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Myelodysplastic SyndromesPhase 2/3NCT05469737

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)

A Phase 2/3 study testing Oral Azacitidine in people with Myelodysplastic Syndromes, run by Bristol-Myers Squibb at 65 sites in 17 countries.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Myelodysplastic Syndromes
What is being tested?
Oral Azacitidine
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
230 participants
Where
65 sitesin 17 countries
Study dates
14 Dec 2022to 31 Jul 2028
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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

Trial locations and contacts

65 sites in 17 countries.

65 sites in 17 countries

United States7 sites

  • Local Institution - 0132

    East Syracuse, New York, 13057

  • Local Institution - 0123

    Fairfax, Virginia, 22031

  • Local Institution - 0014

    Houston, Texas, 77030

  • Local Institution - 0086

    Houston, Texas, 77030

  • Local Institution - 0137

    Miami, Florida, 33136

  • Local Institution - 0073

    Pittsburgh, Pennsylvania, 15224

  • Local Institution - 0147

    Tamarac, Florida, 33321

Other trials in United States

Japan7 sites

  • Local Institution - 0153

    Amagasaki, Hyōgo, 660-8550

  • Local Institution - 0136

    Kitakyushu-shi, Fukuoka, 8068501

  • Local Institution - 0124

    Osaka, 545-8586

  • Local Institution - 0130

    Sagamihara, Kanagawa, 252-0375

  • Local Institution - 0154

    Sapporo, Hokkaido, 064-0804

  • Local Institution - 0135

    Sendai, Miyagi, 980-8574

  • Local Institution - 0150

    Shinagawa-ku, Tokyo, 141-8625

Other trials in Japan

France6 sites

  • Local Institution - 0094

    Angers, Maine-et-Loire, 49933

  • Local Institution - 0056

    Lille, Nord, 59000

  • Local Institution - 0082

    Paris, 75010

  • Local Institution - 0063

    Pessac, Aquitaine, 33600

  • Local Institution - 0024

    Tours, Indre-et-Loire, 37032

  • Local Institution - 0085

    Villejuif, Val-de-Marne, 94805

Other trials in France

Germany6 sites

  • Local Institution - 0037

    Dresden, 01307

  • Local Institution - 0081

    Duisburg, North Rhine-Westphalia, 47166

  • Local Institution - 0128

    Düsseldorf, North Rhine-Westphalia, 40479

  • Local Institution - 0007

    Hamburg, 22081

  • Local Institution - 0055

    Leipzig, Saxony, 04103

  • Local Institution - 0028

    Mutlangen, 73557

Other trials in Germany

Spain6 sites

  • Local Institution - 0107

    Granada, 18012

  • Local Institution - 0111

    Madrid, 28006

  • Local Institution - 0112

    Ourense, 32005

  • Local Institution - 0110

    Oviedo, 33011

  • Local Institution - 0108

    Salamanca, 37007

  • Local Institution - 0109

    Valencia, Valenciana, Comunitat, 46010

Other trials in Spain

Argentina5 sites

  • Local Institution - 0039

    ABB, Buenos Aires F.D., C1199ABB

  • Local Institution - 0016

    Buenos Aires, 1425

  • Local Institution - 0022

    Buenos Aires, 1431

  • Local Institution - 0050

    Buenos Aires, CP1280AEB

  • Local Institution - 0070

    Pilar, Buenos Aires, 1629

Other trials in Argentina

South Korea5 sites

  • Local Institution - 0058

    Hwasun Gun, Jeonranamdo, 58128

  • Local Institution - 0051

    Junggu, Taegu-Kwangyǒkshi, 41944

  • Local Institution - 0048

    Seoul, Seoul Teugbyeolsi, 06591

  • Local Institution - 0036

    Seoul, Seoul-teukbyeolsi [Seoul], 05505

  • Local Institution - 0012

    Seoul, Seoul-teukbyeolsi [Seoul], 06351

Other trials in South Korea

Australia4 sites

  • Local Institution - 0006

    Clayton, Victoria, 3168

  • Local Institution - 0018

    Melbourne, Victoria, 3000

  • Local Institution - 0004

    Melbourne, Victoria, 3065

  • Local Institution - 0003

    Melbourne, 3004

Other trials in Australia

Italy4 sites

  • Local Institution - 0101

    Bologna, 40138

  • Local Institution - 0075

    Florence, Tuscany, 50134

  • Local Institution - 0061

    Rome, Lazio, 00133

  • Local Institution - 0052

    Rozzano, Milano, 20089

Other trials in Italy

Canada3 sites

  • Local Institution - 0090

    Montreal, Quebec, H4A 3J1

  • Local Institution - 0008

    Toronto, Ontario, M4N 3M5

  • Local Institution - 0015

    Toronto, Ontario, M5G 2M9

Other trials in Canada

Greece3 sites

  • Local Institution - 0127

    Alexandroupoli, 08100

  • Local Institution - 0125

    Chaïdári, Attikí, 12462

  • Local Institution - 0129

    Thessaloniki, Thessaloníki, 570 10

Other trials in Greece

Denmark2 sites

  • Local Institution - 0116

    Aalborg, North Denmark, 9000

  • Local Institution - 0115

    Aarhus, Central Jutland, 8200

Other trials in Denmark

Hong Kong2 sites

  • Local Institution - 0178

    Hksar, 0

  • Local Institution - 0180

    Shatin, NT

Other trials in Hong Kong

Sweden2 sites

  • Local Institution - 0119

    Örebro, Örebro Län [se-18], 701 85

  • Local Institution - 0118

    Stockholm, Stockholms Län [se-01], 141 86

Other trials in Sweden

China1 site

  • Local Institution - 0156

    Wuhan, Hubei, 430030

Other trials in China

Czechia1 site

  • Local Institution - 0060

    Hradec Králové, 500 05

Other trials in Czechia

Poland1 site

  • Local Institution - 0097

    Olsztyn, Warmian-Masurian Voivodeship, 10-228

Other trials in Poland

About this study

Official title: 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).

