NCT01757535

Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission

A Phase 3, 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 Subjects With Acute Myeloid Leukemia in Complete Remission

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 472 (actual)
• Sponsor: Celgene · Industry
• Sex: All
• Age: 55 Years
• Healthy volunteers: Not accepted

Summary

This study enrolled 472 participants, aged 55 or older, with a diagnosis of de novo acute myeloid leukemia (AML) or AML secondary to prior myelodysplastic disease or chronic myelomonocytic leukemia (CMML), and who have achieved first complete remission (CR)/ complete remission with incomplete blood count recovery (CRi) following induction with or without consolidation chemotherapy. The study is amended to include an extension phase (EP). The EP allows participants who are currently receiving oral azacitidine and who are demonstrating clinical benefit as assessed by the investigator, to continue receiving oral azacitidine after unblinding by sponsor until the participant meets the criteria for study discontinuation or until oral azacitidine becomes commercially available and reimbursed. In addition, all participants in the placebo arm and participants who had been discontinued from the treatment phase (irrespective of randomization arm) and continuing in the follow-up phase will be followed for survival in the EP.

Detailed description

This is an international, multicenter, placebo-controlled, Phase 3 study with a double-blind, randomized, parallel-group design in subjects with de novo AML or AML secondary to prior diagnosis of myelodysplastic syndromes (MDS) or chronic myelomonocytic leukemia (CMML) aged ≥ 55 years, who are in first CR/CRi following induction therapy with or without consolidation chemotherapy. The study consists of 3 phases; the pre-randomization phase (screening phase), the treatment phase, and the follow-up phase. The study is amended to include an extension phase (EP). The EP allows participants who are currently receiving oral azacitidine and who are demonstrating clinical benefit as assessed by the Investigator, to continue receiving oral azacitidine after unblinding by sponsor until they meet the criteria for study discontinuation or until oral azacitidine becomes commercially available and reimbursed. In addition, all participants in the placebo arm and participants who had been discontinued from the treatment phase (irrespective of randomization arm) and continuing in the follow-up phase will be followed for survival in the EP.

Conditions

• Leukemia, Myeloid, Acute

Interventions

Type	Name	Description
DRUG	Oral Azacitidine	300 mg oral azacitidine on days 1 to 14 of each 28-day treatment cycle.
DRUG	Placebo	Identically matching placebo tablets on days 1 to 14 of each 28-day treatment cycle.

Timeline

• Start date: 2013-04-24
• Primary completion: 2019-07-15
• Completion: 2024-06-18
• First posted: 2012-12-31
• Last updated: 2025-07-08
• Results posted: 2020-11-06

Locations

223 sites across 23 countries: United States, Australia, Austria, Belgium, Brazil, Canada, Czechia, Finland, France, Germany, Ireland, Israel, Italy, Lithuania, Mexico, Poland, Portugal, Russia, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom