/L), neutrophils(>=1.5x10^9/L), platelets(>=100x10^9/L), blasts (0%) and no dysplasia partial remission(PR) is the same as CR for peripheral blood: bone marrow shows blasts decrease by >=50% or a less advanced FAB classification from pretreatment stable disease(SD) is a failure to achieve at least a partial remission, but with no evidence of progression for at least 2 months.
Time Frame
Day 1 to 42 months
Title
Number of Participants Showing Hematologic Improvement Using International Working Group (IWG 2000) Criteria for Myelodysplastic Syndrome (MDS) Assessed by Independent Review Committee
Description
IWG 2000 Criteria: Pretreatment=hemoglobin <100g/L or RBC transfusion-dependent, platelet count <100x10^9/L or platelet transfusion dependent, absolute neutrophil count <1.5x10^9/L. Erythroid response: Major->20g/L increase or transfusion independent. Minor- 10-20g/L increase or >=50% decrease in transfusion requirements. Platelet response: Major-absolute increase of >=30x10^9/L or platelet transfusion independence. Minor->=50% increase. Neutrophil response: Major->=100% increase or an absolute increase of >0.5x10^9/L. Minor->=100% increase and absolute increase of <0.5x10^9/L.
Time Frame
Day 1 to 42 months
Title
Time to Disease Progression, Relapse After Complete or Partial Remission, or Death From Any Cause
Description
The time to disease progression, relapse after complete or partial remission (CR, PR), or death from any cause was defined as the time from the date of randomization until the first date of documented disease progression, relapse after CR or PR, or death from any cause.
Time Frame
Day 1 (randomization) to 42 months
Title
Duration of Any Hematologic Improvement
Description
The duration of improvement was defined as the time from the date of hematologic improvement until the date of first documented progression or relapse after hematologic improvement or death from any cause.
Time Frame
Day 1 (randomization) to 42 months
Title
Number of Infections Per Treatment Year Requiring Intravenous Antibiotics, Antifungals or Antivirals
Description
The on-treatment adverse event rate of infection requiring IV antibiotics, antifungals, or antivirals per patient-years. The on-treatment period was considered the period from the date of randomization to the last treatment study visit.
Time Frame
Day 1 (randomization) to 42 months
Title
Number of Participants in Different Categories of Adverse Experiences During Core Study Period
Description
Patient counts for a variety of subsets of adverse experiences for the core study period (day 1 to 42 months). The individual options for Conventional Care Regimens (Best Supportive Care Only, Low-Dose Cytarabine, and Standard Chemotherapy) are presented as separate treatments.
Time Frame
Day 1 (randomization) to 42 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
CL Beach
Organizational Affiliation
Celgene Corporation
Official's Role
Study Director
Facility Information:
Facility Name
University of Alabama School of Medicine
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
Indiana University Cancer Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Mount Sinai Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10029-6574
Country
United States
Facility Name
Case Western Reserve University
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Oregon Cancer Center
City
Portland
State/Province
Oregon
ZIP/Postal Code
97201
Country
United States
Facility Name
Western Pennsylvania Cancer Institute
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Froedtert Memorial Lutheran Hospital
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Liverpool Hospital
City
Liverpool
State/Province
New South Wales
ZIP/Postal Code
2170
Country
Australia
Facility Name
Royal North Shore Hospital
City
St. Leonards
State/Province
New South Wales
ZIP/Postal Code
2065
Country
Australia
Facility Name
The Newcastle Mater Miseriecordiae Hospital
City
Warratah
State/Province
New South Wales
ZIP/Postal Code
2298
Country
Australia
Facility Name
Royal Brisbane Hospital
City
Hersten
State/Province
Queensland
ZIP/Postal Code
4029
Country
Australia
Facility Name
