A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With International Prognostic Scoring System Revised (IPSS-R) Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)

February 24, 2026 updated by: Bristol-Myers Squibb

A Phase 2/3, Multicenter, Randomized, Dose Optimization (Part I), Double-blind (Part II) Study to Compare the Efficacy and Safety of Oral Azacitidine (Oral-Aza, ONUREG®) Plus Best Supportive Care (BSC) Versus Placebo Plus BSC in Participants With IPSS-R Low- or Intermediate-risk Myelodysplastic Syndrome (MDS)

The purpose of this study is to evaluate the safety and efficacy of oral azacitidine in participants with low to intermediate International Prognostic Scoring System Revised (IPSS-R) myelodysplastic syndrome (MDS).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
230

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: First line of the email MUST contain NCT # and Site #.

Study Contact Backup

  • Name: BMS Study Connect Contact Center http://www.bmsstudyconnect.com
  • Phone Number: 855-907-3286
  • Email: Clinical.Trials@bms.com

Study Locations

  • Argentina
      • Buenos Aires, Argentina, CP1280AEB
        • Local Institution - 0050
      • Buenos Aires, Argentina, 1425
        • Local Institution - 0016
      • Buenos Aires, Argentina, 1431
        • Local Institution - 0022
    • Buenos Aires
      • Pilar, Buenos Aires, Argentina, 1629
        • Local Institution - 0070
    • Buenos Aires F.D.
      • ABB, Buenos Aires F.D., Argentina, C1199ABB
        • Local Institution - 0039
  • Australia
      • Melbourne, Australia, 3004
        • Local Institution - 0003
    • Victoria
      • Clayton, Victoria, Australia, 3168
        • Local Institution - 0006
      • Melbourne, Victoria, Australia, 3000
        • Local Institution - 0018
      • Melbourne, Victoria, Australia, 3065
        • Local Institution - 0004
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M4N 3M5
        • Local Institution - 0008
      • Toronto, Ontario, Canada, M5G 2M9
        • Local Institution - 0015
    • Quebec
      • Montreal, Quebec, Canada, H4A 3J1
        • Local Institution - 0090
  • China
    • Hubei
      • Wuhan, Hubei, China, 430030
        • Local Institution - 0156
  • Czechia
      • Hradec Králové, Czechia, 500 05
        • Local Institution - 0060
  • Denmark
    • Central Jutland
      • Aarhus, Central Jutland, Denmark, 8200
        • Local Institution - 0115
    • North Denmark
      • Aalborg, North Denmark, Denmark, 9000
        • Local Institution - 0116
  • France
      • Paris, France, 75010
        • Local Institution - 0082
    • Aquitaine
      • Pessac, Aquitaine, France, 33600
        • Local Institution - 0063
    • Indre-et-Loire
      • Tours, Indre-et-Loire, France, 37032
        • Local Institution - 0024
    • Maine-et-Loire
      • Angers, Maine-et-Loire, France, 49933
        • Local Institution - 0094
    • Nord
      • Lille, Nord, France, 59000
        • Local Institution - 0056
    • Val-de-Marne
      • Villejuif, Val-de-Marne, France, 94805
        • Local Institution - 0085
  • Germany
      • Dresden, Germany, 01307
        • Local Institution - 0037
      • Hamburg, Germany, 22081
        • Local Institution - 0007
      • Mutlangen, Germany, 73557
        • Local Institution - 0028
    • North Rhine-Westphalia
      • Duisburg, North Rhine-Westphalia, Germany, 47166
        • Local Institution - 0081
      • Düsseldorf, North Rhine-Westphalia, Germany, 40479
        • Local Institution - 0128
    • Saxony
      • Leipzig, Saxony, Germany, 04103
        • Local Institution - 0055
  • Greece
      • Alexandroupoli, Greece, 08100
        • Local Institution - 0127
    • Attikí
      • Chaïdári, Attikí, Greece, 12462
        • Local Institution - 0125
    • Thessaloníki
      • Thessaloniki, Thessaloníki, Greece, 570 10
        • Local Institution - 0129
  • Hong Kong
      • Hksar, Hong Kong
        • Local Institution - 0178
      • Shatin, Hong Kong, NT
        • Local Institution - 0180
  • Italy
      • Bologna, Italy, 40138
        • Local Institution - 0101
    • Lazio
      • Rome, Lazio, Italy, 00133
        • Local Institution - 0061
    • Milano
      • Rozzano, Milano, Italy, 20089
        • Local Institution - 0052
    • Tuscany
      • Florence, Tuscany, Italy, 50134
        • Local Institution - 0075
  • Japan
      • Osaka, Japan, 545-8586
        • Local Institution - 0124
    • Fukuoka
      • Kitakyushu-shi, Fukuoka, Japan, 8068501
        • Local Institution - 0136
    • Hokkaido
      • Sapporo, Hokkaido, Japan, 064-0804
        • Local Institution - 0154
    • Hyōgo
      • Amagasaki, Hyōgo, Japan, 660-8550
        • Local Institution - 0153
    • Kanagawa
      • Sagamihara, Kanagawa, Japan, 252-0375
        • Local Institution - 0130
    • Miyagi
      • Sendai, Miyagi, Japan, 980-8574
        • Local Institution - 0135
    • Tokyo
      • Shinagawa-ku, Tokyo, Japan, 141-8625
        • Local Institution - 0150
  • Poland
    • Warmian-Masurian Voivodeship
      • Olsztyn, Warmian-Masurian Voivodeship, Poland, 10-228
        • Local Institution - 0097
  • South Korea
    • Jeonranamdo
      • Hwasun Gun, Jeonranamdo, South Korea, 58128
        • Local Institution - 0058
    • Seoul Teugbyeolsi
      • Seoul, Seoul Teugbyeolsi, South Korea, 06591
        • Local Institution - 0048
    • Seoul-teukbyeolsi [Seoul]
      • Seoul, Seoul-teukbyeolsi [Seoul], South Korea, 05505
        • Local Institution - 0036
      • Seoul, Seoul-teukbyeolsi [Seoul], South Korea, 06351
        • Local Institution - 0012
    • Taegu-Kwangyǒkshi
      • Junggu, Taegu-Kwangyǒkshi, South Korea, 41944
        • Local Institution - 0051
  • Spain
      • Granada, Spain, 18012
        • Local Institution - 0107
      • Madrid, Spain, 28006
        • Local Institution - 0111
      • Ourense, Spain, 32005
        • Local Institution - 0112
      • Oviedo, Spain, 33011
        • Local Institution - 0110
      • Salamanca, Spain, 37007
        • Local Institution - 0108
    • Valenciana, Comunitat
      • Valencia, Valenciana, Comunitat, Spain, 46010
        • Local Institution - 0109
  • Sweden
    • Stockholms Län [se-01]
      • Stockholm, Stockholms Län [se-01], Sweden, 141 86
        • Local Institution - 0118
    • Örebro Län [se-18]
      • Örebro, Örebro Län [se-18], Sweden, 701 85
        • Local Institution - 0119
  • United States
    • Florida
      • Miami, Florida, United States, 33136
        • Local Institution - 0137
      • Tamarac, Florida, United States, 33321
        • Local Institution - 0147
    • New York
      • East Syracuse, New York, United States, 13057
        • Local Institution - 0132
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Local Institution - 0073
    • Texas
      • Houston, Texas, United States, 77030
        • Local Institution - 0086
      • Houston, Texas, United States, 77030
        • Local Institution - 0014
    • Virginia
      • Fairfax, Virginia, United States, 22031
        • Local Institution - 0123

