Completed

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)

What is being tested

Azacitidine

+ Physician Choice

DrugOther

Who is being recruted

Bone Marrow Diseases+1

+ Hematologic Diseases

+ Hemic and Lymphatic Diseases

Over 18 Years

+20 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Other Study

Phase 3

Interventional

Study Start: November 2003

See protocol details

Summary

Principal SponsorCelgene

Last updated: January 28, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: November 1, 2003

Actual date on which the first participant was enrolled.

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.

Official TitleA 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)

NCT00071799

Principal SponsorCelgene

Last updated: January 28, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

358 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Other Study

Some studies explore topics that don't fall into a specific category. These might include innovative research, new technologies, or emerging healthcare areas.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Over 18 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic Syndromes

Criteria

7 inclusion criteria required to participate

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

Show More Criteria

13 exclusion criteria prevent from participating

Secondary myelodysplastic syndromes (MDS)

Prior treatment with azacitidine;

Prior history of acute myeloid leukemia (AML);

Malignant disease diagnosed within prior 12 months;

Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

2 intervention groups are designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

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

Group II

Active Comparator

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

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 108 locations

Suspended

Universitatsklinikum Kiel II

Kiel, GermanyOpen Universitatsklinikum Kiel II in Google Maps

Suspended

Universitatsklinikum Ulm

Ulm, Germany

Suspended

University Hospital-Attikon

Haidari, Greece

Suspended

University General Hospital of Heraklio Voutes

Heraklio, Greece

Completed108 Study Centers