Acute Myeloid LeukemiaPhase 2NCT05197426
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
A Phase 2 study testing Oral Azacitidine in people with Acute Myeloid Leukemia, run by Bristol-Myers Squibb at 31 sites in 2 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 55 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Oral Azacitidine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 19 participants
- Where
- 31 sitesin 2 countries
- Study dates
- 17 Jan 2022to 30 Apr 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 55 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- ≥ 55 years of age inclusive at the time of signing the informed consent
- Newly diagnosed, histologically confirmed de novo Acute Myeloid Leukemia (AML) or AML secondary to prior myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML)
- Should have undergone induction therapy with intensive chemotherapy with or without consolidation therapy as recommended in appropriate guideline(s) or equivalent regimen according to institutional standard: having achieved first complete remission (CR)/complete remission with incomplete blood count recovery (CRi) status within 4 months prior to starting study therapy
Who cannot join
- Suspected or proven acute promyelocytic leukemia; or AML with previous hematologic disorder such as chronic myeloid leukemia or myeloproliferative neoplasms, excluding MDS and CMML
- Prior bone marrow or stem cell transplantation
- Received therapy with hypomethylating agents for MDS and went on to develop AML within four months of discontinuing the therapy with hypomethylating agents
- Have achieved CR/CRi following therapy with hypomethylating agents
- Other protocol-defined inclusion/exclusion criteria apply
Trial locations and contacts
31 sites in 2 countries.
31 sites in 2 countries
Japan30 sites
Local Institution - 0022
Aomori, Aomori, 0308553
Local Institution - 0032
Bunkyo Ku, Tokyo, 1138677
Local Institution - 0016
Fukuoka, Fukuoka, 810-8539
Local Institution - 0018
Fukuoka, 810-8563
Local Institution - 0027
Fukuoka, 8120033
Local Institution - 0012
Isehara, Kanagawa, 2591193
Local Institution - 0005
Kamogawa, Chiba, 2968602
Local Institution - 0019
Kanazawa, Ishikawa-ken, 9208641
Local Institution - 0003
Kashiwa-shi, Chiba, 2778577
Local Institution - 0001
Maebashi, Gunma, 3710821
Local Institution - 0010
Matsuyama, Ehime, 7900826
Local Institution - 0008
Nagasaki, 8528511
Local Institution - 0017
Nagoya, Aichi-ken, 453-8511
Local Institution - 0023
Nagoya, Aichi-ken, 4668560
Local Institution - 0014
Ōgaki, Gifu, 503-8502
Local Institution - 0025
Okayama, 7000914
Local Institution - 0026
Osaka, Osaka, 5300012
Local Institution - 0021
Ōsaka-sayama, Osaka, 5898511
Local Institution - 0006
Sagamihara-shi, Kanagawa, 2520375
Local Institution - 0028
Saitama, 330-8503
Local Institution - 0004
Sapporo, Hokkaido, 0640804
Local Institution - 0015
Sendai, Miyagi, 9808574
Local Institution - 0031
Shimotsuke, Tochigi, 329-0498
Local Institution - 0002
Shinagawa, Tokyo, 141-8625
Local Institution - 0030
Shinjuku-ku, Tokyo, 1600023
Local Institution - 0029
Sumida Ku, Tokyo, 1308575
Local Institution - 0009
Toyoake, Aichi-ken, 470-1192
Local Institution - 0007
Yamagata, 990-9585
Local Institution - 0013
Yokohama, Kanagawa, 236-0004
Local Institution - 0020
Yoshida Gun, Fukui, 9101193
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-controlled Study to Compare Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Japanese Subjects With Acute Myeloid Leukemia in Complete Remission
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.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Oral Azacitidine, Placebo
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Oral AzacitidineDrug
Also called CC-486, BMS-986345, Onureg
Specified dose on specified days
PlaceboOther
Specified dose of specified days
| Study group | Role | What they receive |
|---|---|---|
| Oral Azacitidine | Experimental | Not described Receives: Drug: Oral Azacitidine |
| Placebo | Placebo comparator | Not described Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recurrence Free Survival (RFS)
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.
Measured: Approximately 17.7 months
Secondary outcomes (17)
Overall Survival (OS)
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.
Measured: Approximately 17.7 months
Time to Relapse From CR or CRi
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.
Measured: Approximately 17.7 months
Time to Discontinuation
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.
Measured: Approximately 17.7 months
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Measured: Approximately 17.7 months
Number of Participants With Clinically Significant Changes in Physical Examination
Number of participants with clinically significant changes in physical examination
Measured: Approximately 17.7 months
Number of Participants With Clinically Significant Changes in Vital Signs
Vital signs include the following: systolic blood pressure, diastolic blood pressure, heart rate, respiratory rate, temperature and weight.
Measured: Approximately 17.7 months
Number of Participants With Clinically Significant Changes in Clinical Laboratory Examinations
Measured: Approximately 17.7 months
Number of Participants Who Received Concomitant Medication
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.
Measured: Approximately 17.7 months
Mean Change From Baseline in Facit-Fatigue Scale
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.
Measured: From C1D1 to C12D1 (approximately 336 days)
Mean Change From Baseline in EQ-5D-5L
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 on the individual responses to the 5 EQ-5D-5L domains ranging from -0.530 to 1.000. The smallest change considered clinically meaningful, is defined as a score difference of 0.08 points. The lower the score the better.
