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Eflornithine (DFMO) and AMXT 1501 for Childhood Cancers

This study is testing two investigational oral drugs, eflornithine (DFMO) and AMXT 1501, in combination. These drugs are not yet approved by the FDA. The study aims to find the best dose of AMXT 1501 when given with DFMO, and to see how safe and effective this combination is for children and young adults up to 26 years old with certain cancers like Atypical Teratoid/Rhabdoid Tumor, Ewing Sarcoma, and Osteosarcoma. The researchers will look at how well participants respond to the treatment and how long they live without their disease getting worse (progression-free survival). This study is for patients up to 26 years old at diagnosis.

Study design
This is an interventional study with a planned enrollment of 289 participants. It will first establish a safe dose (Phase I) and then evaluate effectiveness (Phase II).
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants in Phase II will be followed for progression-free survival for 2 years, with an additional 5 years of follow-up.

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NCT06465199

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

Recruiting
PHASE1Up to 26InterventionalTreatment
Milton S. Hershey Medical Center
~289 participants
Updated 2026-08-12 on ClinicalTrials.gov
What's tested:Eflornithine (DFMO)AMXT 1501 Dicaprate

At a glance

Recruiting sites
14 of 14 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Phase I- Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Measured over 28 days
+2 more outcomes measured

Conditions

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

Explore related trials

Atypical Teratoid/Rhabdoid TumorDiffuse Intrinsic Pontine GliomaEmbryonal Tumor with Multilayered RosettesEwing SarcomaNeuroblastomaOsteosarcoma

Where it's being run

14 sites across 11 states
Florida4
Alabama1
Arkansas1
Connecticut1
Hawaii1
Kansas1
Missouri1
New Jersey1

Study leadership

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

Who to contact

BCC Enroll
[email protected]
7175310003
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Relapsed/refractory Neuroblastoma (NB)
Relapsed/refractory Embryonal tumor with multilayer rosettes (ETMR)
Relapsed/refractory Atypical teratoid rhabdoid tumor (ATRT)
Newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG)- radiologic diagnosis acceptable
Relapsed/refractory Ewing Sarcoma (EWS)
Relapsed/refractory Osteosarcoma (OST) 3. Tumor assessment:
Received at least one recent treatment for their relapse/refractory disease and is stable (SD) or better on this treatment.
Participants must not have disease in any organs (including lungs, liver, or brain).
Hematological: Hematological recovery as defined by ANC ≥750/μL (unsupported- \>24 hrs off G-CSF and 7 days off neulasta)
Liver: Adequate liver function as defined by AST and ALT \<10x upper limit of normal
Cardiac: all participants must have:
Renal: Participants must have adequate renal function defined as:

What this trial measures

  • Phase I- Number of Participants with Adverse Events as a Measure of Safety and Tolerability28 days

    To evaluate the safety, tolerability and recommended phase 2 dose (RP2D) of AMXT 1501 in combination with oral DFMO in pediatric and young adult participants.

  • Phase II- Number of Cohort 1 participants with progression free survival (PFS) during study2 years plus 5 years follow up

    To evaluate, in a prospective randomized clinical trial, the efficacy of eflornithine (DFMO) in combination with AMXT 1501 compared to DFMO alone in neuroblastoma (Cohort 1) based upon Progression Free Survival (PFS)

  • Phase II- Number of Cohort 2-4 participants with progression free survival (PFS) during study2 years plus 5 years follow up

    To evaluate the efficacy of eflornithine (DFMO) in combination with AMXT 1501 in non-randomized (Cohorts 2-4) based upon Progression Free Survival (PFS): 2\. Cohort 2-Relapsed/refractory Embryonal Tumor with Multilayered Rosettes (ETMR) Atypical Teratoid Rhabdoid Tumor (ATRT) 3. Cohort 3-Diffuse Intrinsic Pontine Glioma (DIPG) at diagnosis after standard of care radiation therapy 4. Cohort 4- Relapsed/refractory Ewing Sarcoma (EWS) and Osteosarcoma (OST)