NCT06465199

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

A Dose Escalation Study Using Eflornithine (DFMO) and AMXT 1501 Followed by a Randomized Controlled Trial of DFMO With or Without AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 289 (estimated)
• Sponsor: Milton S. Hershey Medical Center · Academic / Other
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

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

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

Interventions

Type	Name	Description
DRUG	Eflornithine (DFMO)	Oral DFMO capsules
DRUG	AMXT 1501 Dicaprate	Capsule

Timeline

• Start date: 2026-04-28
• Primary completion: 2033-02-01
• Completion: 2035-02-01
• First posted: 2024-06-18
• Last updated: 2026-04-13

Locations

10 sites across 1 country: United States

Regulatory

• FDA-regulated drug study