Being tested
Oral Azacitidine, Placebo for Oral Azacitidine
Keywords
Anemia, Thrombocytopenia
Medical subject headings
Myelodysplastic Syndromes, Anemia, Thrombocytopenia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Oral AzacitidineDrug

    Also called BMS-986345, Oral-Aza, ONUREG®

    Specified dose on specified days

  • Placebo for Oral AzacitidineDrug

    Specified dose on specified days

Study groups in NCT05469737
Study groupRoleWhat they receive
Part I - Oral-Aza (Dose 1)ExperimentalNot described
Receives: Drug: Oral Azacitidine
Part I - Oral-Aza (Dose 2)ExperimentalNot described
Receives: Drug: Oral Azacitidine
Part II - Oral-Aza (RP3D)ExperimentalRP3D: Recommended Phase 3 Dose
Receives: Drug: Oral Azacitidine
Part II - PlaceboExperimentalNot described
Receives: Drug: Placebo for Oral Azacitidine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Phase 2

    Measured: 6 cycles plus 28 days (up to 24 weeks)

  2. Number of participants who achieved complete remission (CR) per International Working Group (IWG) 2006 criteria within 6 cycles

    Phase 2 and 3

    Measured: Up to 24 weeks

Secondary outcomes (20)

  1. Number of participants who achieved Overall Response (OR) per IWG 2006 criteria within 6 cycles

    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

    Measured: Up to 24 weeks

  2. Number of participants who achieved 84-day packed red blood cells transfusion independence (pRBC-TI)

    Phase 2 and Phase 3

    Measured: Up to 32 weeks

  3. pRBC-TI duration

    Phase 2 and Phase 3

    Measured: Over the course of the study, an average of 1 year

  4. Number of participants who achieve 84 day platelet transfusion independence (PLT-TI) within 6 cycles

    Phase 2 and Phase 3

    Measured: Over the course of the study, an average of 1 year

  5. PLT-TI duration

    Phase 2 and Phase 3

    Measured: Over the course of the study, an average of 1 year

  6. Number of participants who achieved pRBC transfusion reduction

    Phase 3

    Measured: Over the course of the study, an average of 1 year

  7. pRBC transfusion reduction duration

    Phase 3

    Measured: Over the course of the study, an average of 1 year

  8. CR duration

    Phase 2 and Phase 3

    Measured: Over the course of the study, an average of 1 year

  9. Best OR

    Phase 2 and Phase 3

    Measured: Over the course of the study, an average of 1 year

  10. OR duration

    Phase 2 and Phase 3

    Measured: Over the course of the study, an average of 1 year

  11. Overall Survival (OS)

    Phase 3

    Measured: Up to 5 years after discontinuation of Investigational Product, approximately 6 years

  12. Event-free Survival (EFS)

    Phase 3

    Measured: Up to 5 years after discontinuation of Investigational Product, approximately 6 years

  13. Time to acute myeloid leukemia (AML)

    Phase 3

    Measured: Up to 5 years after discontinuation of Investigational Product, approximately 6 years

  14. Time to subsequent therapy

    Phase 3

    Measured: Up to 5 years after discontinuation of Investigational Product, approximately 6 years

  15. Iron parameters measured from blood

    Phase 3

    Measured: Over the course of the study, an average of 1 year

  16. 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

    Phase 3

    Measured: Up to end of treatment/early termination, an average of 1 year

  17. Summary statistics for Functional Assessment of Cancer Therapy-Anemia (FACT-An) scales and subscales at each assessment point for each treatment arm

    Phase 3

    Measured: Up to end of treatment/early termination, an average of 1 year

  18. Summary statistics for Quality of Life in Myelodysplasia Scale (QUALMS) scales and subscales at each assessment point for each treatment arm

    Phase 3

    Measured: Up to end of treatment/early termination, an average of 1 year

  19. Summary statistics for the EuroQol 5 Dimension 5 Level (EQ-5D-5L) scales and subscales at each assessment point for each treatment arm

    Phase 3

    Measured: Up to end of treatment/early termination, an average of 1 year

  20. Number of participants with healthcare resource use associated with the investigational product (IP)

    Phase 3

    Measured: Over the course of the study, an average of 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Jul 2022
  2. Study start14 Dec 2022
  3. Primary completion31 Jul 2028 (estimated)
  4. Study completion31 Jul 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
230 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05469737
Sponsor's protocol ID
CA055-026
Other IDs
U1111-1276-5463
Status last verified
Feb 2026
First submitted
8 Jul 2022
Last updated
25 Feb 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: 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

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Oral Azacitidine in people with Myelodysplastic Syndromes, run by Bristol-Myers Squibb at 65 sites in 17 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 25 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 65 sites in United States, Japan, France, Germany, Spain and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05469737.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2026.

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