This is a pilot phase I study evaluating the effect of motixafortide on determination of measurable residual disease (MRD) level in patients with acute myeloid leukemia (AML) who have completed induction treatment. Consenting and eligible patients will undergo standard of care (SOC) bone marrow and peripheral blood assessments with SOC MRD assays, followed by a single injection of motixafortide. Ten to 14 hours after injection, the patient will undergo peripheral blood collection for the same applicable MRD tests
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
DIAGNOSTIC
Masking
NONE
Enrollment
10
Motixafortide is a CXCR4 inhibitor for the mobilization of hematopoietic stem progenitor cells (HSPCs) in patients undergoing autologous stem cell transplantation. It is provided as a single subcutaneous injection.
Washington University School of Medicine
St Louis, Missouri, United States
Efficacy of motixafortide on measurable residual disease (MRD) levels
The efficacy of motixafortide on MRD levels will be measured as the proportion of patients who have at least one of the following, as measured in peripheral blood, before versus after motixafortide: MRD negative to MRD positive conversion, or the 5% absolute increase in MRD level by flow cytometry, or leukemia defining polymerase-chain reaction (PCR) transcript (For fusion-driven leukemias or NPM1 and FLT3-ITD-mutated leukemias) or 5% increase in the VAF of recurrent leukemia associated gene mutations by next-generation sequencing (NGS).
Time frame: Day 1 before motixafortide and Day 2 (estimated total time is 2 days)
Proportion of patients changing from negative to positive MRD levels
The proportion will be calculated by number of patients with negative MRD levels at enrollment who had positive MRD levels measured after treatment compared to total number of patients.
Time frame: Day 1 before motixafortide and Day 2 (estimated total time is 2 days)
Percentage change in variant allele frequency (VAF) levels by next-generation sequencing (NGS) using error-corrected sequencing (MRD-Seq)
Time frame: Day 1 before motixafortide and Day 2 (estimated total time is 2 days)
Percentage change in VAF levels by NGS using MRD-Seq between bone marrow and peripheral blood assessments
Time frame: Day 1 before motixafortide and Day 2 (estimated total time is 2 days)
Percentage change in detectable transcript levels by polymerase chain reaction (PCR)
Time frame: Day 1 before motixafortide and Day 2 (estimated total time is 2 days)
Relapse-free survival (RFS)
RFS is defined as the duration of time from the date of MRD testing to disease relapse or death from any cause, whichever occurs first. Patients who are alive without disease relapses at the time of database lock for analysis will be censored at the last follow up date. Relapse is defined in the ELN 2022 criteria as bone marrow blasts ≥ 5% or reappearance of blasts in the blood in at least 2 peripheral blood samples at least one week apart or development of extramedullary disease.
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Time frame: From Day 1 through completion of follow-up (estimated total time is 18 months)
Time to next line of therapy
Measured as the duration of time between initial MRD testing and next line of therapy.
Time frame: From Day 1 through completion of follow-up (estimated total time is 18 months)
Overall survival (OS)
OS is defined as the duration of time from the date of MRD testing to death from any cause. Patients who are alive at the time of database lock for analysis will be censored at the last follow up date.
Time frame: From Day 1 through completion of follow-up (estimated total time is 18 months)