Princess Alexandra Hospital
City
Woolloongabba
State/Province
Queensland
ZIP/Postal Code
4102
Country
Australia
Facility Name
Royal Adelaide Hospital
City
Adelaide
State/Province
South Australia
ZIP/Postal Code
5000
Country
Australia
Facility Name
Peter MacCallum Cancer Institute
City
East Melbourne
State/Province
Victoria
ZIP/Postal Code
3002
Country
Australia
Facility Name
Royal Melbourne Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Name
The Alfred Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3181
Country
Australia
Facility Name
The Royal Perth Hospital
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6847
Country
Australia
Facility Name
First Clinical Base - Clinic of Hematology, MHAT - Pleven
City
Pleven
ZIP/Postal Code
5800
Country
Bulgaria
Facility Name
MHAT "St George" Clinic of Hematology, Plovdiv
City
Plovdiv
ZIP/Postal Code
4002
Country
Bulgaria
Facility Name
III-rd Internal Department, District Dispensary for Oncology diseases with stationary(DDOncDIU)
City
Plovdiv
ZIP/Postal Code
4004
Country
Bulgaria
Facility Name
National Centre of Hematology and Transfusiology, Sofia
City
Sofia
ZIP/Postal Code
1756
Country
Bulgaria
Facility Name
Multiprofile Hospital for Active Treatment (MHAT), "St. Marina" Clinic of Hematology
City
Varna
ZIP/Postal Code
3010
Country
Bulgaria
Facility Name
University Multiprofile Hospital for Active Treatment "Sveta Marina"
City
Varna
ZIP/Postal Code
9010
Country
Bulgaria
Facility Name
Fakultni nemocnice Brno
City
Jihlavska
State/Province
Brno
ZIP/Postal Code
639 00
Country
Czechia
Facility Name
Fakultni nemocnice Hradec Kralove
City
Sokolska
State/Province
Hradec Kralove
ZIP/Postal Code
500 05
Country
Czechia
Facility Name
Fakultni Nemocnice Olomouc
City
Olomouc
ZIP/Postal Code
775 20
Country
Czechia
Facility Name
Vseobecna Fakultni Nemocnice
City
Praha
ZIP/Postal Code
2 128 08
Country
Czechia
Facility Name
Uslav Hematologie a Krevni Transfuze
City
Praha
ZIP/Postal Code
2 128 20
Country
Czechia
Facility Name
Chu D'Angers
City
Angers
ZIP/Postal Code
49033
Country
France
Facility Name
Hopital Beaujon
City
Clichy
ZIP/Postal Code
92110
Country
France
Facility Name
Che De Lille
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
Hospital Edouard Herriot
City
Lyon
ZIP/Postal Code
69437
Country
France
Facility Name
Institute Paoli Calmettes
City
Marseille
ZIP/Postal Code
13009
Country
France
Facility Name
Chu De Nantes
City
Nantes
ZIP/Postal Code
44093
Country
France
Facility Name
Hospital Saint Louis
City
Paris
ZIP/Postal Code
75010
Country
France
Facility Name
Hopital Cochin
City
Paris
ZIP/Postal Code
75679
Country
France
Facility Name
Centre Henri Becquerel
City
Rouen
ZIP/Postal Code
76038
Country
France
Facility Name
Chu Purpan
City
Toulouse
ZIP/Postal Code
31059
Country
France
Facility Name
Universitatsklinikum Benjamin Franklin
City
Hindenburgdamm
State/Province
Berlin
ZIP/Postal Code
D-12203
Country
Germany
Facility Name
Universitatsklinikum Bonn
City
Bonn
ZIP/Postal Code
53105
Country
Germany
Facility Name
Klinikum Chemnitz gGmbH
City
Chemnitz
ZIP/Postal Code
9113
Country
Germany
Facility Name
Universitatsklinikum Carl Gustav Carus
City
Dresden
ZIP/Postal Code
1307
Country
Germany
Facility Name
St Johannes Hospital
City
Duisburg
ZIP/Postal Code
47166
Country
Germany
Facility Name
Heinrich-Heine University Dusseldorf
City
Dusseldorf
ZIP/Postal Code
40225
Country
Germany
Facility Name
University Essen
City
Essen
ZIP/Postal Code
45147
Country
Germany
Facility Name
Gerorg-August-Universitat Gottingen
City
Gottingen
ZIP/Postal Code
37075
Country
Germany
Facility Name
Allgemeines Krankenhaus St. Georg
City
Hamburg
ZIP/Postal Code
D-20099
Country
Germany
Facility Name
Universitatsklinikum Hambur-Eppendorf
City
Hamburg
ZIP/Postal Code
D-20246
Country
Germany
Facility Name
Universitatsklinikum Kiel II
City
Kiel
ZIP/Postal Code
D-24116
Country
Germany
Facility Name
Universitatsklinikum Ulm
City
Ulm
ZIP/Postal Code
89070
Country
Germany
Facility Name
University Hospital-Attikon
City
Haidari
State/Province
Athens
ZIP/Postal Code
12462
Country
Greece
Facility Name
University General Hospital of Heraklio Voutes
City
Heraklio
State/Province
Crete
