A Study to Evaluate Long-term Safety of CC-486 (Oral Azacitidine) in Subjects With Hematological Disorders

NCT02494258 | Completed Phase 2 Results

• 2 other identifiers: CC-486-GEN-0012023-503272-25
• Study Type: interventional
• Target: 5
• Locations: 2 countries
• Sites: 4

Brief Summary

Rollover study supporting hematological disorder indications from Celgene sponsored CC-486 (oral azacitidine) protocols eligible for participation in the study.

Conditions

Hematologic Neoplasm; Neoplasms; Hematologic Malignancies

Keywords

CC-486; Oral Azacitidine; hematological disorders; rollover study; open label; Onureg

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment Emergent Adverse Events

  A treatment emergent adverse event is any untoward medical occurrence that begins or worsens after the first dose of study treatment, including any unfavorable sign, symptom, disease, or abnormal lab finding, whether or not related to the product, and may include worsening of pre-existing conditions. A Serious Adverse Event (SAE) is any untoward medical occurrence that results in death, is life-threatening, requires or prolongs hospitalization, causes persistent or significant disability/incapacity, leads to a congenital anomaly/birth defect, or is considered an important medical event requiring intervention to prevent these outcomes.

  From informed consent signed (Day 1) and until 28 days after the last dose of CC-486, or until the treatment discontinuation visit, whichever was later (up to approximately 90 months)

Secondary Outcomes (1)

• Number of Participants Who Survived

  From informed consent signed (Day 1) and date of death or last known documented date alive (up to approximately 90 months)

Study Arms (1)

Oral Azacitidine (CC-486)

EXPERIMENTAL

This study is an open-label, single-arm study and is divided into the screening period, treatment period and follow-up period. It is intended to evaluate the long-term safety of CC-486 and is to be taken at the same dose, schedule and frequency used from the last dose of CC-486 given in the parent study.

Drug: CC-486

Interventions

CC-486 DRUG

The subject will continue at the same dose, frequency and schedule used for the last dose of CC-486 given in the preceding CC-486 study. Subjects are allowed to rollover after the last cycle is finished and before the new cycle begins in the parent protocol. There is a 7 day window from End of Study on the parent protocol and when the participant will start Day 1 of the rollover study.

Also known as: Oral Azacitidine, BMS-986345, Onureg

Oral Azacitidine (CC-486)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Previously participated in, and received oral azacitidine, and continues to fulfill the eligibility criteria in one of the parent oral azacitidine clinical trials.
• The Investigator believes the subject is tolerating treatment with oral azacitidine monotherapy and continued oral azacitidine treatment is of benefit to the subject.
• Understand and voluntarily sign an informed consent document prior to any study related assessments or procedures being conducted.
• Willing and able to adhere to the study visit schedule and other protocol requirements.
• Females of childbearing potential (FCBP) may participate, provided that the participant meets the following conditions:
• Have two negative pregnancy tests as verified by the Investigator prior to starting study treatment. She must agree to ongoing pregnancy testing during the course of the study, and after end of study treatment. This applies even if the subject practices true abstinence from heterosexual contact.
• Either commit to true abstinence from heterosexual contact (which must be reviewed on a monthly basis and source documented) or agree to use, and be able to comply with highly effective contraception without interruption, during screening, during the study treatment (including dose interruptions), and for 6 months after discontinuation of study treatment, or longer if required for each compound and/or by local regulations.
• Subjects must satisfy the following criteria to participate in the Survival Follow-up phase:
• In order to be enrolled for the survival follow-up in the Follow-up Phase of the rollover study, subjects must have been in a parent oral azacitidine study where monitoring for survival was required and have signed informed consent for follow-up phase.
• Understand and voluntarily sign an informed consent document for this study.
• Willing and able to adhere to the study visit schedule and other protocol requirements.

You may not qualify if:

• The presence of any of the following will exclude a subject from receiving investigational product in the study:
• Concomitant use of drugs that are prohibited.
• Prior chemotherapy (including injectable azacitidine) or radiotherapy or any investigational agent after the last dose of oral azacitidine administered as part of the parent oral azacitidine study.
• Subjects have met one or more criteria for discontinuation as stipulated in the parent oral azacitidine study.
• Subjects received oral azacitidine in combination with another compound during a parent oral azacitidine study (Subjects form multi-arm parent oral azacitidine studies will be allowed to enroll into the rollover study, if the subject is receiving single-agent oral azacitidine at the time of transition into the rollover study).
• A subject's transition into rollover study ≥ 45 days after End of the Study visit of the parent oral azacitidine study
• Pregnant or lactating females.

Sponsors & Collaborators

• Celgene: lead

Study Sites (4)

Local Institution - 101

Gainesville, Florida, 32610-0277, United States

Location

Local Institution - 103

Baltimore, Maryland, 21231, United States

Location

Local Institution - 102

Houston, Texas, 77030, United States

Location

Manchester Royal Infirmary

Manchester, M13 9WL, United Kingdom

Location

Related Links

• BMS Clinical Trial Information
• BMS Clinical Trial Patient Recruiting

MeSH Terms

Conditions

Hematologic Neoplasms; Neoplasms; Hematologic Diseases

Interventions

cc-486; Azacitidine

Condition Hierarchy (Ancestors)

Neoplasms by Site; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Results Point of Contact

• Title: Bristol-Myers Squibb Study Director
• Organization: Bristol-Myers Squibb

Clinical.Trials@bms.com

Please email

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: July 8, 2015
• First Posted: July 10, 2015
• Study Start: October 22, 2015
• Primary Completion: April 14, 2025
• Study Completion: April 14, 2025
• Last Updated: April 2, 2026
• Results First Posted: April 2, 2026

Record last verified: 2026-03

Locations

US (3); GB (1)