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

• Participant has a documented diagnosis of MDS according to WHO 2016 classification that meets International Prognostic Scoring System Revised (IPSS-R) classification of low- or intermediate-risk disease (IPSS-R score between 1.5 and 4.5).

MDS diagnosis, WHO classification, and IPSS-R risk classification will be prospectively determined by independent central pathology and cytogenetics review, and applicable central laboratory results.

• Participant must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2.

Exclusion Criteria:

  • Participants with prior malignancies must have an expected median life expectancy of at least 12 months at the time of inclusion and no active treatment of any sort for at least 24 weeks prior to randomization (including but not limited to immunotherapy or targeted therapy)
  • Hypoplastic Myelodysplastic Syndrome (MDS) with a marrow cellularity of ≤ 10%
  • Participants diagnosed with MDS with excess blasts-2 (MDS-EB2)
  • Prior treatment with azacitidine (any formulation), decitabine, or other hypomethylating agent

Other protocol-defined inclusion/exclusion criteria apply

Study Plan

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

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Part I - Oral-Aza (Dose 1)
Drug: Oral Azacitidine
Specified dose on specified days
Other Names:
  • BMS-986345
  • Oral-Aza
  • ONUREG®
Experimental: Part I - Oral-Aza (Dose 2)
Drug: Oral Azacitidine
Specified dose on specified days
Other Names:
  • BMS-986345
  • Oral-Aza
  • ONUREG®
Experimental: Part II - Oral-Aza (RP3D)
RP3D: Recommended Phase 3 Dose
Drug: Oral Azacitidine
Specified dose on specified days
Other Names:
  • BMS-986345
  • Oral-Aza
  • ONUREG®
Experimental: Part II - Placebo
Drug: Placebo for Oral Azacitidine
Specified dose on specified days

