Oral Azacitidine for Transplant-Eligible Patients with Acute Myeloid Leukemia

REMAIN1: Relapse Prevention With Maintenance Oral Azacitidine in Transplant Eligible Patients With Acute Myeloid Leukemia Not Proceeding to Transplant Due to Racial or Socioeconomic Disparities

This study is testing if an oral medication called azacitidine can help patients with acute myeloid leukemia who are eligible for a transplant and face health challenges stay healthy after treatment.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Virginia Commonwealth University (Academic / other)
• Locations: 1 site (Richmond, Virginia)
• Trial ID: NCT06370000 on ClinicalTrials.gov

What this trial studies

This pilot study evaluates the feasibility, toxicity, and efficacy of oral azacitidine as a maintenance therapy for transplant-eligible patients with acute myeloid leukemia (AML) who are medically underserved. The study focuses on patients in complete response who do not have FLT3 mutations and face disadvantages in key social determinants of health. It is a non-randomized, open-label study conducted at a single institution, aiming to address health inequalities in this patient population.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with non-FLT3 mutated AML in complete response, who are medically eligible for allogeneic hematopoietic cell transplant and face social disadvantages.

Not a fit: Patients with FLT3 mutated AML or those who are not medically eligible for allogeneic hematopoietic cell transplant may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could improve maintenance treatment options for underserved AML patients, potentially enhancing their outcomes post-transplant.

How similar studies have performed: While there have been studies on azacitidine, this specific approach targeting health inequalities in transplant-eligible AML patients is novel.

Eligibility 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

Where this trial is running

Richmond, Virginia

• Virginia Commonwealth University — Richmond, Virginia, United States (Recruiting)

Study contacts

• Principal investigator: Keri Maher, DO — Virginia Commonwealth University
• Study coordinator: Massey IIT Research Operations

Conditions Acute Myeloid Leukemia