RecruitingPhase 1Phase 2NCT06465199

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


Sponsor

Milton S. Hershey Medical Center

Enrollment

289 participants

Start Date

Apr 28, 2026

Study Type

INTERVENTIONAL

Conditions

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


Eligibility

Max Age: 21 Years

Plain Language Summary

Simplified for easier understanding

This study is testing a combination of two drugs — eflornithine (DFMO) and AMXT 1501 — in children, adolescents, and young adults with neuroblastoma, certain brain tumors, or sarcomas that have returned or stopped responding to treatment. The drugs target a cellular pathway that helps tumors grow. **You may be eligible if...** - You are 21 years old or younger at the time of diagnosis - You have a confirmed diagnosis of neuroblastoma, a specific CNS tumor, or sarcoma that has relapsed or is refractory - You are in reasonable health with adequate organ function **You may NOT be eligible if...** - You have had too many prior lines of therapy without adequate recovery - Your disease has spread in ways that make safe participation difficult Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGEflornithine (DFMO)

Oral DFMO capsules

DRUGAMXT 1501 Dicaprate

Capsule


Locations(10)

University of Alabama/Children's of Alabama

Birmingham, Alabama, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Connecticut Children's Hospital

Hartford, Connecticut, United States

Nicklaus Children's Hospital

Miami, Florida, United States

Arnold Palmer Hospital for Children

Orlando, Florida, United States

St. Joseph's Children's Hospital

Tampa, Florida, United States

Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, United States

Penn State Milton S. Hershey Medical Center and Children's Hospital

Hershey, Pennsylvania, United States

Monroe Carrell Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, United States

Children's Medical Center

Dallas, Texas, United States

View Full Details on ClinicalTrials.gov

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NCT06465199


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