Measured: From C1D1 to C12D1 (approximately 336 days)
Pharmacokinetic Evaluation: CMax
Cmax is defined as maximum plasma concentration of the drug.
Measured: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: Tmax
Tmax is defined is the time to maximum plasma concentration
Measured: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: AUC(0-T)
Area under the plasma concentration time-curve. AUC from time 0 to the last time of quantifiable concentration.
Measured: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: AUC(INF)
Measured: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: T-Half
the time required for the amount or concentration of a drug to decrease by one-half
Measured: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: CLT/F(INF)
the volume of plasma from which a substance is completely removed per unit time.
Measured: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: Vz/F
the amount of drug in the body to the concentration of drug measured in a biological fluid
Measured: on C1D1 (after first dose on day 1)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2022
- Study start17 Jan 2022
- Primary completion27 Jan 2025
- Study completion30 Apr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Bristol-Myers SquibbThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bristol-Myers Squibb
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Bristol-Myers Squibb, Study director, Bristol-Myers Squibb
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05197426
- Sponsor's protocol ID
- CA055-005
- Status last verified
- Feb 2026
- First submitted
- 5 Jan 2022
- Last updated
- 11 Feb 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-Blind, Placebo-controlled Study to Compare Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Japanese Subjects With Acute Myeloid Leukemia in Complete Remission
- ClinicalTrials.gov ID
- NCT05197426
- Acronym / sponsor ID
- CA055-005
- Conditions
- Acute Myeloid Leukemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Bristol-Myers SquibbIndustry (pharma, biotech, devices)
- Study start
- 17 Jan 2022 (actual)
- Primary completion
- 27 Jan 2025 (actual)
- Study completion
- 30 Apr 2026 (estimated)
- First posted
- 19 Jan 2022 (actual)
- Results
- Posted 11 Feb 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Oral Azacitidine | Experimental | Drug: Oral Azacitidine |
| Placebo | Placebo comparator | Other: Placebo |
Who may qualify
- Ages
- 55 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- ≥ 55 years of age inclusive at the time of signing the informed consent
- Newly diagnosed, histologically confirmed de novo Acute Myeloid Leukemia (AML) or AML secondary to prior myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML)
- Should have undergone induction therapy with intensive chemotherapy with or without consolidation therapy as recommended in appropriate guideline(s) or equivalent regimen according to institutional standard: having achieved first complete remission (CR)/complete remission with incomplete blood count recovery (CRi) status within 4 months prior to starting study therapy
Key exclusion criteria (5 listed)
- Suspected or proven acute promyelocytic leukemia; or AML with previous hematologic disorder such as chronic myeloid leukemia or myeloproliferative neoplasms, excluding MDS and CMML
- Prior bone marrow or stem cell transplantation
- Received therapy with hypomethylating agents for MDS and went on to develop AML within four months of discontinuing the therapy with hypomethylating agents
- Have achieved CR/CRi following therapy with hypomethylating agents
- Other protocol-defined inclusion/exclusion criteria apply
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Oral AzacitidineAlso: CC-486, BMS-986345, OnuregSpecified dose on specified days | Drug | Oral Azacitidine |
| PlaceboSpecified dose of specified days | Other | Placebo |
Primary outcome
Recurrence Free Survival (RFS)
Time frame: 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.
17 secondary outcomes
Overall Survival (OS)
Time frame: Approximately 17.7 months
Time to Relapse From CR or CRi
Time frame: Approximately 17.7 months
Time to Discontinuation
Time frame: Approximately 17.7 months
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: Approximately 17.7 months
Number of Participants With Clinically Significant Changes in Physical Examination
Time frame: Approximately 17.7 months
Number of Participants With Clinically Significant Changes in Vital Signs
Time frame: Approximately 17.7 months
Number of Participants With Clinically Significant Changes in Clinical Laboratory Examinations
Time frame: Approximately 17.7 months
Number of Participants Who Received Concomitant Medication
Time frame: Approximately 17.7 months
Mean Change From Baseline in Facit-Fatigue Scale
Time frame: From C1D1 to C12D1 (approximately 336 days)
Mean Change From Baseline in EQ-5D-5L
Time frame: From C1D1 to C12D1 (approximately 336 days)
Pharmacokinetic Evaluation: CMax
Time frame: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: Tmax
Time frame: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: AUC(0-T)
Time frame: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: AUC(INF)
Time frame: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: T-Half
Time frame: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: CLT/F(INF)
Time frame: on C1D1 (after first dose on day 1)
Pharmacokinetic Evaluation: Vz/F
Time frame: on C1D1 (after first dose on day 1)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- BMS Clinical Trial InformationLink provided by the sponsor.
- BMS Clinical Trial Patient RecruitingLink provided by the sponsor.
Related trials for Acute Myeloid Leukemia
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingPhase 1/2
Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
Acute Biphenotypic Leukemia, Acute Myeloid Leukemia
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bristol-Myers Squibb study director
Bristol-Myers Squibb
31 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Oral Azacitidine in people with Acute Myeloid Leukemia, run by Bristol-Myers Squibb at 31 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 11 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in Japan, United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 11 Feb 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recurrence Free Survival (RFS) (time frame: Approximately 17.7 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Feb 2026.