Recruiting

DFMO and AMXT 1501 Combination for Neuroblastoma, CNS Tumors, and Sarcomas

Study Aim

This study aims to evaluate the safety, tolerability, and effectiveness of combining DFMO and AMXT 1501 in treating neuroblastoma, CNS tumors, and sarcomas in pediatric and young adult subjects, with a focus on progression-free survival.

What is being tested

Eflornithine (DFMO)

+ AMXT 1501 Dicaprate

Drug

Who is being recruted

Diffuse Intrinsic Pontine Glioma+26

+ Brain Diseases

+ Brain Neoplasms

Until 21 Years

+24 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 1 & 2

Interventional

Study Start: February 2026

See protocol details

Summary

Principal SponsorMilton S. Hershey Medical Center

Study ContactBCC Enroll

Last updated: February 4, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: February 28, 2026

Actual date on which the first participant was enrolled.

This study is exploring a new combination of medications for treating certain types of cancer, including neuroblastoma, central nervous system (CNS) tumors, and sarcomas. The research is focused on understanding how effective and safe the combination of an investigational drug called AMXT 1501, taken as a pill, and difluoromethylornithine (DFMO), given through an intravenous (IV) infusion, can be. This study is important because it aims to find the best dose of AMXT 1501 to use with DFMO and to see if this combination can help patients live longer without their disease getting worse. Participants in the study will take AMXT 1501 orally and receive DFMO through an IV. The study will look at how well patients tolerate this treatment and evaluate its safety. Researchers will also monitor how well the cancer responds to the treatment and how long the participants can go without the cancer progressing. Since AMXT 1501 has not been approved by health authorities like the FDA, this research is a key step in understanding its potential benefits and risks for patients with these types of cancer.

Official TitleA 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

NCT06465199

Principal SponsorMilton S. Hershey Medical Center

Study ContactBCC Enroll

Last updated: February 4, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

289 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Until 21 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Diffuse Intrinsic Pontine GliomaBrain DiseasesBrain NeoplasmsCentral Nervous System DiseasesGliomaNeoplasmsNeoplasms by Histologic TypeNeoplasms by SiteNeoplasms, Connective TissueNeoplasms, Germ Cell and EmbryonalNeoplasms, Glandular and EpithelialNeoplasms, Nerve TissueNervous System DiseasesNervous System NeoplasmsNeuroblastomaSarcomaSarcoma, EwingOsteosarcomaInfratentorial NeoplasmsCentral Nervous System NeoplasmsNeuroectodermal TumorsNeoplasms, Complex and MixedNeoplasms, Connective and Soft TissueNeoplasms, Bone TissueNeuroectodermal Tumors, Primitive, PeripheralNeuroectodermal Tumors, PrimitiveNeoplasms, NeuroepithelialRhabdoid TumorBrain Stem Neoplasms

Criteria

19 inclusion criteria required to participate

Subjects must be >=12 years of age at enrollment.

Subjects must be < 12 years of age at enrollment; may start only after DSMB review confirms the RP2D from the AYA cohort. No subject < 12 years will be treated at a dose level higher than the RP2D established in the Phase I-AYA Cohort.

Subjects must be <= 21 years of age at diagnosis (with possibly two different age-specific RP2Ds).

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).

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5 exclusion criteria prevent from participating

BSA of <0.25 m2.

Subjects who are currently receiving another investigational drug are excluded from participation.

Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy.

Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.

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Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

4 intervention groups are designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

The initial phase I will be an AYA (adolescents and young adult) cohort that will be for subjects ≥12 years of age at enrollment in a standard 3+3 design in which groups of 3 subjects per cohort will be treated and assessed. Subjects will receive up to twenty-four (24), 28-day cycles of AMXT 1501 combined with DFMO. Subjects will receive oral AMXT 1501 at a starting dose of 350 mg/m2 BID each day. The dose escalation scheme for subsequent groups and modifications for dose limiting toxicities (DLT) are detailed in the protocol.

Group II

Experimental

The second phase I will be a pediatric cohort that will be for subjects \< 12 years of age at enrollment in a standard 3+3 design in which groups of 3 subjects per cohort will be treated and assessed. Subjects will receive up to twenty-four (24), 28-day cycles of AMXT 1501 combined with DFMO. Subjects will receive oral AMXT 1501 at a starting dose of 350 mg/m2 BID each day. The dose escalation scheme for subsequent groups and modifications for dose limiting toxicities (DLT) are detailed in the protocol.

Group III

Experimental

In this portion of the study, cohort 1 will be randomized to either receive Arm A: oral AMXT 1501 at the recommended phase 2 dose (RP2D) found in the Phase I along with oral DFMO at the RP2D found in the Phase I on each day of study or Arm B: oral DFMO alone at the recommended phase 2 dose (RP2D) found in the Phase I. Subjects will receive up to twenty-four (24), 28-day cycles of their assigned treatment. Cohorts 2 (ETMR/ATRT), 3 (DIPG), and 4 (Sarcomas) will automatically be assigned to Arm A with AMXT 1501 in combination with DFMO. Subjects in cohort 1 who progress on DFMO alone (and have met the primary PFS endpoint) may cross over to AMXT 1501+DFMO.

Group IV

Active Comparator

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 4 locations

Recruiting

Arkansas Children's Hospital

Little Rock, United StatesOpen Arkansas Children's Hospital in Google Maps

Recruiting

Connecticut Children's Hospital

Hartford, United States

Recruiting

St. Joseph's Children's Hospital

Tampa, United States

Recruiting

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

Hershey, United States

Recruiting

4 Study Centers