NCT05197426

A Study to Compare the Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Japanese Participants With Acute Myeloid Leukemia (AML) in Complete Remission

Active, enrolled Phase 2 Results posted Last updated 11 February 2026

What this trial tests

Phase 2 trial testing Oral Azacitidine in Acute Myeloid Leukemia in 19 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline

17 January 2022

Primary endpoint
27 January 2025

30 April 2026

Quick facts

Lead sponsor	Bristol-Myers Squibb
Phase	Phase 2
Status	Active, enrolled
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	19
Start date	17 January 2022
Primary completion	27 January 2025
Estimated completion	30 April 2026
Sites	31 locations across Japan, United States

Drugs / interventions tested

• Oral Azacitidine
• Placebo

Conditions studied

• Acute Myeloid Leukemia — all drugs for Acute Myeloid Leukemia →

Sponsor

Bristol-Myers Squibb — full company profile →

Who can join

55 and older, any sex, with Acute Myeloid Leukemia. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Recurrence Free Survival (RFS) Primary · Approximately 17.7 months

The time from randomization to the date of documented relapse after CR or CRi per central review, or death from any cause, whichever occurs first. Participants who are still alive without documented relapse after CR or CRi, or who were lost to follow-up without documented relapse, will be censored at the date of their last response assessment.

Group	Value	95% CI
Treatment 1	NA	10.2 – NA
Treatment 2	7.4	1.4 – NA

Overall Survival (OS) Secondary · Approximately 17.7 months

The time from randomization to death from any cause and will be calculated using the randomization date and date of death, or date of last follow-up for censored participants. All participants will be followed until dropout, death, or study termination. Participants who dropout or are alive at study termination will have their OS times censored at the time of last contact, as appropriate.

Group	Value	95% CI
Treatment 1	NA	18.1 – NA
Treatment 2	15.1	12.3 – NA

Time to Relapse From CR or CRi Secondary · Approximately 17.7 months

The interval from the date of randomization to the date of documented relapse after CR or CRi, as defined according to the IWG AML response criteria. Time to relapse will be analyzed using a competing risk analysis where death without documented relapse is treated as a competing risk for relapse from CR/CRi. Similar censoring rules as in primary analysis of RFS will be applied.

Group	Value	95% CI
Treatment 1	NA	10.2 – NA
Treatment 2	7.4	1.4 – NA

Time to Discontinuation Secondary · Approximately 17.7 months

The interval from the date of randomization to the date of discontinuation from IP. Subjects who are ongoing in treatment at the time of study closure will be censored at the date of last visit.

Group	Value	95% CI
Treatment 1	NA	6.7 – NA
Treatment 2	5.8	1.8 – 10.4

Number of Participants With Treatment Emergent Adverse Events (TEAEs) Secondary · Approximately 17.7 months

at least 1 TEAE

Group	Value	95% CI
Treatment 1	12
Treatment 2	7

TEAE related to study drug

Group	Value	95% CI
Treatment 1	12
Treatment 2	3

at least 1 Serious TEAE

Group	Value	95% CI
Treatment 1	4
Treatment 2	2

Serious TEAE releated to study drug

Group	Value	95% CI
Treatment 1	3
Treatment 2	1

Grade 3 or 4 TEAE

Group	Value	95% CI
Treatment 1	9
Treatment 2	4

Grade 3 or 4 TEAE related to study drug

Group	Value	95% CI
Treatment 1	8
Treatment 2	1

Number of Participants With Clinically Significant Changes in Physical Examination Secondary · Approximately 17.7 months

Number of participants with clinically significant changes in physical examination

Group	Value	95% CI
Treatment 1	0
Treatment 2	0

Number of Participants With Clinically Significant Changes in Vital Signs Secondary · Approximately 17.7 months

Vital signs include the following: systolic blood pressure, diastolic blood pressure, heart rate, respiratory rate, temperature and weight.

Group	Value	95% CI
Treatment 1	0
Treatment 2	0

Number of Participants With Clinically Significant Changes in Clinical Laboratory Examinations Secondary · Approximately 17.7 months

Group	Value	95% CI
Treatment 1	0
Treatment 2	0

Number of Participants Who Received Concomitant Medication Secondary · Approximately 17.7 months

Concomitant medications are defined as non-study medications that are started after the date of randomization but before the end of the study treatment period or started on or before the date of randomization but ended or remain ongoing during the study treatment period.

Group	Value	95% CI
Treatment 1	12
Treatment 2	7

Mean Change From Baseline in Facit-Fatigue Scale Secondary · From C1D1 to C12D1 (approximately 336 days)

The FACIT-Fatigue Scale is a short, 13-item, easy-to-administer tool that measures an individual's level of fatigue during usual daily activities over the past week. The level of fatigue is measured on a five-point Likert scale (0 = not at all fatigued to 4 = very much fatigued). It has scores that range from 0 to 52, with higher scores indicating less fatigue. Quality of life (QoL)scores on these FACIT scales significantly decline as patient performance status worsens.

