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)

Phase 3Active Not RecruitingNCT05469737

Bristol-Myers Squibb230 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

230

Conditions

Myelodysplastic Syndromes

Interventions

Oral AzacitidineDRUG

Specified dose on specified days

Placebo for Oral AzacitidineDRUG

Specified dose on specified days

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (65)

Outcomes

Primary Outcomes

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

Time frame: 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

Time frame: Up to 24 weeks

Secondary Outcomes

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

Time frame: Up to 24 weeks

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

Phase 2 and Phase 3

Time frame: Up to 32 weeks

pRBC-TI duration

Phase 2 and Phase 3

Time frame: 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

Phase 2 and Phase 3

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

PLT-TI duration

Phase 2 and Phase 3

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

Data from ClinicalTrials.gov

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Local Institution - 0137

Miami, Florida, United States

Local Institution - 0147

Tamarac, Florida, United States

Local Institution - 0132

East Syracuse, New York, United States

Local Institution - 0073

Pittsburgh, Pennsylvania, United States

Local Institution - 0014

Houston, Texas, United States

Local Institution - 0086

Houston, Texas, United States

Local Institution - 0123

Fairfax, Virginia, United States

Local Institution - 0070

Pilar, Buenos Aires, Argentina

Local Institution - 0039

ABB, Buenos Aires F.D., Argentina

Local Institution - 0016

Buenos Aires, Argentina

...and 55 more locations

Number of participants who achieved pRBC transfusion reduction

Phase 3

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

pRBC transfusion reduction duration

Phase 3

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

CR duration

Phase 2 and Phase 3

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

Best OR

Phase 2 and Phase 3

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

OR duration

Phase 2 and Phase 3

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

Overall Survival (OS)

Phase 3

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

Event-free Survival (EFS)

Phase 3

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

Time to acute myeloid leukemia (AML)

Phase 3

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

Time to subsequent therapy

Phase 3

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

Iron parameters measured from blood

Phase 3

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

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

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

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

Phase 3

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

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

Phase 3

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

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

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

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

Phase 3

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