ZIP/Postal Code
71110
Country
Greece
Facility Name
District General Hospital of Athens
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
General Hospital of Chest Disease
City
Athens
ZIP/Postal Code
11527
Country
Greece
Facility Name
University General Hospital of Ioannina
City
Ioannina
ZIP/Postal Code
45500
Country
Greece
Facility Name
University General Hospital of Patra Rio
City
Patra
ZIP/Postal Code
26500
Country
Greece
Facility Name
Orszagos Gyogyintezeti Kozpont
City
Budapest
ZIP/Postal Code
1135
Country
Hungary
Facility Name
University of Pecs, 1st Dept of Internal Medicine
City
Pecs
ZIP/Postal Code
7624
Country
Hungary
Facility Name
University of Szeged, 2nd Department of Internal Medicine
City
Szeged
ZIP/Postal Code
6701
Country
Hungary
Facility Name
Policlinico S. Orsola-Malpighi
City
Bologna
ZIP/Postal Code
40138
Country
Italy
Facility Name
Universita di Firenze
City
Firenze
ZIP/Postal Code
50139
Country
Italy
Facility Name
Ospedale San Martino
City
Genova
ZIP/Postal Code
I-16132
Country
Italy
Facility Name
Instituto Nazionale Dei Tumori
City
Milano
ZIP/Postal Code
20133
Country
Italy
Facility Name
Centro Oncologico Modenese
City
Modena
ZIP/Postal Code
41100
Country
Italy
Facility Name
Ospedale San Eugenio
City
Roma
ZIP/Postal Code
00144
Country
Italy
Facility Name
Policlinico Gemelli
City
Roma
ZIP/Postal Code
00168
Country
Italy
Facility Name
Instituto Nazionale Tumori "Regina Elena"
City
Roma
ZIP/Postal Code
144
Country
Italy
Facility Name
Ospedale Casa Sollievo Della Sofferenza - Irrc
City
San Giovanni Rotondo
ZIP/Postal Code
71013
Country
Italy
Facility Name
Universita Degli Studi Di Sassari
City
Sassari
ZIP/Postal Code
7100
Country
Italy
Facility Name
VU University Medical Center Amsterdam
City
Amsterdam
ZIP/Postal Code
1081 HV
Country
Netherlands
Facility Name
Univ Hospital St. Radboud
City
Nijmejen
Country
Netherlands
Facility Name
Samodzielny Publiczny Szpital Kliniczny Nr 1
City
Gdansk
ZIP/Postal Code
80-952
Country
Poland
Facility Name
Wojewodzki Szpital Specjalistyczny
City
Lodz
ZIP/Postal Code
93-510
Country
Poland
Facility Name
Samodzielny Publiczny Szpital Kliniczny
City
Lublin
ZIP/Postal Code
20081
Country
Poland
Facility Name
Wojskowy Instytut Medyczny
City
Warszawa
ZIP/Postal Code
00-909
Country
Poland
Facility Name
Samodzelny Publiczny Centralny Szpital Kliniczny
City
Warszawa
ZIP/Postal Code
02-097
Country
Poland
Facility Name
Samodzielny Publiczny Szpital Kliniczny Nr 1
City
Wroclaw
ZIP/Postal Code
50-367
Country
Poland
Facility Name
Burdenko Central Military Clinical Hospital
City
Moscow
ZIP/Postal Code
105299
Country
Russian Federation
Facility Name
Blokhin Cancer Research Center
City
Moscow
ZIP/Postal Code
115487
Country
Russian Federation
Facility Name
Scientific Haematology Center, Moscow
City
Moscow
ZIP/Postal Code
125167
Country
Russian Federation
Facility Name
Institute of Haematology & Blood Transfusion
City
St. Petersburg
ZIP/Postal Code
193024
Country
Russian Federation
Facility Name
Pavlov State Medical University
City
St. Petersburg
ZIP/Postal Code
197022
Country
Russian Federation
Facility Name
Pavlov State Medical University
City
St. Petersburg
ZIP/Postal Code
197089
Country
Russian Federation
Facility Name
City Hospital #31
City
St. Petersburg
ZIP/Postal Code
197110
Country
Russian Federation
Facility Name
Hospital Santa Creu I Sant Pau
City
Barcelona
ZIP/Postal Code
08025
Country
Spain
Facility Name
Hospital Clinic
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Hospital Universitario Germans Trias I Pujol
City
Barcelona
Country
Spain
Facility Name
Hospital de Leon
City
Leon
ZIP/Postal Code
24071
Country
Spain
Facility Name
Hospital Universitario De La Princesa
City
Madrid
ZIP/Postal Code
28006
Country
Spain
Facility Name
Hospital Ramon Y Cajal
City
Madrid
ZIP/Postal Code
28034
Country
Spain
Facility Name
Hospital La Paz, Madrid
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Hospital Clinico San Carlos
City
Madrid
ZIP/Postal Code
28048
Country
Spain
Facility Name
Hospital Son Llatzer
City
Palma de Mallorca
ZIP/Postal Code
07198
Country
Spain
Facility Name
Hospital Universitario Del Salamanca
City
Salamanca
ZIP/Postal Code
