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)

Bristol-Myers Squibb (BMS)

Status and phase: Active, not recruiting; Phase 3; Phase 2

Conditions: Myelodysplastic Syndromes

Treatments: Drug: Oral Azacitidine; Drug: Placebo for Oral Azacitidine

Study type: Interventional

Funder types: Industry

Identifiers

NCT05469737

CA055-026

U1111-1276-5463 (Other Grant/Funding Number)

Details and patient eligibility

About

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).

Enrollment: 230 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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230 participants in 4 patient groups

Part I - Oral-Aza (Dose 1)

Experimental group

Treatment:

Drug: Oral Azacitidine

Part I - Oral-Aza (Dose 2)

Experimental group

Treatment:

Drug: Oral Azacitidine

Part II - Oral-Aza (RP3D)

Experimental group

Description:

RP3D: Recommended Phase 3 Dose

Treatment:

Drug: Oral Azacitidine

Part II - Placebo

Experimental group

Treatment:

Drug: Placebo for Oral Azacitidine