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Testing the drug AMXT 1501 with eflornithine (DFMO) for neuroblastoma, brain and spinal cord tumors, and sarcomas

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

How far along is this research?

This is a trial that is enrolling or has been updated. It is not a finding yet.

This is an early study that is still working out the right dose and checking safety in people.

Status: Recruiting. It is looking for patients now. Talk to your care team about whether it fits you.

The trial team last updated this record on . Always check the trial page for the latest.

The short version

This study is testing an experimental pill, AMXT 1501, taken together with a pill called eflornithine (DFMO).

Who this trial is for. This study is for people with neuroblastoma, tumors of the brain or spinal cord, or sarcomas. It tests AMXT 1501 taken by mouth along with eflornithine (DFMO) taken by mouth. AMXT 1501 is an investigational drug, which means the FDA has not approved it.

Where it stands. This trial has not reported results. The team is working out a recommended dose of AMXT 1501 given with DFMO. They are also checking how safe it is and how well people handle it. They are watching how people respond to the treatment, and how long people go without their disease coming back or getting worse. Those answers are not in yet.

What this means, and what it doesn't

What it could mean: If you or the person you care for has one of these cancers, this is one study to know about. You could ask your care team whether it is worth looking into. Your team can help you understand what taking part would involve.

What it doesn't mean: This does not mean AMXT 1501 works. A trial is a test, not a proven treatment. Being in a trial is not a guarantee that it will help you. It is not a promise of a cure. The drug has not been approved by the FDA for this use.

Source: ClinicalTrials.gov, August 12, 2026 · Read the original

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