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of participants with Adverse Events (AEs) evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) criteria v.5.0
Time Frame: 6 cycles plus 28 days (up to 24 weeks)
Phase 2
6 cycles plus 28 days (up to 24 weeks)
Number of participants who achieved complete remission (CR) per International Working Group (IWG) 2006 criteria within 6 cycles
Time Frame: Up to 24 weeks
Phase 2 and 3
Up to 24 weeks

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Number of participants who achieved Overall Response (OR) per IWG 2006 criteria within 6 cycles
Time Frame: Up to 24 weeks

Phase 2 and Phase 3

Overall Response is defined as complete response (CR), partial remission (PR), marrow complete response (mCR), hematologic improvement-erythroid response (HI-E), hematologic improvement-platelet response (HI-P), or hematologic improvement-neutrophil response (HI-N) as per IWG 2006 criteria
Up to 24 weeks
Number of participants who achieved 84-day packed red blood cells transfusion independence (pRBC-TI)
Time Frame: Up to 32 weeks
Phase 2 and Phase 3
Up to 32 weeks
pRBC-TI duration
Time Frame: Over the course of the study, an average of 1 year
Phase 2 and Phase 3
Over the course of the study, an average of 1 year
Number of participants who achieve 84 day platelet transfusion independence (PLT-TI) within 6 cycles
Time Frame: Over the course of the study, an average of 1 year
Phase 2 and Phase 3
Over the course of the study, an average of 1 year
PLT-TI duration
Time Frame: Over the course of the study, an average of 1 year
Phase 2 and Phase 3
Over the course of the study, an average of 1 year
Number of participants who achieved pRBC transfusion reduction
Time Frame: Over the course of the study, an average of 1 year
Phase 3
Over the course of the study, an average of 1 year
pRBC transfusion reduction duration
Time Frame: Over the course of the study, an average of 1 year
Phase 3
Over the course of the study, an average of 1 year
CR duration
Time Frame: Over the course of the study, an average of 1 year
Phase 2 and Phase 3
Over the course of the study, an average of 1 year
Best OR
Time Frame: Over the course of the study, an average of 1 year
Phase 2 and Phase 3
Over the course of the study, an average of 1 year
OR duration
Time Frame: Over the course of the study, an average of 1 year
Phase 2 and Phase 3
Over the course of the study, an average of 1 year
Overall Survival (OS)
Time Frame: Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Phase 3
Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Event-free Survival (EFS)
Time Frame: Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Phase 3
Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Time to acute myeloid leukemia (AML)
Time Frame: Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Phase 3
Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Time to subsequent therapy
Time Frame: Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Phase 3
Up to 5 years after discontinuation of Investigational Product, approximately 6 years
Iron parameters measured from blood
Time Frame: Over the course of the study, an average of 1 year
Phase 3
Over the course of the study, an average of 1 year
Number of participants with Adverse Events (AEs) evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) criteria v.5.0
Time Frame: Up to end of treatment/early termination, an average of 1 year
Phase 3
Up to end of treatment/early termination, an average of 1 year
Summary statistics for Functional Assessment of Cancer Therapy-Anemia (FACT-An) scales and subscales at each assessment point for each treatment arm
Time Frame: Up to end of treatment/early termination, an average of 1 year
Phase 3
Up to end of treatment/early termination, an average of 1 year
Summary statistics for Quality of Life in Myelodysplasia Scale (QUALMS) scales and subscales at each assessment point for each treatment arm
Time Frame: Up to end of treatment/early termination, an average of 1 year
Phase 3
Up to end of treatment/early termination, an average of 1 year
Summary statistics for the EuroQol 5 Dimension 5 Level (EQ-5D-5L) scales and subscales at each assessment point for each treatment arm
Time Frame: Up to end of treatment/early termination, an average of 1 year
Phase 3
Up to end of treatment/early termination, an average of 1 year
Number of participants with healthcare resource use associated with the investigational product (IP)
Time Frame: Over the course of the study, an average of 1 year
Phase 3
Over the course of the study, an average of 1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Bristol-Myers Squibb

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 14, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 31, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 31, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 8, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 19, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 22, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 25, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 24, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CA055-026
  • U1111-1276-5463 (Other Grant/Funding Number: WHO)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at https://www.bms.com/researchers-and-partners/clinical-trials-and-research.html

IPD Sharing Time Frame

See Plan Description

IPD Sharing Access Criteria

See Plan Description

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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