Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas (NCT06465199) | Clinical Trial Compass
RecruitingPhase 1/2
Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas
United States289 participantsStarted 2026-05-13
Plain-language 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
Who can participate
Age range
26 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age:
. 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
. Tumor assessment:
. Disease Status:
. Participants must be able to swallow capsules.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial is still in Phase 1/Phase 2, which means the main focus right now is figuring out whether the combination of eflornithine and AMXT 1501 is safe — so how much is actually known yet about whether it might help my child's specific tumor type, like neuroblastoma or DIPG?
2Since Phase 1 is measuring adverse events and tolerability, what kinds of side effects have been seen so far with this drug combination, and are there any risks that would be especially important to consider given my child's current health?
3The trial is tracking progression-free survival as the key Phase 2 outcome — can you help me understand what 'progression-free survival' means in this context, and how that compares to what we might expect from standard treatment options for this diagnosis?
4My child has a specific tumor type listed in this trial — do you think their situation fits one of the cohorts being studied, and would enrolling in this trial mean delaying or replacing any standard-of-care treatment that might otherwise be available?
5Since this trial is actively recruiting, what would the treatment schedule actually look like for our family — things like how often we'd need to come in, where treatment takes place, and what kind of monitoring would be required throughout?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Phase I- Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Timeframe: 28 days
2
Phase II- Number of Cohort 1 participants with progression free survival (PFS) during study
Timeframe: 2 years plus 5 years follow up
3
Phase II- Number of Cohort 2-4 participants with progression free survival (PFS) during study
. Participants with CNS disease currently taking steroids must have been on a stable dose of steroids for at least one week and must not have progressive hydrocephalus at enrollment.
Exclusion criteria
. 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.