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

Recruiting now · Phase 1/Phase 2

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

In brief

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

Key facts

Study ID

NCT06465199

Run by

Milton S. Hershey Medical Center

People needed

289

Starts

2026-05-13

Expected to finish

2035-05-01

Last updated by the study team

2026-07-01

Who can join

Age: any, up to 26. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age:
• All participants : Must be a maximum of 26 years of age at diagnosis
• Age at enrollment by Phase:
• Safety Run-in (Dose level 1)-The first three (3) participants enrolled will be ≥ 12 years of age at enrollment. Once evaluated for safety by DSMB, we will move on to the next three (3) participants enrolled who will be ≥6 years of age at enrollment. Once evaluated for safety by DSMB, we will move on to the Phase I.
• Phase I and II: ≤ 26 years of age at diagnosis.
• Pathology
• All participants must have a confirmed pathologic diagnosis of tumor type (except for DIPG):
• 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)
• Tumor assessment:
• Disease staging must be performed at baseline during the 28 day screening period prior to first dose of study drug.
• Disease Status:
• Relapsed or Refractory Neuroblastoma Relapsed disease defined as: High-risk neuroblastoma that was previously in remission after standard therapy (at least 4 cycles of aggressive multi-drug induction chemotherapy, with or without radiation, surgery, and immunotherapy, or according to a standard high-risk treatment/neuroblastoma protocol).
• Refractory disease defined as: High-risk neuroblastoma that 1) failed to achieve CR after at least 4 cycles of aggressive multi-drug induction chemotherapy with or without radiation and surgery, followed by immunotherapy, or according to a standard high-risk treatment/neuroblastoma protocol, or 2) progression during upfront therapy or 3) with disease remaining after standard immunotherapy.
• Eligible NB participants may have active disease or no active disease.
• NB participants with no active disease need to meet the following criteria:
• Timing from prior therapy: Enrollment (first dose of study drug) no later than 60 days from most recent therapy.
• NB participants with active disease need to meet the following criteria:
• 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).
• Relapsed or refractory ETMR/ATRT Participants that have relapsed following standard of care therapy or having progressed during standard of care therapy and non-responsive/progressive to accepted curative therapy, including up-front chemotherapy and radiation and/or high-dose chemotherapy with stem cell rescue.

You may not qualify if…

• BSA of <0.25 m2
• Investigational Drugs: Participants who are currently receiving another investigational drug are excluded from participation.
• Anti-cancer Agents: Participants who are currently receiving other anticancer agents are not eligible. Participants must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.
• Infection: Participants who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
• Participants who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.

Where it is running

• University of Alabama/Children's of Alabama — Birmingham, Alabama, United States (enrolling)
• Arkansas Children's Hospital — Little Rock, Arkansas, United States (enrolling)
• Connecticut Children's Hospital — Hartford, Connecticut, United States (enrolling)
• Nicklaus Children's Hospital — Miami, Florida, United States (enrolling)
• Arnold Palmer Hospital for Children — Orlando, Florida, United States (enrolling)
• St. Joseph's Children's Hospital — Tampa, Florida, United States (enrolling)
• Kapiolani Medical Center for Women and Children — Honolulu, Hawaii, United States (enrolling)
• Children's Mercy Hospitals and Clinics — Kansas City, Kansas, United States (enrolling)
• Cardinal Glennon Children's Medical Center — St Louis, Missouri, United States (enrolling)
• Penn State Milton S. Hershey Medical Center and Children's Hospital — Hershey, Pennsylvania, United States (enrolling)
• Monroe Carrell Jr. Children's Hospital at Vanderbilt — Nashville, Tennessee, United States (enrolling)
• Children's Medical Center — Dallas, Texas, United States (enrolling)

Full record on ClinicalTrials.gov

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