NCT06180863

Oral Azacitidine (CC-486) Epigenetic Priming and Maintenance for Adult Acute Myeloid Leukemia (AML) Patients

A Pilot Study of Oral Azacitidine (CC-486) Epigenetic Priming and Maintenance in Adult AML Patients Eligible for Reduced Intensity Allogeneic Stem Cell Transplant

• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Yale University · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

To investigate the feasibility of delivering oral azacitidine (CC-486) as a consolidation regimen from the time of first complete remission (CR1), in patients with acute myelogenous leukemia (AML) eligible for curative intent Allogeneic Stem Cell Transplant (ASCT).

Detailed description

A significant proportion of patients with Acute myeloid leukemia (AML) who are fit to receive intensive chemotherapy and reach first complete remission (CR), relapse despite further high or low intensity therapies. Relapse/refractory (R/R) AML generally portends poor outcomes and available agents to treat this condition have modest efficacy. Until the recent approval of CC-486 as a maintenance therapy, no other drug had shown overall survival (OS) advantage for patients with AML in first complete remission (CR1). In the CC-486 registration trial (QUAZAR AML-01), remission induction was achieved by intensive chemotherapy, but patients were ineligible to proceed to allogeneic stem cell transplant (ASCT) at the time of screening. Post CR1, CC-486 apart from prolonging remission was also successful in deepening the quality of remission by eradicating measurable residual disease (MRD). It is therefore logical to investigate the feasibility of CC-486 as a consolidation regimen from the time of CR1, in patients eligible for curative intent ASCT. It is anticipated that continued epigenetic priming peri-ASCT with CC-486 can favorably alter disease biology for AML patients in CR1, modulate immune surveillance to decrease relapse risk, mitigate adverse graft versus host disease (GVHD) biology without significant compromise on the quality of life. This pilot study will assess the suitability of CC-486 as a bridge to transplant, will help optimize the timing between CC-486 administration and the start of transplant preparative regimen, and will assess the utility of continued CC-486 maintenance post-transplant.

Conditions

• Acute Myeloid Leukemia

Interventions

Type	Name	Description
DRUG	oral azacitidine (CC-486)	Oral azacitidine is indicated for continued treatment of adult patients with acute myeloid leukemia who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy.

Timeline

• Start date: 2023-11-01
• Primary completion: 2028-11-01
• Completion: 2029-04-01
• First posted: 2023-12-26
• Last updated: 2023-12-26

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study