An Efficacy and Safety Study of Oral Azacitidine (CC-486) as Maintenance Therapy in Chinese Participants With Acute Myeloid Leukemia in Complete Remission

A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Compare Efficacy and Safety of Oral Azacitidine (CC-486) Plus Best Supportive Care Versus Best Supportive Care as Maintenance Therapy in Chinese Patients With Acute Myeloid Leukemia in Complete Remission

The purpose of this study is to evaluate the efficacy and safety of Oral Azacitidine (CC-486) in Chinese participants with acute myeloid leukemia in complete remission.

Study Overview

• Status: Active, not recruiting
• Conditions: Leukemia, Myeloid, Acute
• Intervention / Treatment: Other: Placebo; Drug: CC-486

• Study Type: Interventional
• Enrollment (Actual): 34
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Changchun, China, 130021
    • Local Institution - 0017
  • Changsha, China, 410008
    • Local Institution - 0019
  • Changsha, China, 410013
    • Local Institution - 0015
  • Ganzhou, China, 341000
    • Local Institution - 0035
  • Guangzhou, China, 510060
    • Local Institution - 0012
  • Hangzhou, China, 310009
    • Local Institution - 0014
  • Harbin, China, 150086
    • Local Institution - 0032
  • Jinan, China, 250012
    • Local Institution - 0011
  • Kunming, China, 650032
    • Local Institution - 0024
  • Lanzhou, China, 730030
    • Local Institution - 0026
  • Nanchang, China, 330006
    • Local Institution - 0018
  • Nanjing, China, 210029
    • Local Institution - 0029
  • Suzhou, China, 215006
    • Local Institution - 0021
  • Tianjin, China, 300052
    • Local Institution - 0023
  • Zhangzhou, China, 363000
    • Local Institution - 0034
  • Zhengzhou, China, 450008
    • Local Institution - 0004
  • Anhui
    • Hefei, Anhui, China, 230001
      • Local Institution - 0031
  • BJ
    • Beijing, BJ, China, 100044
      • Local Institution - 0013
    • Beijing, BJ, China, 100191
      • Local Institution - 0027
  • CQ
    • Chongqing, CQ, China, 400000
      • Local Institution - 0028
  • GD
    • Guangzhou, GD, China, 510080
      • Local Institution - 0003
    • Guangzhou, GD, China, 510080
      • Local Institution - 0008
    • Shenzhen, GD, China, 518055
      • Local Institution - 0010
  • HE
    • Shijiazhuang, HE, China, 050000
      • Local Institution - 0002
  • Jiangsu
    • Xuzhou, Jiangsu, China, 221000
      • Local Institution - 0022
  • LN
    • Shenyang, LN, China, 110022
      • Local Institution - 0020
  • Liaoning
    • Shenyang, Liaoning, China, 110001
      • Local Institution - 0005
  • SC
    • Chengdu, SC, China, 610041
      • Local Institution - 0016
  • SH
    • Shanghai, SH, China, 200025
      • Local Institution - 0006
  • SN
    • Xi'an, SN, China, 710100
      • Local Institution - 0030
  • SX
    • Taiyuan, SX, China, 030013
      • Local Institution - 0033
  • TJ
    • Tianjin, TJ, China, 300020
      • Local Institution - 0001
  • Xinjiang
    • Ürümqi, Xinjiang, China, 830011
      • Local Institution - 0009
  • ZJ
    • Wenzhou, ZJ, China, 325015
      • Local Institution - 0007

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 55 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Newly diagnosed, histologically confirmed de novo acute myeloid leukemia (AML) or AML secondary to prior myelodysplastic disease or chronic myelomonocytic leukemia (CMML)
• Eastern cooperative oncology group performance status of 0, 1, or 2
• Has undergone induction therapy with intensive chemotherapy with or without consolidation therapy
• Must have achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) status within 6 months (+/- 7 days) prior to starting study therapy

Exclusion Criteria:

