Recruiting
Early Phase 1

DFMO & AMXT 1501

Sponsor:

Mayo Clinic

Code:

NCT05717153

Conditions

Diffuse Glioma

Malignant Glioma

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Biospecimen Collection

Computed Tomography

Eflornithine

Magnetic Resonance Imaging

Polyamine Transport Inhibitor AMXT-1501 Dicaprate

Study Details

Brief summary:

This early phase I trial studies brain tumor (glioma) metabolism in response to eflornithine (DFMO) and polyamine transport inhibitor AMXT-1501 dicaprate (AMXT 1501) in patients with diffused or high grade glioma. Brain tumors use and produce certain molecules to survive and grow. DFMO is an irreversible inhibitor of ornithine decarboxylase, the enzyme catalyzing polyamine synthesis. AMXT 1501 is a polyamine transport inhibitor which prevents uptake of polyamines from the extracellular environment. This trial is being done to analyze how DFMO and AMXT 1501 affect brain tumor metabolism based on the molecules in the tumor's fluid.

Conditions

Diffuse Glioma

Malignant Glioma

Study ID

NCT05717153

Start date

Oct 1, 2023

Status verified date

Oct, 2025

Completion date

Sep 15, 2027

Anticipated

Primary completion date

Jan 15, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • Age >= 18 years
  • Clinical and radiographic evidence suggesting a diagnosis of a diffuse high grade glioma (HGG), or a prior diagnosis of a diffuse glioma
  • Planned subtotal resection or biopsy due to tumor location, size, or other clinical indication deemed appropriate by the surgeon
  • Provide written informed consent for the current study and the Neuro-Oncology biorepository for archiving of cerebrospinal fluid (CSF) and blood samples collected on this protocol. Willing to remain in the hospital at Mayo Clinic (Rochester, MN) for three days added to their standard post-operative stay to undergo longitudinal microdialysis
  • Absolute neutrophil count (ANC) >= 1.5 x 10\^9/L without transfusion within 7 days preceding the lab assessment (obtained =< 14 days prior to registration)
  • Platelet >= 100 x 10\^9/L, without transfusion within 7 days preceding the lab assessment (obtained =< 14 days prior to registration)
  • Hemoglobin >= 9 g/dL, without transfusion support within 7 days preceding the lab assessment (obtained =< 14 days prior to registration)
  • Activated partial thromboplastin time or partial thromboplastin time (aPTT or PTT) =< 1.5 x upper limit of normal (ULN) (obtained =< 14 days prior to registration)
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 2.5 x ULN (obtained =< 14 days prior to registration)
  • Total serum bilirubin =< 1.5 x ULN (obtained =< 14 days prior to registration)
  • The patient is clinically euthyroid \[Thyroid Stimulating Hormone (TSH)\]
  • Serum creatinine =< 1.5 x ULN or creatinine clearance >= 60 mL/min/1.73 m\^2 for patients with serum creatinine levels above 1.5 x ULN (obtained =< 14 days prior to registration)
  • Negative serum or urine pregnancy test is required for female subjects of childbearing age < 14 days prior to registration

Exclusion Criteria:

  • Inappropriate surgical candidates due to current or past medical history or uncontrolled concurrent illness which limits safety of or compliance to study proceedings
  • Vulnerable populations: pregnant or nursing women, prisoners, mentally handicapped
  • Unable to swallow tablets or who are at risk for impaired absorption of oral medication. NOTE: This includes but not limited to, refractory vomiting, gastric resection/bypass, and duodenal/jejunal resection
  • Known hypersensitivity or allergy to DFMO or AMXT 1501
  • Contraindication to MRI or administration of gadolinium

Study Design

Enrollment

18 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Basic Science

Interventions and Outcome Measures

Arms

experimental: Arm I (MRI, resection, DFMO, AMXT 1501)

Patients undergo magnetic resonance imaging (MRI) and surgical resection at baseline. Patients receive eflornithine PO in combination with AMXT 1501 PO on days 1-5 post-surgery. Patients also undergo CT after surgery and collection of blood on study.

placebo comparator: Arm II (MRI, resection, placebo, DMFO, AMXT 1501)

Patients undergo magnetic MRI and surgical resection at baseline. Patients receive placebo PO on days 1 and 2 post-surgery, and then receive eflornithine PO and AMXT 1501 PO on days 3-5 post-surgery. Patients also undergo CT after surgery and collection of blood on study.

active comparator: Arm III (MRI, resection, DMFO, AMXT 1501)

Patients undergo magnetic MRI and surgical resection at baseline. Patients receive eflornithine PO alone on days 1 and 2 post-surgery, then receive eflornithine PO in combination with AMXT 1501 PO on days 3-5 post-surgery. Patients also undergo CT after surgery and collection of blood on study.

Interventions

Biospecimen Collection

Undergo collection of blood

Computed Tomography

Undergo CT

Eflornithine

Given PO

Magnetic Resonance Imaging

Undergo MRI

Polyamine Transport Inhibitor AMXT-1501 Dicaprate

Given PO

Resection

Undergo surgical resection

Microdialysis

Undergo Microdialysis

Placement

Undergo placement of catheters

Primary outcome measure

  • Change in the tumor/brain extracellular guanidinoacetate ratio [ Time Frame: Baseline up to 2 months ]

Central Contacts and Locations

Central contacts

Clinical Trials Referral Office

855-776-0015mayocliniccancerstudies@mayo.edu

Locations

Mayo Clinic in Rochester

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Clinical Trials Referral Office

855-776-0015mayocliniccancerstudies@mayo.edu

Principal Investigator:

Terry Burns, MD, PhD

More Information

Sponsor

Mayo Clinic

Last update posted

Nov 4, 2025

Last verified

Oct, 2025

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Mayo Clinic on 2025-11-04.