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Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas

NCT06465199 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 289

Last updated 2026-04-13

No results posted yet for this study

Summary

The purpose of this study is to evaluate the investigational oral drug AMXT 1501 in combination with oral eflornithine (DFMO). An investigational drug is one that has not been approved by the U.S. Food \& Drug Administration (FDA), or any other regulatory authorities around the world for use alone or in combination with any drug, for the condition or illness it is being used to treat.

The goals of this part of the study are:

* Establish a recommended dose of AMXT 1501 in combination with DFMO
* Test the safety and tolerability of AMXT 1501 in combination with DFMO
* To determine the activity of study treatments chosen based on:
* How each subject responds to the study treatment
* How long a subject lives without their disease returning/progressing

Conditions

  • DIPG Brain Tumor
  • Atypical Teratoid/Rhabdoid Tumor
  • Embryonal Tumor With Multilayered Rosettes
  • Ewing Sarcoma
  • Diffuse Intrinsic Pontine Glioma
  • Osteosarcoma
  • Neuroblastoma

Interventions

DRUG

Eflornithine (DFMO)

Oral DFMO capsules

DRUG

AMXT 1501 Dicaprate

Capsule

Sponsors & Collaborators

  • Aminex Therapeutics, Inc.

    collaborator INDUSTRY

  • Penn State University

    collaborator OTHER

  • Milton S. Hershey Medical Center

    lead OTHER

Principal Investigators

  • Giselle Saulnier Sholler, MD · Penn State Health Children's Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
CROSSOVER

Eligibility

Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-04-28
Primary Completion
2033-02-28
Completion
2035-02-28
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06465199 on ClinicalTrials.gov