37007
Country
Spain
Facility Name
Hospital Universitario La Fe
City
Valencia
ZIP/Postal Code
46009
Country
Spain
Facility Name
Sahlgrenska University Hospital
City
Goteborg
ZIP/Postal Code
S-413 45
Country
Sweden
Facility Name
Lund Universtiy Hospital
City
Lund
ZIP/Postal Code
22185
Country
Sweden
Facility Name
University Hospital MAS
City
Malmo
ZIP/Postal Code
S-205 02
Country
Sweden
Facility Name
Huddinge University Hospital
City
Stockholm
ZIP/Postal Code
14186
Country
Sweden
Facility Name
Uppsala University Hospital
City
Uppsala
ZIP/Postal Code
S-751 85
Country
Sweden
Facility Name
Royal Bournemouth General Hospital
City
Bournemouth
ZIP/Postal Code
BH7 7DW
Country
United Kingdom
Facility Name
St. Bartholomew's Hospital
City
London
ZIP/Postal Code
EC1A 7BE
Country
United Kingdom
Facility Name
Kings College Hospital NHS Trust
City
London
Country
United Kingdom
Facility Name
Christie Hospital
City
Manchester
ZIP/Postal Code
M20 4BX
Country
United Kingdom
Facility Name
Norfolk and Norwich University Hospital
City
Norwich
ZIP/Postal Code
NR4 7UY
Country
United Kingdom
Facility Name
John Radcliffe Hospital
City
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom
Facility Name
Royal Cornwall Hospital
City
Truro
ZIP/Postal Code
TR1 3LJ
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
20026804
Citation
Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Gattermann N, Germing U, Sanz G, List AF, Gore S, Seymour JF, Dombret H, Backstrom J, Zimmerman L, McKenzie D, Beach CL, Silverman LR. Azacitidine prolongs overall survival compared with conventional care regimens in elderly patients with low bone marrow blast count acute myeloid leukemia. J Clin Oncol. 2010 Feb 1;28(4):562-9. doi: 10.1200/JCO.2009.23.8329. Epub 2009 Dec 21.
Results Reference
result
PubMed Identifier
20136825
Citation
Fenaux P, Gattermann N, Seymour JF, Hellstrom-Lindberg E, Mufti GJ, Duehrsen U, Gore SD, Ramos F, Beyne-Rauzy O, List A, McKenzie D, Backstrom J, Beach CL. Prolonged survival with improved tolerability in higher-risk myelodysplastic syndromes: azacitidine compared with low dose ara-C. Br J Haematol. 2010 Apr;149(2):244-9. doi: 10.1111/j.1365-2141.2010.08082.x. Epub 2010 Feb 5. Erratum In: Br J Haematol. 2010 Jun;149(6):919.
Results Reference
result
PubMed Identifier
20394651
Citation
Santini V, Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Silverman LR, List A, Gore SD, Seymour JF, Backstrom J, Beach CL. Management and supportive care measures for adverse events in patients with myelodysplastic syndromes treated with azacitidine*. Eur J Haematol. 2010 Aug;85(2):130-8. doi: 10.1111/j.1600-0609.2010.01456.x. Epub 2010 Apr 12.
Results Reference
result
PubMed Identifier
20451404
Citation
Seymour JF, Fenaux P, Silverman LR, Mufti GJ, Hellstrom-Lindberg E, Santini V, List AF, Gore SD, Backstrom J, McKenzie D, Beach CL. Effects of azacitidine compared with conventional care regimens in elderly (>/= 75 years) patients with higher-risk myelodysplastic syndromes. Crit Rev Oncol Hematol. 2010 Dec;76(3):218-27. doi: 10.1016/j.critrevonc.2010.04.005. Epub 2010 May 6.
Results Reference
result
PubMed Identifier
19230772
Citation
Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR; International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. doi: 10.1016/S1470-2045(09)70003-8. Epub 2009 Feb 21.
Results Reference
result
PubMed Identifier
23585522
Citation
Gore SD, Fenaux P, Santini V, Bennett JM, Silverman LR, Seymour JF, Hellstrom-Lindberg E, Swern AS, Beach CL, List AF. A multivariate analysis of the relationship between response and survival among patients with higher-risk myelodysplastic syndromes treated within azacitidine or conventional care regimens in the randomized AZA-001 trial. Haematologica. 2013 Jul;98(7):1067-72. doi: 10.3324/haematol.2012.074831. Epub 2013 Apr 12.
Results Reference
derived
Links:
URL
http://www.clinicaltrials.gov/ct2/show/NCT01186939?term=NCT01186939&rank=1
Description
Study record for the extension study of AZA PH GL 2003 CL 001

Learn more about this trial

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

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