Motixafortide for MRD Sensitization in AML

NCT07392970 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 202603045
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Acute Myeloid Leukemia; Measurable Residual Disease

Keywords

AML; MRD; NGS; PCR

Outcome Measures

Primary Outcomes (1)

• 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).

  Day 1 before motixafortide and Day 2 (estimated total time is 2 days)

Secondary Outcomes (7)

• Proportion of patients changing from negative to positive MRD levels

  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)

  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

  Day 1 before motixafortide and Day 2 (estimated total time is 2 days)

• Percentage change in detectable transcript levels by polymerase chain reaction (PCR)

  Day 1 before motixafortide and Day 2 (estimated total time is 2 days)

• Relapse-free survival (RFS)

  From Day 1 through completion of follow-up (estimated total time is 18 months)

Study Arms (1)

Motixafortide Injection

EXPERIMENTAL

Consenting and eligible patients will bone marrow and peripheral blood testing assessments along with measurable residual disease (MRD) assays. Following completion of assessments, patients will receive a single injection of Motixafortide. The following day, 10-14 hours after the injection, patients will repeat the peripheral blood testing for MRD tests. Investigators will follow up with patients every 4 months for 18 months after study treatment.

Drug: Motixafortide

Interventions

Motixafortide DRUG

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.

Also known as: Aphexda

Motixafortide Injection

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosed with acute myeloid leukemia (AML), excluding APL, treated with 1-2 cycles of front-line chemotherapy.
• Achieved CBC parameters compatible with complete remission as defined by ELN 2022. This must be done within 5 days prior to study enrollment.
• Planning to undergo a standard of care blood draw and bone marrow assessment with SOC MRD assays, including morphology, flow cytometry for MRD, NGS panels for MRD, and PCR tests for MRD as applicable.
• At least 18 years of age.
• ECOG performance status ≤ 2
• Life expectancy \> 3 months.
• Adequate organ function as defined below:
• Total bilirubin ≤ 2.0 x IULN
• AST(SGOT)/ALT(SGPT) ≤ 5.0 x IULN
• Creatinine clearance \> 30 mL/min by Cockcroft-Gault
• Ability to understand and willingness to sign an IRB approved written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants.

You may not qualify if:

• Evidence of more than 5% blasts in in the peripheral blood by manual differential within 5 days prior to study enrollment.
• Prior history of allogeneic stem cell transplant.
• Prior or concurrent malignancy whose natural history has the potential to interfere with the safety or efficacy assessment of the investigational regimen. Patients with prior or concurrent malignancy that does NOT meet that definition are eligible for this trial
• Currently receiving any other investigational agents.
• A history of allergic reactions attributed to compounds of similar chemical or biologic composition to motixafortide.
• Uncontrolled intercurrent illness including, but not limited to: ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia. Patients with a known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Function Classification; to be eligible for this trial, patients should be a class 2B or better.
• Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of study entry.
• HIV-infected if not on effective anti-retroviral therapy with undetectable viral load for 6 months. Patients with HIV who are receiving effective anti-retroviral therapy and have had an undetectable viral load for at least 6 months are eligible. HIV testing not required in the absence of known history of infection.
• Evidence of chronic hepatitis B virus (HBV) that is detectable on suppressive therapy. Patients with evidence of chronic HBV infection with undetectable HBV viral load on suppressive therapy are eligible. HBV testing not required in the absence of known history of infection.
• History of hepatitis C virus (HCV) infection that has not been cured or that has a detectable viral load. Patients with a history of HCV that has been treated and cured are eligible. Patients with HCV infection who are currently on treatment and have an undetectable HCV viral load are eligible. HCV testing not required in the absence of known history of infection.

Sponsors & Collaborators

• Washington University School of Medicine: lead
• Gamida Cell ltd: collaborator

Study Sites (1)

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Related Links

• Alvin J. Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Neoplasm, Residual

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Neoplastic Processes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Samuel Urrutia, MD

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Samuel Urrutia, MD

CONTACT

314-273-6734; surrutia@wustl.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 29, 2026
• First Posted: February 6, 2026
• Study Start (Estimated): May 31, 2026
• Primary Completion (Estimated): June 2, 2028
• Study Completion (Estimated): December 2, 2029
• Last Updated: April 9, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: Data will be shared starting 12 months after publication through 24 months after publication.
• Access Criteria: Proposed use of data has been approved by the Princpal Investigator and Sponsor. Data will be shared via direct communication with the PI.

Locations

US (1)