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

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

NCT Number	NCT06465199
Lead Sponsor	Milton S. Hershey Medical Center Collaborators: Aminex Therapeutics, Inc., Penn State University
Conditions	Atypical Teratoid/Rhabdoid Tumor, Embryonal Tumor With Multilayered Rosettes, Ewing Sarcoma, Diffuse Intrinsic Pontine Glioma, Osteosarcoma, Neuroblastoma, DIPG Brain Tumor
Enrollment	289 participants
Start Date	2026-05-13
Primary Completion	2033-05 (estimated)
Study Completion	2035-05 (estimated)
Updated on ClinicalTrials.gov	2026-08-12