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)

NCT05469737 | Active Not Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: CA055-026U1111-1276-5463
• Study Type: interventional
• Target: 230
• Locations: 17 countries
• Sites: 65

Brief 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).

Conditions

Myelodysplastic Syndromes

Keywords

Anemia; Thrombocytopenia

Outcome Measures

Primary Outcomes (2)

• 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

  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

  Phase 2 and 3

  Up to 24 weeks

Secondary Outcomes (20)

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

  Up to 24 weeks

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

  Up to 32 weeks

• pRBC-TI duration

  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

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

• PLT-TI duration

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

Study Arms (4)

Part I - Oral-Aza (Dose 1)

EXPERIMENTAL

Drug: Oral Azacitidine

Part I - Oral-Aza (Dose 2)

EXPERIMENTAL

Drug: Oral Azacitidine

Part II - Oral-Aza (RP3D)

EXPERIMENTAL

RP3D: Recommended Phase 3 Dose

Drug: Oral Azacitidine

Part II - Placebo

EXPERIMENTAL

Drug: Placebo for Oral Azacitidine

Interventions

Oral Azacitidine DRUG

Specified dose on specified days

Also known as: BMS-986345, Oral-Aza, ONUREG®

Part I - Oral-Aza (Dose 1); Part I - Oral-Aza (Dose 2); Part II - Oral-Aza (RP3D)

Placebo for Oral Azacitidine DRUG

Specified dose on specified days

Part II - Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (65)

Local Institution - 0137

Miami, Florida, 33136, United States

Location

Local Institution - 0147

Tamarac, Florida, 33321, United States

Location

Local Institution - 0132

East Syracuse, New York, 13057, United States

Location

Local Institution - 0073

Pittsburgh, Pennsylvania, 15224, United States

Location

Local Institution - 0014

Houston, Texas, 77030, United States

Location

Local Institution - 0086

Houston, Texas, 77030, United States

Location

Local Institution - 0123

Fairfax, Virginia, 22031, United States

Location

Local Institution - 0070

Pilar, Buenos Aires, 1629, Argentina

Location

Local Institution - 0039

ABB, Buenos Aires F.D., C1199ABB, Argentina

Location

Local Institution - 0016

Buenos Aires, 1425, Argentina

Location

Local Institution - 0022

Buenos Aires, 1431, Argentina

Location

Local Institution - 0050

Buenos Aires, CP1280AEB, Argentina

Location

Local Institution - 0006

Clayton, Victoria, 3168, Australia

Location

Local Institution - 0018

Melbourne, Victoria, 3000, Australia

Location

Local Institution - 0004

Melbourne, Victoria, 3065, Australia

Location

Local Institution - 0003

Melbourne, 3004, Australia

Location

Local Institution - 0008

Toronto, Ontario, M4N 3M5, Canada

Location

Local Institution - 0015

Toronto, Ontario, M5G 2M9, Canada

Location

Local Institution - 0090

Montreal, Quebec, H4A 3J1, Canada

Location

Local Institution - 0156

Wuhan, Hubei, 430030, China

Location

Local Institution - 0060

Hradec Králové, 500 05, Czechia

Location

Local Institution - 0115

Aarhus, Central Jutland, 8200, Denmark

Location

Local Institution - 0116

Aalborg, North Denmark, 9000, Denmark

Location

Local Institution - 0063

Pessac, Aquitaine, 33600, France

Location

Local Institution - 0024

Tours, Indre-et-Loire, 37032, France

Location

Local Institution - 0094

Angers, Maine-et-Loire, 49933, France

Location

Local Institution - 0056

Lille, Nord, 59000, France

Location

Local Institution - 0085

Villejuif, Val-de-Marne, 94805, France

Location

Local Institution - 0082

Paris, 75010, France

Location

Local Institution - 0081

Duisburg, North Rhine-Westphalia, 47166, Germany

Location

Local Institution - 0128

Düsseldorf, North Rhine-Westphalia, 40479, Germany

Location

Local Institution - 0055

Leipzig, Saxony, 04103, Germany

Location

Local Institution - 0037

Dresden, 01307, Germany

Location

Local Institution - 0007

Hamburg, 22081, Germany

Location

Local Institution - 0028

Mutlangen, 73557, Germany

Location

Local Institution - 0125

Chaïdári, Attikí, 12462, Greece

Location

Local Institution - 0129

Thessaloniki, Thessaloníki, 570 10, Greece

Location

Local Institution - 0127

Alexandroupoli, 08100, Greece

Location

Local Institution - 0178

Hksar, 0, Hong Kong

Location

Local Institution - 0180

Shatin, NT, Hong Kong

Location

Local Institution - 0061

Rome, Lazio, 00133, Italy

Location

Local Institution - 0052

Rozzano, Milano, 20089, Italy

Location

Local Institution - 0075

Florence, Tuscany, 50134, Italy

Location

Local Institution - 0101

Bologna, 40138, Italy

Location

Local Institution - 0136

Kitakyushu-shi, Fukuoka, 8068501, Japan

Location

Local Institution - 0154

Sapporo, Hokkaido, 064-0804, Japan

Location

Local Institution - 0153

Amagasaki, Hyōgo, 660-8550, Japan

Location

Local Institution - 0130

Sagamihara, Kanagawa, 252-0375, Japan

Location

Local Institution - 0135

Sendai, Miyagi, 980-8574, Japan

Location

Local Institution - 0150

Shinagawa-ku, Tokyo, 141-8625, Japan

Location

Local Institution - 0124

Osaka, 545-8586, Japan

Location

Local Institution - 0097

Olsztyn, Warmian-Masurian Voivodeship, 10-228, Poland

Location

Local Institution - 0058

Hwasun Gun, Jeonranamdo, 58128, South Korea

Location

Local Institution - 0048

Seoul, Seoul Teugbyeolsi, 06591, South Korea

Location

Local Institution - 0036

Seoul, Seoul-teukbyeolsi [Seoul], 05505, South Korea

Location

Local Institution - 0012

Seoul, Seoul-teukbyeolsi [Seoul], 06351, South Korea

Location

Local Institution - 0051

Junggu, Taegu-Kwangyǒkshi, 41944, South Korea

Location

Local Institution - 0109

Valencia, Valenciana, Comunitat, 46010, Spain

Location

Local Institution - 0107

Granada, 18012, Spain

Location

Local Institution - 0111

Madrid, 28006, Spain

Location

Local Institution - 0112

Ourense, 32005, Spain

Location

Local Institution - 0110

Oviedo, 33011, Spain

Location

Local Institution - 0108

Salamanca, 37007, Spain

Location

Local Institution - 0118

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

Location

Local Institution - 0119

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

Location

Related Links

• BMS Clinical Trial Information
• BMS Clinical Trial Patient Recruiting

MeSH Terms

Conditions

Myelodysplastic Syndromes; Anemia; Thrombocytopenia

Interventions

Azacitidine

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet Disorders; Cytopenia

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 8, 2022
• First Posted: July 22, 2022
• Study Start: December 14, 2022
• Primary Completion (Estimated): July 31, 2028
• Study Completion (Estimated): July 31, 2028
• Last Updated: February 25, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: See Plan Description
• Access Criteria: See Plan Description

Locations

CA (3); JP (7); AU (4); ES (6); DE (6); FR (6); SE (2); KR (5); GR (3); IT (4); CN (1); DK (2); US (7); PL (1); AR (5); HK (2); CZ (1)