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 2026-04-28
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 range21 Years
SexALL
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Inclusion criteria
✓. Age:
✓. Phase I-AYA (adolescents and young adult) Cohort: ≥12 years of age at enrollment.
✓. Phase I-Pediatric Cohort: \< 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.
✓. Phase II: ≤ 21 years of age at diagnosis (with possibly two different age-specific RP2Ds).
✓. Pathology
✓. Tumor assessment:
✓. Disease Status:
✓. Subjects must be able to swallow capsules.
Exclusion criteria
✕. BSA of \<0.25 m2
✕. Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
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
. Anti-cancer Agents: 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.
✕. Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
✕. Subjects 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.