Actively Recruiting

Early Phase 1
Age: 18Years +
All Genders
ID05717153

Intratumoral Extracellular Metabolic Impact of DFMO and AMXT 1501 in Patients With Diffuse or High Grade Glioma

Led by Mayo Clinic · Updated on 2025-11-04

18

Participants Needed

1

Research Sites

34 weeks

Total Duration

On this page

Sponsors

M

Mayo Clinic

Lead Sponsor

N

National Cancer Institute (NCI)

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are studying how the brain tumor metabolism in patients with diffuse or high grade glioma responds to two drugs: eflornithine (DFMO) and the polyamine transport inhibitor AMXT-1501. This early phase I trial aims to understand how these drugs affect molecules in the tumor's fluid, which may influence tumor survival and growth. The study also examines the safety and feasibility of using microdialysis to monitor drug effects in the brain after surgery. Participants are randomly assigned to one of three groups. All undergo surgical tumor removal, MRI, and catheter placement for microdialysis at baseline. Treatment varies by group with oral doses of DFMO and AMXT-1501 given on specific days after surgery. Some groups receive DFMO alone initially or a placebo for the first two days, followed by combination therapy. Additional procedures include CT scans and blood collection during the study. Throughout the study, patients remain in the hospital for three extra days after surgery for continuous microdialysis monitoring. Researchers collect tumor fluid samples and blood to measure drug levels and changes in metabolites. They track the primary outcome of changes in extracellular guanidinoacetate ratios and secondary outcomes including glutamate levels, drug concentrations, and adverse events. The study period includes up to two months of observation post-treatment to assess drug effects and safety.

CONDITIONS

Brief Title

Intratumoral Extracellular Metabolic Impact of DFMO and AMXT 1501 in Patients With Diffuse or High Grade Glioma

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Age 2018 years or older
  • Clinical and radiographic evidence suggesting a diagnosis of diffuse high grade glioma or prior diagnosis of diffuse glioma
  • Planned subtotal resection or biopsy due to tumor location, size, or clinical indication deemed appropriate by surgeon
  • Provide written informed consent for this study and Neuro-Oncology biorepository for CSF and blood sample archiving
  • Willing to remain hospitalized for three days beyond standard post-operative stay for longitudinal microdialysis
  • Absolute neutrophil count (ANC) 201.5 x 10^9/L without transfusion within 7 days before lab assessment
  • Platelet count 20100 x 10^9/L without transfusion within 7 days before lab assessment
  • Hemoglobin 209 g/dL without transfusion support within 7 days before lab assessment
  • Activated partial thromboplastin time or partial thromboplastin time 201.5 x upper limit of normal (ULN)
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) 202.5 x ULN
  • Total serum bilirubin 201.5 x ULN
  • Clinically euthyroid based on thyroid stimulating hormone (TSH)
  • Serum creatinine 201.5 x ULN or creatinine clearance 2060 mL/min/1.73 m^2 for elevated creatinine
  • Negative serum or urine pregnancy test for females of childbearing age within 14 days prior to registration
Not Eligible

You will not qualify if you...

  • Not suitable for surgery due to medical history or uncontrolled illness limiting safety or study compliance
  • Pregnant or nursing women, prisoners, or mentally handicapped individuals
  • Unable to swallow tablets or risk impaired absorption of oral medication, including refractory vomiting or certain gastrointestinal surgeries
  • Known allergy or hypersensitivity to DFMO or AMXT 1501
  • Contraindication to MRI or gadolinium administration

AI-Screening

AI-Powered Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Surgery and Immediate Post-operative Care

Duration - 1 day

Participants undergo magnetic resonance imaging (MRI), surgical tumor resection, and placement of catheters for microdialysis at baseline.

1 baseline visit (in-person)

Treatment

Duration - 5 days post-surgery

Participants receive oral medications (DFMO, AMXT 1501, and/or eflornithine) for up to 5 days post-surgery depending on their assigned treatment arm. Microdialysate collection, computed tomography (CT) scans, and blood collection occur during this period.

Daily visits or assessments during hospital stay for up to 5 days

Post-operative Follow-up

Duration - Up to 2 months

Participants are monitored for changes in tumor and brain extracellular metabolites, drug levels, and adverse events for up to 2 months after surgery and treatment.

Periodic visits for assessments and sample collections over 2 months

Trial Site Locations

Total: 1 location

1

Mayo Clinic in Rochester

Rochester, Minnesota, United States, 55905

Actively Recruiting

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Research Team

C

Clinical Trials Referral Office

How is the study designed?

Study Type

INTERVENTIONAL

Masking

DOUBLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

BASIC_SCIENCE

Number of Arms

3

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