Motixafortide for Acute Myeloid Leukemia

Overseen By Samuel Urrutia, MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Washington University School of Medicine

Must not be taking: Investigational agents

Disqualifiers: Stem cell transplant, Cardiac disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

Duration: 2 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores how the drug motixafortide affects measurable residual disease (MRD) levels in patients with acute myeloid leukemia (AML), a type of blood cancer. Researchers aim to determine if a single injection of motixafortide can help detect MRD, which consists of a small number of cancer cells remaining after treatment. The trial includes standard blood and bone marrow tests before and after administering the drug. Individuals with AML, excluding the APL subtype, who have completed initial chemotherapy might be suitable for this study. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants, providing an opportunity to contribute to significant findings.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, you cannot participate if you are currently receiving any other investigational agents.

Is there any evidence suggesting that motixafortide is likely to be safe for humans?

Research shows that motixafortide is generally safe for patients. In a previous study, researchers administered it as a single injection and monitored its safety. The treatment did not cause any serious side effects. Some patients experienced mild reactions at the injection site, such as redness or swelling, but these were temporary and resolved on their own.

This treatment is also being tested in other studies and has been used in different situations. In those cases, motixafortide did not cause serious problems, confirming its safety. Notably, motixafortide has received special status for treating acute myeloid leukemia (AML) in the U.S., indicating the FDA sees promise in this treatment.

For those considering joining a trial with motixafortide, current research suggests it is safe, though some mild risks exist. Always consult your doctor to determine if this trial is suitable for you. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Motixafortide is unique because it targets the CXCR4 receptor, which plays a key role in the survival and spread of Acute Myeloid Leukemia (AML) cells. Unlike standard chemotherapy treatments for AML, which often involve combinations of drugs like cytarabine and anthracyclines, Motixafortide specifically disrupts the interaction between AML cells and their protective bone marrow environment, potentially making the cancer cells more vulnerable to treatment. Researchers are excited about this targeted approach because it might reduce the cancer's ability to resist treatment and could lead to better outcomes for patients with fewer side effects.

What evidence suggests that motixafortide might be an effective treatment for acute myeloid leukemia?

Research has shown that motixafortide may improve outcomes for patients with acute myeloid leukemia (AML). In studies, motixafortide, combined with a drug called G-CSF, greatly increased stem cell collection. Nearly 90% of patients successfully gathered enough stem cells, crucial for effective treatment. This trial will evaluate motixafortide's potential to reduce measurable residual disease (MRD), the amount of cancer remaining after treatment. These findings offer hope that motixafortide could enhance treatment results for AML patients. ^{1 2 6 7 8}

Who Is on the Research Team?

Samuel Urrutia, MD

Principal Investigator

Washington University School of Medicine

Are You a Good Fit for This Trial?

This trial is for patients with acute myeloid leukemia (AML) who have finished their initial treatment. They must be able to undergo bone marrow and blood tests to check for minimal residual disease (MRD), which are cancer cells that remain after treatment.

Inclusion Criteria

I have AML and have had 1-2 rounds of initial chemotherapy.

I am scheduled for standard blood and bone marrow tests to check for minimal disease.

I am 18 years old or older.

Exclusion Criteria

I have chronic hepatitis B but it's under control with medication.

Evidence of more than 5% blasts in the peripheral blood by manual differential within 5 days prior to study enrollment

I have another cancer that won't affect this trial's treatment.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive a single injection of motixafortide followed by peripheral blood collection for MRD tests 10-14 hours later

2 days

2 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, with follow-up every 4 months

18 months

What Are the Treatments Tested in This Trial?

Interventions

• Motixafortide

Trial Overview

The study is testing the effect of a single injection of motixafortide on MRD levels in AML patients. After the injection, doctors will collect blood samples to see if there's any change in the number of remaining cancer cells.

How Is the Trial Designed?

Group I: Motixafortide Injection Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Washington University School of Medicine

Lead Sponsor

Trials

2,027

Recruited

2,353,000+

Gamida Cell ltd

Industry Sponsor

Trials

9

Recruited

290+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07392970?locStr=United+States&country=United+States

Motixafortide for MRD Sensitization in AML

This is a pilot phase I study evaluating the effect of motixafortide on determination of measurable residual disease (MRD) level in patients ...

2.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC10115633/

Motixafortide and G-CSF to mobilize hematopoietic stem cells ...

Mobilization with motixafortide + G-CSF resulted in 92.5% of patients meeting the primary endpoint of collecting ≥6 × 106 CD34+ cells kg–1 ...

3.

synapse.patsnap.com

synapse.patsnap.com/article/what-clinical-trials-have-been-conducted-for-motixafortide

What clinical trials have been conducted for Motixafortide?

The GENESIS Phase III trial showed that motixafortide in combination with G-CSF significantly enhanced stem cell collection, with nearly 90% of ...

4.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/nda/2023/217159Orig1s000IntegratedR.pdf

217159Orig1s000 - accessdata.fda.gov

Motixafortide is administered at 1.25 mg/kg, via slow. (approximately 2 minutes) subcutaneous injection 10 to 14 hours prior to the initiation ...

5.

gamida-cell.com

gamida-cell.com/press_release/ayrmid-reports-promising-new-real-world-data-on-motixafortide-for-stem-cell-mobilization-in-sickle-cell-disease/

Ayrmid Reports Promising New Real-World Data on ...

Among the seven patients who previously failed plerixafor, motixafortide led to a substantial improvement in stem cell mobilization, enabling ...

6.

oncologynewscentral.com

oncologynewscentral.com/drugs/monograph/187510-323068/motixafortide-subcutaneous

Motixafortide: uses, dosing, warnings, adverse events, ...

The safety and efficacy of motixafortide for this use are based principally on the results of a randomized, double-blind, placebo-controlled trial (GENESIS) in ...

7.

clinicaltrials.gov

clinicaltrials.gov/study/NCT03154827?term=MOTIXAFORTIDE&viewType=Table&rank=5

Safety, Tolerability and Efficacy of the BL-8040 and ...

This study will assess the safety and effectiveness of a combination of BL-8040 and atezolizumab to find out what is the Safety, Tolerability and Efficacy of ...

8.

synapse.patsnap.com

synapse.patsnap.com/article/what-are-the-approved-indications-for-motixafortide

What are the approved indications for Motixafortide?

Motixafortide has been granted orphan drug designation for AML in the USA; however, its use in this setting is still under clinical evaluation.