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)

NCT ID: NCT05469737

Last Updated: 2025-10-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

230 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-12-14

Study Completion Date

2026-07-29

Brief Summary

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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).

Detailed Description

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Conditions

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Myelodysplastic Syndromes

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Part I - Oral-Aza (Dose 1)

Group Type EXPERIMENTAL

Oral Azacitidine

Intervention Type DRUG

Specified dose on specified days

Part I - Oral-Aza (Dose 2)

Group Type EXPERIMENTAL

Oral Azacitidine

Intervention Type DRUG

Specified dose on specified days

Part II - Oral-Aza (RP3D)

RP3D: Recommended Phase 3 Dose

Group Type EXPERIMENTAL

Oral Azacitidine

Intervention Type DRUG

Specified dose on specified days

Part II - Placebo

Group Type EXPERIMENTAL

Placebo for Oral Azacitidine

Intervention Type DRUG

Specified dose on specified days

Interventions

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Oral Azacitidine

Specified dose on specified days

Intervention Type DRUG

Placebo for Oral Azacitidine

Specified dose on specified days

Intervention Type DRUG

Other Intervention Names

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BMS-986345 Oral-Aza ONUREG®

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Bristol-Myers Squibb

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Bristol-Myers Squibb

Role: STUDY_DIRECTOR

Bristol-Myers Squibb

Locations

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Local Institution - 0137

Miami, Florida, United States

Site Status

Local Institution - 0147

Tamarac, Florida, United States

Site Status

Local Institution - 0132

East Syracuse, New York, United States

Site Status

Local Institution - 0073

Pittsburgh, Pennsylvania, United States

Site Status

Local Institution - 0014

Houston, Texas, United States

Site Status

Local Institution - 0086

Houston, Texas, United States

Site Status

Local Institution - 0123

Fairfax, Virginia, United States

Site Status

Local Institution - 0070

Pilar, Buenos Aires, Argentina

Site Status

Local Institution - 0039

ABB, Buenos Aires F.D., Argentina

Site Status

Local Institution - 0016

Buenos Aires, , Argentina

Site Status

Local Institution - 0022

Buenos Aires, , Argentina

Site Status

Local Institution - 0050

Buenos Aires, , Argentina

Site Status

Local Institution - 0006

Clayton, Victoria, Australia

Site Status

Local Institution - 0018

Melbourne, Victoria, Australia

Site Status

Local Institution - 0004

Melbourne, Victoria, Australia

Site Status

Local Institution - 0003

Melbourne, , Australia

Site Status

Local Institution - 0008

Toronto, Ontario, Canada

Site Status

Local Institution - 0015

Toronto, Ontario, Canada

Site Status

Local Institution - 0090

Montreal, Quebec, Canada

Site Status

Local Institution - 0156

Wuhan, Hubei, China

Site Status

Local Institution - 0060

Hradec Králové, , Czechia

Site Status

Local Institution - 0115

Aarhus, Central Jutland, Denmark

Site Status

Local Institution - 0116

Aalborg, North Denmark, Denmark

Site Status

Local Institution - 0063

Pessac, Aquitaine, France

Site Status

Local Institution - 0024

Tours, Indre-et-Loire, France

Site Status

Local Institution - 0094

Angers, Maine-et-Loire, France

Site Status

Local Institution - 0056

Lille, Nord, France

Site Status

Local Institution - 0085

Villejuif, Val-de-Marne, France

Site Status

Local Institution - 0082

Paris, , France

Site Status

Local Institution - 0081

Duisburg, North Rhine-Westphalia, Germany

Site Status

Local Institution - 0128

Düsseldorf, North Rhine-Westphalia, Germany

Site Status

Local Institution - 0055

Leipzig, Saxony, Germany

Site Status

Local Institution - 0037

Dresden, , Germany

Site Status

Local Institution - 0007

Hamburg, , Germany

Site Status

Local Institution - 0028

Mutlangen, , Germany

Site Status

Local Institution - 0125

Chaïdári, Attikí, Greece

Site Status

Local Institution - 0129

Thessaloniki, Thessaloníki, Greece

Site Status

Local Institution - 0127

Alexandroupoli, , Greece

Site Status

Local Institution - 0178

Hksar, , Hong Kong

Site Status

Local Institution - 0180

Shatin, , Hong Kong

Site Status

Local Institution - 0061

Rome, Lazio, Italy

Site Status

Local Institution - 0052

Rozzano, Milano, Italy

Site Status

Local Institution - 0075

Florence, Tuscany, Italy

Site Status

Local Institution - 0101

Bologna, , Italy

Site Status

Local Institution - 0136

Kitakyushu-shi, Fukuoka, Japan

Site Status

Local Institution - 0154

Sapporo, Hokkaido, Japan

Site Status

Local Institution - 0153

Amagasaki, Hyōgo, Japan

Site Status

Local Institution - 0130

Sagamihara, Kanagawa, Japan

Site Status

Local Institution - 0135

Sendai, Miyagi, Japan

Site Status

Local Institution - 0150

Shinagawa-ku, Tokyo, Japan

Site Status

Local Institution - 0124

Osaka, , Japan

Site Status

Local Institution - 0097

Olsztyn, Warmian-Masurian Voivodeship, Poland

Site Status

Local Institution - 0058

Hwasun Gun, Jeonranamdo, South Korea

Site Status

Local Institution - 0048

Seoul, Seoul Teugbyeolsi, South Korea

Site Status

Local Institution - 0036

Seoul, Seoul-teukbyeolsi [Seoul], South Korea

Site Status

Local Institution - 0012

Seoul, Seoul-teukbyeolsi [Seoul], South Korea

Site Status

Local Institution - 0051

Junggu, Taegu-Kwangyǒkshi, South Korea

Site Status

Local Institution - 0109

Valencia, Valenciana, Comunitat, Spain

Site Status

Local Institution - 0107

Granada, , Spain

Site Status

Local Institution - 0111

Madrid, , Spain

Site Status

Local Institution - 0112

Ourense, , Spain

Site Status

Local Institution - 0110

Oviedo, , Spain

Site Status

Local Institution - 0108

Salamanca, , Spain

Site Status

Local Institution - 0118

Stockholm, Stockholms Län [se-01], Sweden

Site Status

Local Institution - 0119

Örebro, Örebro Län [se-18], Sweden

Site Status

Countries

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United States Argentina Australia Canada China Czechia Denmark France Germany Greece Hong Kong Italy Japan Poland South Korea Spain Sweden

Related Links

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Other Identifiers

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U1111-1276-5463

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

CA055-026

Identifier Type: -

Identifier Source: org_study_id

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