Actively Recruiting
Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas
Led by Milton S. Hershey Medical Center · Updated on 2026-04-13
289
Participants Needed
10
Research Sites
457 weeks
Total Duration
On this page
Sponsors
M
Milton S. Hershey Medical Center
Lead Sponsor
A
Aminex Therapeutics, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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
CONDITIONS
Official Title
Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Must be 21 years old or younger at diagnosis
- Phase I AYA cohort: 12 years or older at enrollment
- Phase I Pediatric cohort: under 12 years at enrollment, starting after safety review
- Confirmed diagnosis of relapsed or refractory neuroblastoma, embryonal tumor with multilayer rosettes, atypical teratoid rhabdoid tumor, newly diagnosed DIPG, relapsed or refractory Ewing sarcoma, or osteosarcoma
- Disease staging must be done during 28-day screening before treatment
- Neuroblastoma subjects with active or no active disease meeting specific disease stability and timing criteria
- ETMR/ATRT subjects with relapsed or refractory disease meeting specific stability and timing requirements
- Newly diagnosed DIPG subjects starting treatment 30 to 60 days post radiation therapy
- Ewing sarcoma and osteosarcoma subjects with relapsed or refractory disease meeting disease stability and timing requirements
- Must be able to swallow capsules
- Stable steroid dose for CNS disease patients for at least 1 week
- Fully recovered from prior chemotherapy and treatments within specified time frames
- Performance score of 60 or higher on Lansky or Karnofsky scale
- Adequate organ function including blood counts, liver, heart, and kidney function
- Negative pregnancy test and use of effective birth control if of childbearing potential
- Written informed consent obtained
You will not qualify if you...
- Body surface area less than 0.25 m2
- Currently receiving another investigational drug
- Currently receiving other anticancer agents or not fully recovered from prior chemotherapy effects
- Uncontrolled infection
- Unable or unlikely to comply with safety monitoring requirements as judged by investigator
- Progressive hydrocephalus in CNS disease patients
- Evidence of active graft vs. host disease after stem cell transplant
- Subjects with metastatic disease for DIPG
- Subjects with progression or recurrence after initial standard of care radiation for DIPG
- Subjects with H3K27m altered DMG outside the brainstem
- Subjects with a biopsy and no evidence of H3K27m mutation not meeting radiographic criteria
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 10 locations
1
University of Alabama/Children's of Alabama
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
3
Connecticut Children's Hospital
Hartford, Connecticut, United States, 06106
Actively Recruiting
4
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
5
Arnold Palmer Hospital for Children
Orlando, Florida, United States, 32806
Actively Recruiting
6
St. Joseph's Children's Hospital
Tampa, Florida, United States, 33614
Actively Recruiting
7
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States, 96813
Actively Recruiting
8
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, United States, 17033
Actively Recruiting
9
Monroe Carrell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, United States, 37232
Actively Recruiting
10
Children's Medical Center
Dallas, Texas, United States, 75235
Actively Recruiting
Research Team
B
BCC Enroll
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
4
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