Oral Azacitidine in Transplant-Eligible Patients With Acute Myeloid Leukemia (AML) Suffering From Health-Inequality (REMAIN1)

Virginia Commonwealth University (VCU)

Status and phase

Enrolling

Phase 4

Conditions

Acute Myeloid Leukemia

Treatments

Drug: Oral Azacitidine

Study type

Interventional

Funder types

Other

Identifiers

NCT06370000

MCC-23-20817

HM20029540 (Other Identifier)

Details and patient eligibility

About

Test feasibility of an oral maintenance strategy for transplant eligible AML patients in first CR who are medically underserved or have a disadvantage in the CDC SDOH domains

Full description

This is a non-randomized open-label single institution pilot study that will evaluate the feasibility, toxicity, and efficacy of maintenance oral azacitidine in medically transplant eligible non-FMS-like tyrosine kinase 3 (FLT3) mutated AML patients with a disadvantage in at least 1 of the 5 key Center for Disease Control and Prevention (CDC) defined social determinants of health (SDOH) domains that are preclusive to transplant at time of study enrollment, as identified either by the patient or a member of the healthcare team.

Enrollment

12 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

Patients must have histologically or cytologically confirmed non-Acute Promyelocytic (APL) FLT3 negative AML and have completed induction and consolidation as defined by the treating physician and must be in complete response (CR), Complete response with partial hematologic recovery (CRh), or Complete response with incomplete count recovery (CRi) at time of study enrollment
For patients in CR1, AML disease phenotype must be one that is considered for allo HCT in CR1 (intermediate or high risk by European Leukemia Net (ELN), MRD+ CR, slow clearance of MRD) or any AML phenotype (aside from FLT3+ and APL) in CR2 and beyond
Medically eligible for allogeneic hematopoietic cell transplant (allo HCT) as defined by either: treating physician discretion, transplant physician discretion, or hematopoietic cell transplantation-specific Comorbidity index (HCT-CI) index of 5 or less
Age ≥ 18 years
Enrollment must occur within 4 months of completion of therapy
A patient or staff identified health disparity in 1 of the 5 Centers for Disease Control (CDC) defined social determinants of health (SDOH). This may include financial difficulties, lack of caregiver support, difficulties with medical literacy, rurality, appropriate access to health care, lack of an appropriate allogeneic hematopoietic cell transplant (allo HCT) donor, substance abuse
Patient must have adequate organ function defined as: Creatinine clearance (by Cockroft-Gault formula) greater than or equal to 29 mL/min, total bilirubin and aspartate aminotransferase/ alanine transaminase (AST/ALT) ≤ to institutional 2x upper limit of normal (except Gilbert's syndrome, which may enroll if < 2x patient's baseline total bilirubin)
Eastern Cooperative Oncology Group (ECOG) 0,1,2,3
Ability to take oral medications
No history of malabsorption syndrome which, in the investigator's opinion, may inhibit absorption of oral medications
Women of childbearing potential must consent to effective contraception during study treatment and at least 6 months following the last dose. Women who are breastfeeding are also excluded
Male patients must consent to effective contraception during study and at least 3 months after last dose
Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria A patient who meets any of the following exclusion criteria is ineligible to participate in the study.

FMS-like tyrosine kinase 3 (FLT3 ITD) or tyrosine kinase domain (TKD) mutation
Uncontrolled central nervous system (CNS) involvement
History of hypersensitivity or allergic reaction to azacitidine or its components
Stem cell transplant within previous 3 months prior to initiation of study therapy
Uncontrolled intercurrent illness or infection
History of prior therapy with oral azacitidine
Female patients who are pregnant or intend to donate eggs during the study or for 6 months after receiving their last dose of study drug
Male patients who intend to donate sperm during the course of this study or for 3 months after last dose
Other malignancy for which the patient is currently receiving therapy (except excisable skin cancer)
Medical, psychological, or social condition that, in the opinion of the investigator, may increase the participant's risk or limit the participant's adherence with study requirements