Cycle 2 Day 1

Group	Value	95% CI
Treatment 1	3.2	± 6.12
Treatment 2	2.3	± 3.90

Cycle 6 Day 1

Group	Value	95% CI
Treatment 1	1.0	± 6.94
Treatment 2	-3.0	± 8.52

Cycle 12 Day 1

Group	Value	95% CI
Treatment 1	3.9	± 4.94
Treatment 2	-1.0	± 1.41

Mean Change From Baseline in EQ-5D-5L Secondary · From C1D1 to C12D1 (approximately 336 days)

The European Quality of Life 5D-5L Scale (EQ-5D-5L) assesses general health-related quality of life. Health is defined in 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems, and extreme problems. Responses are coded so that a '1' indicates no problem, and '5' indicates the most serious problem. The responses for the 5 dimensions are combined in a 5-digit number. The EQ-5D-5L health utility index (HUI) is assessed using the Crosswalk algorithm for France based

Cycle 2 Day 1

Group	Value	95% CI
Treatment 1	0.0267	± 0.12963
Treatment 2	-0.0106	± 0.02797

Cycle 6 Day 1

Group	Value	95% CI
Treatment 1	0.0413	± 0.11735
Treatment 2	-0.0485	± 0.23776

Cycle 12 Day 1

Group	Value	95% CI
Treatment 1	0.0254	± 0.08160
Treatment 2	-0.0855	± 0.12092

Pharmacokinetic Evaluation: CMax Secondary · on C1D1 (after first dose on day 1)

Cmax is defined as maximum plasma concentration of the drug.

Group	Value	95% CI
Treatment 1	155	± NA

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse Events and Serious Adverse Events: (From first dose to last dose + 28 days): Approximately 18.7 months. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Treatment 1

Serious: 4/12 (33%)

Deaths: 1/12

Treatment 2

Serious: 2/7 (29%)

Deaths: 2/7

Serious adverse events (7 terms)

Reaction	System	Treatment 1	Treatment 2
Febrile neutropenia	Blood and lymphatic system disorders	—	—
Vertigo	Ear and labyrinth disorders	—	—
Anal fistula	Gastrointestinal disorders	—	—
COVID-19	Infections and infestations	—	—
Pneumonia	Infections and infestations	—	—
Pyelonephritis	Infections and infestations	—	—
Acute myeloid leukaemia recurrent	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—

Other adverse events (82 terms — click to expand)

Reaction	System	Treatment 1	Treatment 2
Diarrhoea	Gastrointestinal disorders	—	—
Vomiting	Gastrointestinal disorders	—	—
Neutropenia	Blood and lymphatic system disorders	—	—
Nausea	Gastrointestinal disorders	—	—
COVID-19	Infections and infestations	—	—
Anaemia	Blood and lymphatic system disorders	—	—
Febrile neutropenia	Blood and lymphatic system disorders	—	—
Nasopharyngitis	Infections and infestations	—	—
Decreased appetite	Metabolism and nutrition disorders	—	—
Arthralgia	Musculoskeletal and connective tissue disorders	—	—
Leukopenia	Blood and lymphatic system disorders	—	—
Thrombocytopenia	Blood and lymphatic system disorders	—	—
Constipation	Gastrointestinal disorders	—	—
Dental caries	Gastrointestinal disorders	—	—
Malaise	General disorders	—	—
Hepatic function abnormal	Hepatobiliary disorders	—	—
Contusion	Injury, poisoning and procedural complications	—	—
Platelet count decreased	Investigations	—	—
Hypokalaemia	Metabolism and nutrition disorders	—	—
Acute myeloid leukaemia	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—
Headache	Nervous system disorders	—	—
Insomnia	Psychiatric disorders	—	—
Disseminated intravascular coagulation	Blood and lymphatic system disorders	—	—
Hypoglobulinaemia	Blood and lymphatic system disorders	—	—
Cardiac failure	Cardiac disorders	—	—
Tachycardia	Cardiac disorders	—	—
Ear pain	Ear and labyrinth disorders	—	—
Tinnitus	Ear and labyrinth disorders	—	—
Vertigo	Ear and labyrinth disorders	—	—
Hypothyroidism	Endocrine disorders	—	—
Cheilitis	Gastrointestinal disorders	—	—
Gastric ulcer	Gastrointestinal disorders	—	—
Gastritis	Gastrointestinal disorders	—	—
Gastrooesophageal reflux disease	Gastrointestinal disorders	—	—
Stomatitis	Gastrointestinal disorders	—	—
Trichoglossia	Gastrointestinal disorders	—	—
Chest pain	General disorders	—	—
Injection site erythema	General disorders	—	—
Injection site reaction	General disorders	—	—
Cystitis	Infections and infestations	—	—

Most-reported serious reactions: Febrile neutropenia, Vertigo, Anal fistula, COVID-19, Pneumonia, Pyelonephritis, Acute myeloid leukaemia recurrent.

Data from ClinicalTrials.gov NCT05197426 adverse events section.

Sponsor's own description

The purpose of this study is to assess the efficacy and safety of oral azacitidine plus best supportive care versus best supportive care as maintenance therapy in a cohort of Japanese participants ≥ 55 years of age with Acute Myeloid Leukemia (AML) and in complete remission/complete remission with incomplete blood count recovery after conventional induction chemotherapy with or without consolidation chemotherapy.

Publications & conference data

2 peer-reviewed publications reference this trial (live from Europe PMC):

1. Optimal Post-Remission Consolidation Therapy in Patients with AML.
   Jimenez-Chillon C, Dillon R, Russell N. · · 2024 · cited 5× · PMID 38008085 · DOI 10.1159/000535457
2. Clinical perspectives on post-induction maintenance therapy in patients with acute myeloid leukaemia in remission who are ineligible for allogeneic haematopoietic stem cell transplantation.
   Sweet K, Cluzeau T. · · 2025 · cited 3× · PMID 39622271 · DOI 10.1111/bjh.19924