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NCT Number: NCT05469737

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)

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).

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Myelodysplastic Syndromes Anemia Blood Platelet Disorders Bone Marrow Diseases Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Thrombocytopenia

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Local Institution - 0070, Pilar, Buenos Aires, Argentina

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Oral Azacitidine

Drug

Specified dose on specified days

Other names: BMS-986345, Oral-Aza, ONUREG®

Placebo for Oral Azacitidine

Drug

Specified dose on specified days

Primary outcomes

  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

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

    Phase 2

  2. 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

Secondary outcomes

  1. 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

  2. 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

  3. pRBC-TI duration

    Time frame: Over the course of the study, an average of 1 year

    Phase 2 and Phase 3

  4. 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

  5. PLT-TI duration

    Time frame: Over the course of the study, an average of 1 year

    Phase 2 and Phase 3

  6. Number of participants who achieved pRBC transfusion reduction

    Time frame: Over the course of the study, an average of 1 year

    Phase 3

  7. pRBC transfusion reduction duration

    Time frame: Over the course of the study, an average of 1 year

    Phase 3

  8. CR duration

    Time frame: Over the course of the study, an average of 1 year

    Phase 2 and Phase 3

  9. Best OR

    Time frame: Over the course of the study, an average of 1 year

    Phase 2 and Phase 3

  10. OR duration

    Time frame: Over the course of the study, an average of 1 year

    Phase 2 and Phase 3

  11. Overall Survival (OS)

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

    Phase 3

  12. Event-free Survival (EFS)

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

    Phase 3

  13. Time to acute myeloid leukemia (AML)

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

    Phase 3

  14. Time to subsequent therapy

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

    Phase 3

  15. Iron parameters measured from blood

    Time frame: Over the course of the study, an average of 1 year

    Phase 3

  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

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

    Phase 3

  17. 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

  18. 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

  19. 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

  20. 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

Sponsors and collaborators

Lead sponsor

Bristol-Myers Squibb

Industry

Registry information

Official study 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)

Important dates

Study start
2022
Primary completion
2028
Study completion
2028
First posted
Jul 22, 2022
Registry last updated
Feb 25, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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