• Suspected or proven acute promyelocytic leukemia or acute myeloid leukemia with previous hematologic disorder such as chronic myeloid leukemia or myeloproliferative neoplasms, excluding myelodysplastic syndromes and chronic myelomonocytic leukemia
• Candidate for allogeneic bone marrow or stem cell transplant at screening
• Have achieved CR/CRi following therapy with hypomethylating agents
• AML associated with inv(16), t(8;21), t(16;16), t(15;17), or t(9;22) karyotypes or molecular evidence of such translocations
• Proven central nervous system leukemia
• Prior bone marrow or stem cell transplantation

Other protocol-defined inclusion/exclusion criteria apply

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo Administration	Other: Placebo; Specified dose on specified days
Experimental: CC-486/Oral Azacitidine Administration	Drug: CC-486; Specified dose on specified days; Other Names: Azacitidine; Onureg

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Relapse-free survival (RFS)	Up to 30 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival (OS)		Up to approximately 42 months
Time to relapse		Up to approximately 30 months
Time to discontinuation of treatment		Up to approximately 42 months
Number of participants with adverse events (AEs)		Up to approximately 42 months
Number of participants with physical examination abnormalities		Up to approximately 42 months
Number of participants with vital sign abnormalities		Up to approximately 42 months
Number of participants with clinical laboratory abnormalities		Up to approximately 42 months
Area under the concentration-time curve from time zero to the time of the last quantifiable concentration (AUC(0-t))		Up to 8 weeks
Maximum observed plasma concentration (Cmax)		Up to 8 weeks
Time of maximum observed concentration (Tmax)		Up to 8 weeks
Terminal elimination half-life (T1/2)		Up to 8 weeks
Minimal/measurable residual disease (MRD) assessment by flow cytometric analysis of hematopoietic cell immunophenotypes		Up to approximately 30 months
Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Scale		Up to approximately 30 months
EQ-5D-5L scale		Up to approximately 30 months
Visual analog scale (VAS)		Up to approximately 30 months
Healthcare Resource Utilization (HRU): Rate of Hospital Events Per Year	HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the participant. HRU is a key component to understand treatment costs and budget impact of new treatments from a provider perspective.	Up to approximately 30 months
Healthcare Resource Utilization (HRU): Number of Medications	HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the participant. HRU is a key component to understand treatment costs and budget impact of new treatments from a provider perspective.	Up to approximately 30 months
Healthcare Resource Utilization (HRU): Rate of Clinic Visits Per Year	HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the participant. HRU is a key component to understand treatment costs and budget impact of new treatments from a provider perspective.	Up to approximately 30 months
Healthcare Resource Utilization (HRU): Rate of Medical/Diagnostic Events Per Year	HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the participant. HRU is a key component to understand treatment costs and budget impact of new treatments from a provider perspective.	Up to approximately 30 months
Healthcare Resource Utilization (HRU): Number of Treatments for AEs Per Year	HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the participant. HRU is a key component to understand treatment costs and budget impact of new treatments from a provider perspective.	Up to approximately 30 months

Collaborators and Investigators

• Sponsor: Bristol-Myers Squibb
• Investigators: Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

Helpful Links

• BMS Clinical Trial Information

Study record dates

Study Major Dates

• Study Start (Actual): August 19, 2022
• Primary Completion (Actual): October 31, 2025
• Study Completion (Estimated): October 31, 2026

Study Registration Dates

• First Submitted: May 25, 2022
• First Submitted That Met QC Criteria: June 7, 2022
• First Posted (Actual): June 9, 2022

Study Record Updates

• Last Update Posted (Actual): April 28, 2026
• Last Update Submitted That Met QC Criteria: April 23, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Acute Myeloid Leukemia; Azacitidine; CC-486; Onureg
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Hematologic Diseases; Leukemia, Myeloid; Leukemia; Hemic and Lymphatic Diseases; Leukemia, Myeloid, Acute; Organic Chemicals; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleic Acids, Nucleotides, and Nucleosides; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Aza Compounds; Nucleosides; Ribonucleosides; Azacitidine; cc-486
• Other Study ID Numbers: CA055-006

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at https://www.bms.com/researchers-and-partners/clinical-trials-and-research.html
• IPD Sharing Time Frame: See Plan Description
• IPD Sharing Access Criteria: See Plan Description
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes